Disclosures and Funding
|
|
- Luke Heath
- 6 years ago
- Views:
Transcription
1 S894 A Phase 1, Open-Label Study to Determine the Safety, Tolerability, and Pharmacokinetics of Escalating Doses of LJPC-41 (Synthetic Human Hepcidin) in Patients With Iron Overload Ashutosh Lal, 1 Antonio Piga, 2 Vip Viprakasit, 3 James Maynard, 4 Antonis Kattamis, 5 Dan Yaeger, 6 Brian Byrnes, 6 Lakhmir Chawla, 6 George Tidmarsh 6 1 University of California San Francisco Benioff Children's Hospital, Oakland, CA, USA; 2 Università degli Studi di Torino, Turin, Italy; 3 Siriraj-Thalassemia Center, Mahidol University, Bangkok, Thailand; 4 CTI Clinical Research Center, Cincinnati, OH, USA; 5 National and Kapodistrian University of Athens, 'Aghia Sophia' Children's Hospital, Athens, Greece; 6 La Jolla Pharmaceutical Company, San Diego, CA, USA
2 Disclosures and Funding V. Viprakasit is a consultant for and receives research grants from La Jolla Pharmaceutical Company, Novartis, Sideris, Ferrokin, Vifor, Celgene, Protagonist, Biorad, Sanofi-Genzyme, SEBIA, Roche, Roche Diagnostics and Agios. A. Lal and A. Piga are consultants for and receives research grants from La Jolla Pharmaceutical Company D. Yaeger, B. Byrnes, L. Chawla, and G. Tidmarsh are employees of La Jolla Pharmaceutical Company Funding: This study was funded by La Jolla Pharmaceutical Company, San Diego, CA
3 Background Iron overload is a significant complication in patients with hereditary hemolytic anemias and hereditary hemochromatosis 1-3 Hepcidin Can cause damage to the liver, heart, and endocrine glands and may result in death reduced serum hepcidin levels characterize iron overload Endogenous hepcidin regulates dietary iron absorption and tissue distribution 4,5 Iron efflux to the bloodstream is restrained by hepcidin As a result, recycled iron remains in macrophages, and dietary iron absorption is inhibited 1. Porter J, Garbowski M. Hematology Am Soc Hematol Educ Program. 213;213: Leecharoenkiatx K et al. Asian Pac J Trop Med. 216;9: Gozzelino R, Arosio P. Int J Mol Sci. 216;17:E Liu J et al. Medicine (Baltimore). 216;95:e Sebastiani G et al. Front Pharmacol. 216;7:16.
4 Background (cont d) In animal models, increasing hepcidin levels by synthetic hepcidin injection or genetic induction has been shown to improve iron overload 1 A moderate increase in expression of hepcidin in β-thalassemic mice limited iron overload and improved anemia 2 Supply of exogenous hepcidin or increased expression of hepcidin ameliorated hemochromatosis and β-thalassemia in mice 3,4 These observations suggest that increasing hepcidin levels may help treat the abnormal iron absorption in individuals with β-thalassemia and related disorders 1. Ruchala P, Nemeth E. Trends Pharmacol Sci. 214;35(3): Gardenghi S et al. J Clin Invest. 21;12(12): Corradini E, et al. Gastroenterology. 21;139(5): Guo S et al. J Clin Invest. 213;123(4):
5 Potential effects of hepcidin agonists or activators on iron absorption under normal and β-thalassemic conditions Normal condition β thalassemia (NTDT) Drug treatment Gardenghi S et al. J Clin Invest. 21;12(12):
6 Background (cont d) LJPC-41, a synthetic human hepcidin, is being developed as a therapeutic intervention for iron overload Tested in 3 clinical studies to date 2 single-dose studies NHV1 in healthy volunteers (poster presentation PF47) TPP1 in patients at risk for iron overload 1 multidose study NHV2 in healthy volunteers
7 TPP1 Study Design and Objectives Population: Adult patients at risk of iron overload Design: Phase 1, open-label, dose-escalation study Study Duration: Single SC dose, 7-day observation LJPC-41 dosing at 1, 5, 1, 2, or 3 mg PRIMARY ENDPOINT Safety and tolerability TEAEs, laboratory values, ECGs, vital signs, and physical examination data SECONDARY ENDPOINT Serum iron level Eligible patients were adults with 1 of the following: Transfusion-dependent anemia and hemochromatosis Iron chelation therapy in the past 6 months Serum ferritin level >1 μg/l, or hemochromatosis* *Patients with hemochromatosis that required phlebotomy at least once every 2 months or had received iron chelation therapy in the past 6 months. ECG, electrocardiogram; SC, subcutaneous; TEAE, treatment-emergent adverse event.
8 Study Assessments Safety assessments TEAEs Physical examinations Laboratory evaluations Immunogenicity Visits/sampling on day 1 predose, day 8, and day 22 PK assessments Parameters of baselinecorrected serum LJPC-41 obtained by noncompartmental analysis Blood samples collected at predose and.5, 2, 4, 8, 24, 48, and 168 hours postdose PD assessments Effects on Serum iron level Transferrin level Transferrin saturation Ferritin level Samples collected at screening, day 1 predose, 24, 48, and 168 hours postdose PD, pharmacodynamics; PK, pharmacokinetics; TEAE, treatment-emergent adverse event.
9 Patient Baseline Characteristics LJPC-41 dose Parameter, n (%) 1 mg (n = 3) 5 mg (n = 3) 1 mg (n = 3) 2 mg (n = 6) 3 mg (n = 3) Total (N = 18) Age <65 years 65 years 3 (1) 1 (33.3) 2 (66.7) 3 (1) 6 (1) 3 (1) 16 (88.9) 2 (11.1) Gender Male Female 2 (66.7) 1 (33.3) 3 (1) 3 (1) 2 (33.3) 4 (66.7) 1 (33.3) 2 (66.7) 8 (44.4) 1 (55.6) Iron overload disease Hemochromatosis BL serum ferritin, mean (SD), 248 (592) ng/ml 1 (33.3) 3 (1) 3 (1) 3 (5.) 1 (33.3) 11 (61.1) Sickle cell disease BL serum ferritin, mean (SD), 1151 (7375) ng/ml 2 (66.7) 1 (16.7) 1 (33.3) 4 (22.2) TD β-thalassemia BL serum ferritin, mean (SD), (24) ng/ml 2 (33.3) 1 (33.3) 3 (16.7) BL, baseline; SD, standard deviation.
10 Summary of Safety Parameter, n (%) 1 mg (n = 3) 5 mg (n = 3) LJPC-41 dose 1 mg (n = 3) 2 mg (n = 6) 3 mg (n = 3) Number of TEAEs a Total (N = 18) Patients with 1 TEAEs 2 (66.7) 3 (1) 2 (66.7) 6 (1) 3 (1) 16 (88.9) Number of treatment-related b TEAEs Patients with 1 treatment-related b TEAEs 3 (1) 1 (33.3) 6 (1) 3 (1) 13 (72.2) Number of SAEs 1 c 1 Most frequently occurring TEAEs ( 2 patients) Injection site reactions 3 (1) 6 (1) 3 (1) 12 (66.7) Nausea 2 (66.7) 2 (11.1) ALT increased 1 (33.3) 1 (33.3) 2 (11.1) Decreased appetite 1 (33.3) 1 (16.7) 2 (11.1) Hypoesthesia 1 (33.3) 1 (33.3) 2 (11.1) No severe (grade 3) TEAEs, TEAEs leading to discontinuation, or deaths. Individual patient clinical labs reviewed by the study investigator suggested no clinically significant shifts. ALT, alanine aminotransferase; PD, pharmacodynamics; PK, pharmacokinetics; SAE, severe adverse event; TEAE, treatment-emergent adverse event. a 34 mild and 4 moderate in severity; b Possibly, probably, or definitely related to study drug administration; c Sickle cell pain crisis, not considered treatment-related, fully recovered.
11 Immunogenicity Assay and Results Positive Control/Assay Format Streptavidin ( ) conjugated to SulfoTag ( ) Sample or serum spiked with anti-ljpc surrogate antibody Hepcidin Species-specific anti-ig conjugated to biotin ( ) LJPC-41 coat Detection Control Species-specific anti-ig conjugated to biotin ( ) In 3 clinical trials, which included analysis of a total of 227 samples, all samples were confirmed negative for antibody to LJPC-41. Human IgG at 1 ng/ml LOD 125 ng/ml MSD High Bind assay plate Signal from positive control demonstrates the assay is properly detecting Ab to hepcidin MSD High Bind assay plate Signal generated by the detection control verifies the assay is capable of detecting at least 1 ng/ml of Ab
12 Mean Baseline-Corrected Serum LJPC Mean (SE) concentration, ng/ml Time, h Dose, mg Dose-dependent increase in exposure (except 3 mg) Peak concentrations occurred at 2-4 h Half-life ~6-13 h SE, standard error.
13 Mean Serum Iron Concentration at 8 Hours Postdose Mean Serum Iron Change From Baseline (SD), % Dose Response P= mg (n=3) 5 mg (n=3) 1 mg (n=3) 2 mg (n=6) 3 mg (n=3) LJPC-41 dosing cohorts Note: For analyses excluding the 3-mg dose group, a dose-dependent, statistically significant reduction in serum iron level was observed (P=.8 for dose response; not adjusted for multiple comparisons). SD, standard deviation.
14 Percent Changes in Iron Parameters in Individual Subjects (2- and 3-mg Dose Groups) 5 Ferritin Serum Iron TSAT% 2 mg (n=6) 3 mg (n=3) Percent change from baseline Time, h Ferritin Serum Iron TSAT% Time, h * * *One outlier patient in the 3-mg group had an exchange transfusion 2 days prior to study entry that may have altered iron homeostasis. SD, standard deviation; TSAT%, transferrin saturation.
15 Mean Serum Iron Concentration at 8 Hours Postdose Post Hoc Analysis, Excluding 1 Subject Outlier Mean Serum Iron Change From Baseline (SD), % Dose Response P= mg (n=3) 5 mg (n=3) 1 mg (n=3) 2 mg (n=6) 3 mg (n=2) LJPC-41 dosing cohorts SD, standard deviation.
16 Sustained Iron-Lowering Effect With Comparable PK Exposures Between Healthy Subjects and Patients Mean (SE) value Hepcidin Exposures AUC, (ng.h/ml) ng h/ml Dose, mg Healthy Subjects Patients C max, ng/ml 1 2 Heathy subjects (1 mg: n=6; 2 mg: n=6) Patients (1 mg: n=3; 2 mg: n=6) Mean (SE) iron %change Iron-Lowering Effect Healthy Subjects (NHV1) Time, h Mean (SE) iron %change Dose, mg 1 2 Patients (TPP1) Time, h PK exposures (AUC and C max ) are generally comparable between healthy subjects and patients Longer and sustained iron-lowering effect observed in patients returning toward baseline after 1 week PD effect likely due to difference in iron hemostasis and regulation between the 2 populations AUC, Area under the curve; C max, maximum drug concentration.
17 Improved Hepcidin Production Human hepcidin is difficult to manufacture, with challenges around stability and aggregate formation The original methods for synthesis of hepcidin resulted in ~15% higher molecular weight hepcidin aggregate that was prone to further aggregation under in-use conditions The updated proprietary process for producing hepcidin results in <1% aggregate that is stable under in-use conditions HPLC Trace Red: Original process Black: Improved process High-order aggregate HPLC, high-performace liquid chromatography.
18 Improved Formulation Enhanced Bioavailability 4 AUC, ng.h/ml 4 2 C max ng.h/ml Dose, mg Dose, mg Formulation Improved (NHV2) Original (NHV1) Original (TPP1) A new improved formulation increased subcutaneous bioavailability (AUC and C max ) up to 3-fold.
19 Improved Formulation- Enhanced Iron-Lowering Effect Mean (SE) Iron %Change at Hour Dose, mg Formulation Improved (NHV2) Original (NHV1) Original (TPP1) Enhanced bioavailability resulted in greater iron reduction at the same dose.
20 Conclusions LJPC-41 was well tolerated at doses between 1 mg and 3 mg, with the maximum iron-lowering effect observed at 2 mg LJPC-41 showed significant decreases in serum iron levels compared with baseline, which were sustained in most patients for up to 8 days In comparison to healthy adults, in whom LJPC-41 caused a decrease in serum iron levels that returned to baseline levels within 48 hours, 1 the iron-lowering effect in iron overload patients was more sustained 1. Yaeger D et al. Presented at the 23rd Congress of the European Hematology Association; June 14-17, 218; Stockholm, Sweden; poster PF47.
21 Conclusions (cont d) New formulation has improved PK exposure and PD effect with no corresponding increase of injection site reaction severity or duration Additional studies are ongling to further explore the iron-regulating effects of LJPC-41 in patients with iron-overload disorders 1. Pivotal study in patients with transfusion-dependent beta thalassemia (HELIOS) 1 patient, 12 mo., parallel group study, evaluating the effects of LJPC-41 on myocardial iron 2. Phase 2 study in patients with hereditary hemochromatosis (HERCULES Study) 6 patient, 4 mo., single-blind, placebo-controlled study evaluating the effects of LJPC-41 on TSAT and phlebotomy requirements
LJPC-401 Phase 1 Results and Development Update. September 7, 2016
LJPC-401 Phase 1 Results and Development Update September 7, 2016 Forward-Looking Statements These slides contain "forward-looking" statements within the meaning of the Private Securities Litigation Reform
More informationPresented at the 60th Annual Meeting of the American Society of Hematology (ASH); December 1 4, 2018; San Diego, CA, USA.
The BELIEVE Trial: Results of a Phase 3, Randomized, Double-Blind, -Controlled Study of in Adult Beta-Thalassemia Patients Who Require Regular Red Blood Cell (RBC) Transfusions Maria Domenica Cappellini,
More informationClinical Trial Results Summary Study EN
Study Number: EN3288-113 Title of Study: A Double-blind, Dose-Ranging, Pilot Study to Evaluate the Safety, Subjective Effects, and Pharmacokinetics of Oxymorphone Hydrochloride in Healthy Subjects Who
More informationCorporate Presentation
Corporate Presentation Developing Innovative Therapies for Patients Suffering from Life-threatening Diseases NASDAQ: LJPC January 2018 Forward-Looking Statements These slides contain forward-looking statements
More informationCorporate Presentation
Corporate Presentation Developing Innovative Therapies for Patients Suffering from Life-threatening Diseases NASDAQ: LJPC March 2019 Forward-Looking Statements These slides contain forward-looking statements
More informationSponsor / Company: Sanofi Drug substance: SAR236553/REGN727 (alirocumab)
These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert in the country of prescription. Sponsor / Company: Sanofi Drug substance:
More informationSponsor: Sanofi Drug substance(s): GZ316455
These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert in the country of prescription. Sponsor: Sanofi Drug substance(s):
More informationMipomersen (ISIS ) Page 2 of 1979 Clinical Study Report ISIS CS3
(ISIS 301012) Page 2 of 1979 2 SYNOPSIS ISIS 301012-CS3 synopsis Page 1 Title of Study: A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics,
More informationStudy Centers: This study was conducted in 2 centers in Italy.
Title of Trial: A randomised, double-blind, placebo-controlled, two-period, two-sequence-crossover interaction study to assess the effect of safinamide on levodopa pharmacokinetics in subjects with Parkinson
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationCorporate Presentation
Corporate Presentation Developing Innovative Therapies for Patients Suffering from Life-threatening Diseases NASDAQ: LJPC September 2016 Forward-Looking Statements These slides contain "forward-looking"
More informationThis clinical study synopsis is provided in line with Boehringer Ingelheim s Policy on Transparency and Publication of Clinical Study Data.
abcd Clinical Study Synopsis for Public Disclosure This clinical study synopsis is provided in line with s Policy on Transparency and Publication of Clinical Study Data. The synopsis which is part of the
More informationTo assess safety profiles: significant laboratory changes and adverse events (AEs).
These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert in the country of prescription. Sponsor / Company: Sanofi Drug substance(s):
More informationHydrocodone/Acetaminophen Extended-Release Tablets M Clinical Study Report R&D/09/1109
2.0 Synopsis Abbott Laboratories Individual Study Table Referring to Part of Dossier: (For National Authority Use Only) Name of Study Drug: ABT-712 Volume: Hydrocodone/Acetaminophen Extended-Release Name
More informationPresentation #: OP221
Pharmacokinetics, Safety and Tolerability of, a Novel Prodrug of Tenofovir, Administered as Ascending Multiple Doses to Healthy Volunteers and HBV-Infected Subjects Presentation #: OP22 Tawesak Tanwandee,
More informationResults. Subject Disposition and Demographics Of 37 enrolled subjects, 23 (62.2%) completed the study
Poster 5-20 Effect of Gastric ph on the Bioavailability of in Healthy Subjects James Longstreth, PhD, Marijke H. Adams, PharmD, PhD, 2 Vasi Sperry, PhD, 3 Dan Kajdasz, PhD, 3 Carol R. Reed, MD 3 Longstreth
More informationPFIZER INC. THERAPEUTIC AREA AND FDA APPROVED INDICATIONS: See USPI
PFIZER INC. These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert. For publications based on this study, see associated bibliography.
More informationSYNOPSIS. Number of subjects: Planned: 22 Randomized: 23 Treated: 23. Evaluated: Pharmacodynamic: 22 Safety: 23 Pharmacokinetics: 22
SYNOPSIS Title of the study: A randomized, cross-over, open, euglycemic clamp study on the relative bioavailability and activity of 0.6 U/kg insulin glargine and 20 µg lixisenatide, given as on-site mix
More information10/27/2013. Study Design. Disclosures
/27/23 Disclosures Drug Exposure Limitations of Oral Methotrexate (MTX) at Doses mg May be Overcome by Using a Subcutaneous MTX Auto-Injector in Patients With Rheumatoid Arthritis (RA) M.H. Schiff, L.S.
More informationCorporate Presentation
Corporate Presentation Developing Innovative Therapies for Patients Suffering from Life-threatening Diseases NASDAQ: LJPC March 2017 Forward-Looking Statements These slides contain forward-looking statements
More informationLVHN Scholarly Works. Lehigh Valley Health Network. Nelson Kopyt DO, FASN, FACP Lehigh Valley Health Network,
Lehigh Valley Health Network LVHN Scholarly Works Department of Medicine Efficacy and Safety of Oral Ferric Maltol (FM) in Treating Iron-Deficiency Anemia (IDA) in Patients with Chronic Kidney Disease
More informationClinical Study Synopsis for Public Disclosure
abcd Clinical Study Synopsis for Public Disclosure This clinical study synopsis is provided in line with s Policy on Transparency and Publication of Clinical Study Data. The synopsis - which is part of
More informationThis clinical study synopsis is provided in line with Boehringer Ingelheim s Policy on Transparency and Publication of Clinical Study Data.
abcd Clinical Study for Public Disclosure This clinical study synopsis is provided in line with s Policy on Transparency and Publication of Clinical Study Data. The synopsis which is part of the clinical
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationSYNOPSIS OF RESEARCH REPORT (PROTOCOL BC20779)
TITLE OF THE STUDY / REPORT No. / DATE OF REPORT INVESTIGATORS / CENTERS AND COUNTRIES Clinical Study Report Protocol BC20779: Multicenter, double-blind, randomized, placebo-controlled, dose ranging phase
More information(For National Authority Use Only) Name of Study Drug: to Part of Dossier:
2.0 Synopsis Abbott Laboratories Individual Study Table Referring to Part of Dossier: (For National Authority Use Only) Name of Study Drug: Volume: Vicodin CR Name of Active Ingredient: Page: Hydrocodone/Acetaminophen
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationGSK Medicine: Study Number: Title: Rationale: Phase: Study Period: Study Design: Centres: Indication: Treatment: Objectives:
The study listed may include approved and non-approved uses, formulations or treatment regimens. The results reported in any single study may not reflect the overall results obtained on studies of a product.
More informationSYNOPSIS. First subject enrolled 15 August 2003 Therapeutic confirmatory (III) Last subject completed 03 February 2005
Drug product: SYMBICORT pmdi 160/4.5 μg Drug substance(s): Budesonide/formoterol Study code: SD-039-0728 Edition No.: FINAL Date: 27 February 2006 SYNOPSIS A 52-week, randomized, double-blind, single-dummy,
More information2018 F. Hoffmann-La Roche Ltd. All rights reserved.
PRELIMINARY EVIDENCE FOR PHARMACODYNAMIC EFFECTS OF RG7916 IN JEWELFISH A STUDY IN PATIENTS WITH SPINAL MUSCULAR ATROPHY WHO PREVIOUSLY PARTICIPATED IN A STUDY WITH ANOTHER SMN2-SPLICING TARGETING THERAPY
More informationSYNOPSIS. Study centre(s) The study was performed in Denmark, Norway and Sweden at 20 sites.
Drug substance(s): AZD0837 Edition No.: 1 Study code: D1250C00007 Date: 22 May, 2006 SYNOPSIS (For national authority use only) A Controlled, Randomised, Parallel, Multicentre Study to Assess Safety and
More informationClinical Study Synopsis for Public Disclosure
abcd Clinical Study for Public Disclosure This clinical study synopsis is provided in line with s Policy on Transparency and Publication of Clinical Study Data. The synopsis - which is part of the clinical
More informationPage: 17 December 2012 (Study M13-692) 22 October 2013 (Study M13-692)
2.0 Synopsis AbbVie Inc. Name of Study Drug: Adalimumab Name of Active Ingredient: D2E7 Individual Study Table Referring to Part of Dossier: Volume: Page: (For National Authority Use Only) Title of Studies:
More informationClinical Study Synopsis for Public Disclosure
abcd Clinical Study Synopsis for Public Disclosure This clinical study synopsis is provided in line with s Policy on Transparency and Publication of Clinical Study Data. The synopsis - which is part of
More informationKevin Fitzgerald, PhD
A Subcutaneously Administered Investigational RNAi Therapeutic (ALN-PCSsc), Targeting PCSK9 for the Treatment of Hypercholesterolemia: Initial Phase 1 Study Results Kevin Fitzgerald, PhD Co-authors: Amy
More informationStudy Day 1 Study Days 2 to 9 Sequence 1 Placebo for moxifloxacin Study Days 2 to 8: placebo for pazopanib (placebopaz) 800 mg;
The study listed may include approved non-approved uses, formulations or treatment regimens. The results reported in any single study may not reflect the overall results obtained on studies of a product.
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationCorporate Presentation
Developing Innovative Therapies for Patients Suffering from Life-threatening Diseases Corporate Presentation NasdaqCM: LJPC July 2014 Forward-Looking Statements These slides contain "forward-looking" statements
More informationSponsor / Company: Sanofi Drug substance(s): SAR302503
These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert in the country of prescription. Sponsor / Company: Sanofi Drug substance(s):
More informationSYNOPSIS. Administration: subcutaneous injection Batch number(s):
SYNOPSIS Title of the study: A randomized, double-blind, placebo-controlled, 2-arm parallel-group, multicenter 24-week study followed by an extension assessing the efficacy and safety of AVE0010 on top
More informationClinical Study Synopsis
Clinical Study Synopsis This Clinical Study Synopsis is provided for patients and healthcare professionals to increase the transparency of Bayer's clinical research. This document is not intended to replace
More informationSponsor/Company: sanofi-aventis Drug substance: Elitek/Fasturtec (rasburicase, SR29142)
These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert in the country of prescription. Sponsor/Company: sanofi-aventis Drug
More informationINDIVIDUAL STUDY TABLE REFERRING TO PART OF THE DOSSIER Volume: Page:
SYNOPSIS Protocol No.: CR004357 Title of Study: A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of 50 and 100 mg eq. of Paliperidone Palmitate in Subjects With
More informationNuovi Approcci alla Ferrochelazione
Il Deficit di PKD 1 patient day Nuovi Approcci alla rrochelazione M. Domenica Cappellini Fondazione Ca Granda Policlinico Università di Milano Milano May 16 2015 Genetic and acquired iron overload Genetic
More informationB Kahl 1, M Hamadani 2, P Caimi 3, E Reid 4, K Havenith 5, S He 6, JM Feingold 6, O O Connor 7
First Clinical Results of ADCT-42, a Novel Pyrrolobenzodiazepine-Based Antibody Drug Conjugate (ADC), in Relapsed/Refractory B-cell Lineage Non-Hodgkin Lymphoma B Kahl 1, M Hamadani 2, P Caimi 3, E Reid
More information1st International Working Group on Thalassemia:
1st International Working Group on Thalassemia: IS IT TIME TO REVISIT CLASSIFICATION OF THALASSEMIA SYNDROMES? CAMPUS OF HEMATOLOGY "Franco e Piera Cutino" A.O.R. "Villa Sofia - V. Cervello" Palermo ()
More informationPFIZER INC. THERAPEUTIC AREA AND FDA APPROVED INDICATIONS: See United States Package Insert (USPI)
PFIZER INC. These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert. For publications based on this study, see associated bibliography.
More information1. BACKGROUND. Lovastatin β-hydroxyacid No effect on methane production Inhibits cholesterol synthesis
1. BACKGRUND Methane production by the archeon Methanobrevibacter smithii (M. smithii) is a ubiquitous process in the intestine, disposing of hydrogen and other by-products formed during carbohydrate digestion
More informationStudy Population: 12 years and older A EF Calcipotriene Foam, 0.005%
Study No.: CAL.203 Title: A Randomized, Open-Label Study to Assess the Bioavailability of Emulsion Formulation Foam, 0.005%, and Dovonex, 0.005%, in Patients with Mild to Moderate Plaque-Type Psoriasis
More informationHemosiderin. Livia Vida 2018
Hemosiderin Livia Vida 2018 Questions Histochemical caracteristics of the different pigments. Exogenous pigments. Hemoglobinogenic pigments. Causes and forms of jaundice. Hemoglobinogenic pigments. Pathological
More informationThis clinical study synopsis is provided in line with Boehringer Ingelheim s Policy on Transparency and Publication of Clinical Study Data.
abcd Clinical Study for Public Disclosure This clinical study synopsis is provided in line with s Policy on Transparency and Publication of Clinical Study Data. The synopsis which is part of the clinical
More informationNovel Formulations to Modify Mealtime Insulin Kinetics
Novel Formulations to Modify Mealtime Insulin Kinetics Alan Krasner, Roderike Pohl, Patrick Simms, Philip Pichotta, Robert Hauser, Errol De Souza Biodel, Inc. Danbury, CT Disclosure All authors are employees
More informationIron Overload in Sickle Cell Disease Review of Cause and Treatment
Iron Overload in Sickle Cell Disease Review of Cause and Treatment Susan M. Carson RN, MSN, CPNP Nurse Practitioner III Hematology Program Children s Hospital Los Angeles Objectives Describe the effect
More informationLuspatercept Increases Hemoglobin, Decreases Transfusion Burden, and Improves Patient-Reported Outcomes in Adults with Beta-Thalassemia
Luspatercept Increases Hemoglobin, Decreases Transfusion Burden, and Improves Patient-Reported Outcomes in Adults with Beta-Thalassemia Antonio G. Piga, MD 1, Immacolata Tartaglione, MD 2, Rita Gamberini,
More informationThis clinical study synopsis is provided in line with Boehringer Ingelheim s Policy on Transparency and Publication of Clinical Study Data.
abcd Clinical Study Synopsis for Public Disclosure This clinical study synopsis is provided in line with Boehringer Ingelheim s Policy on Transparency and Publication of Clinical Study Data. The synopsis
More informationEffect of Multiple-Dose Ketoconazole and the Effect of Multiple-Dose Rifampin on Pharmacokinetics (PK) of the HCV NS3 Protease Inhibitor Asunaprevir
Effect of Multiple-Dose Ketoconazole and the Effect of Multiple-Dose Rifampin on Pharmacokinetics (PK) of the HCV NS3 Protease Inhibitor Asunaprevir Eley T, 1 He B, 1 Huang S-P, 2 Stonier M, 1 Bedford
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationHormone related problems (Endocrinopathies and osteoporosis) Vincenzo de Sanctis Ferrara.
Hormone related problems (Endocrinopathies and osteoporosis) Vincenzo de Sanctis Ferrara vdesanctis@libero.it 6 th EUROPEAN SYMPOSIUM ON RARE ANAEMIAS 1 st Dutch-Belgian meeting for patients and health
More informationSYNOPSIS Final Clinical Study Report for Study AI444031
Name of Sponsor/Company: Bristol-Myers Squibb Name of Finished Product: Name of Active Ingredient: () Individual Study Table Referring to the Dossier (For National Authority Use Only) SYNOPSIS for Study
More informationOral Soluble Film Products for Epilepsy: Clobazam (COSF) and Diazepam (DBSF)
Oral Soluble Film Products for Epilepsy: Clobazam (COSF) and Diazepam (DBSF) Michael A. Rogawski, M.D., Ph.D. Professor of Neurology and Pharmacology School of Medicine University of California, Davis
More informationAN2728 Clinical Data in Atopic Dermatitis
AN2728 Clinical Data in Atopic Dermatitis Karl Beutner, MD, PhD 1 Overview of AN2728 in Atopic Dermatitis Target Product Profile Safe and effective topical therapy for atopic dermatitis Efficacy in the
More informationSupporting Materials for a 31-Day Study of Cobalt(II)chloride Ingestion in Humans: Pharmacokinetics and Clinical Effects
Supporting Materials for a 31- Study of Cobalt(II)chloride Ingestion in Humans: Pharmacokinetics and Clinical Effects Brent L. Finley a, Kenneth M. Unice b, Brent D. Kerger c, Joanne M. Otani a, Dennis
More informationJohnson & Johnson Pharmaceutical Research & Development, L.L.C.
SYNOPSIS Issue Date: 27 April 2009 Document No.: EDMS-PSDB-9908562:2.0 Name of Sponsor/Company Name of Finished Product Name of Active Ingredient Johnson & Johnson Pharmaceutical Research & Development,
More informationSponsor: Sanofi Drug substance(s): SAR342434
These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert in the country of prescription. Sponsor: Sanofi Drug substance(s):
More informationA prospective clinical trial of autologous TARGT prolonged EPO secretion showed EPO-independence in EPO-dependent ESRD patients
A prospective clinical trial of autologous TARGT prolonged EPO secretion showed EPO-independence in EPO-dependent ESRD patients September 19, 2015 TARGT TM (Transduced Autologous Restorative Gene Therapy)
More informationManagement of anemia in CKD
Management of anemia in CKD Pierre Cochat, MD PhD Professor of Pediatrics Chair, Pediatrics & Pediatric Surgery Department Head, Center for Rare Renal Diseases Néphrogones Hospices Civils de Lyon & University
More informationClinical Study Synopsis for Public Disclosure
abcd Clinical Study Synopsis for Public Disclosure This clinical study synopsis is provided in line with s Policy on Transparency and Publication of Clinical Study Data. The synopsis - which is part of
More informationMicrocytic Hypochromic Anemia An Approach to Diagnosis
Microcytic Hypochromic Anemia An Approach to Diagnosis Decreased hemoglobin synthesis gives rise to microcytic hypochromic anemias. Hypochromic anemias are characterized by normal cellular proliferation
More informationClinical Trials A Practical Guide to Design, Analysis, and Reporting
Clinical Trials A Practical Guide to Design, Analysis, and Reporting Duolao Wang, PhD Ameet Bakhai, MBBS, MRCP Statistician Cardiologist Clinical Trials A Practical Guide to Design, Analysis, and Reporting
More informationAPDW 2016 Poster No. a90312
APDW 2016 Poster No. a90312 SYN-010, a Proprietary Modified-Release Formulation of Lovastatin Lactone, Lowered Breath Methane and Improved Stool Frequency in Patients with IBS-C Results of a multi-center,
More informationSubjects from the Safety Population who had GSK PK parameter estimates from any portion of the study. Cohort 1 (N=8)
The study listed may include approved and non-approved uses, formulations or treatment regimens. The results reported in any single study may not reflect the overall results obtained on studies of a product.
More informationPresented at the Annual Congress of ECTRIMS, October 11, 2018, Berlin, Germany
Final Results of a Placebo Controlled, Phase 2 Multicenter Study of Ublituximab (UTX), a Novel Glycoengineered Anti-CD20 Monoclonal Antibody (mab), in Patients with Relapsing Forms of Multiple Sclerosis
More informationPFIZER INC. These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert.
Public Disclosure Synopsis Protocol A7772 September 25 Final PFIZER INC. These results are supplied for informational purposes only. Prescribing decisions should be made based on the approved package insert.
More informationSYNOPSIS. Study center(s) This study was conducted in the United States (128 centers).
Drug product: Drug substance(s): Document No.: Edition No.: Study code: Date: SYMBICORT pmdi 160/4.5 µg Budesonide/formoterol SD-039-0725 17 February 2005 SYNOPSIS A Twelve-Week, Randomized, Double-blind,
More informationPFIZER INC. These results are supplied for informational purpose only. Prescribing decisions should be made based on the approved package insert.
Public Disclosure Synopsis Protocol A3924 4 November 24 Final PFIZER INC. These results are supplied for informational purpose only. Prescribing decisions should be made based on the approved package insert.
More informationEmricasan (IDN-6556) administered orally for 28 days lowers portal pressure in patients with compensated cirrhosis and severe portal hypertension
Emricasan (IDN-6556) administered orally for 28 days lowers portal pressure in patients with compensated cirrhosis and severe portal hypertension Guadalupe Garcia-Tsao, Michael Fuchs, Mitchell Shiffman,
More informationSynopsis. Clinical Report Synopsis for Protocol Name of Company: Otsuka Pharmaceutical Development & Commercialization, Inc.
Synopsis Clinical Report Synopsis for Protocol 197-02-220 Name of Company: Otsuka Pharmaceutical Development & Commercialization, Inc. Name of Product: Tetomilast (OPC-6535) Study Title: A Phase 3, Multicenter,
More information1st International Working Group on Thalassemia:
1st International Working Group on Thalassemia: IS IT TIME TO REVISIT CLASSIFICATION OF THALASSEMIA SYNDROMES? CAMPUS OF HEMATOLOGY "Franco e Piera Cutino" A.O.R. "Villa Sofia - V. Cervello" Palermo ()
More informationSYNOPSIS. Drug substance(s) Budesonide/formoterol Document No. Edition No. Study code SD Date 16 December 2004
Drug product SYMBICORT pmdi 160/4.5 µg SYNOPSIS Drug substance(s) Budesonide/formoterol Document No. Edition No. Date 16 December 2004 A Twelve-Week, Randomized, Double-Blind, Double-Dummy, Placebo-Controlled
More informationClinical Study Synopsis for Public Disclosure
abcd Clinical Study Synopsis for Public Disclosure This clinical study synopsis is provided in line with s Policy on Transparency and Publication of Clinical Study Data. The synopsis - which is part of
More informationThe clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only.
The clinical trial information provided in this public disclosure synopsis is supplied for informational purposes only. Please note that the results reported in any single trial may not reflect the overall
More informationThe study listed may include approved and non-approved uses, formulations or treatment regimens. The results reported in any single study may not
The study listed may include approved and non-approved uses, formulations or treatment regimens. The results reported in any single study may not reflect the overall results obtained on studies of a product.
More informationSponsor. Novartis Pharmaceuticals Corporation Generic Drug Name. Agomelatine Therapeutic Area of Trial. Major depressive disorder Approved Indication
Clinical Trial Results Database Page 1 Sponsor Novartis Pharmaceuticals Corporation Generic Drug Name Therapeutic Area of Trial Major depressive disorder Approved Indication Investigational drug Study
More information2011 ASH Annual Meeting Targeting the Hepcidin Pathway with RNAi Therapeutics for the Treatment of Anemia. December 12, 2011
211 ASH Annual Meeting Targeting the Hepcidin Pathway with RNAi Therapeutics for the Treatment of Anemia December 12, 211 Hepcidin is Central Regulator of Iron Homeostasis Hepcidin is liver-expressed,
More informationThis clinical study synopsis is provided in line with Boehringer Ingelheim s Policy on Transparency and Publication of Clinical Study Data.
abcd Clinical Study Synopsis for Public Disclosure This clinical study synopsis is provided in line with Boehringer Ingelheim s Policy on Transparency and Publication of Clinical Study Data. The synopsis
More informationProduct: Denosumab (AMG 162) Clinical Study Report: month Primary Analysis Date: 21 November 2016 Page 1
Date: 21 November 2016 Page 1 2. SYNOPSIS Name of Sponsor: Amgen Inc., Thousand Oaks, CA, USA Name of Finished Product: Prolia Name of Active Ingredient: denosumab Title of Study: Randomized, Double-blind,
More informationComparison of Omadacycline and Tigecycline Pharmacodynamics in the Plasma, Epithelial Lining Fluid, and Alveolar Macrophages in Healthy Subjects
Comparison of Omadacycline and Tigecycline Pharmacodynamics in the Plasma, Epithelial Lining Fluid, and Alveolar Macrophages in Healthy Subjects Karolyn S. Horn, Mark H. Gotfried, Judith N. Steenbergen,
More informationNuove terapie in ambito Nefrologico: Etelcalcetide (AMG-416)
Nuove terapie in ambito Nefrologico: Etelcalcetide (AMG-416) Antonio Bellasi, MD, PhD U.O.C. Nefrologia & Dialisi ASST-Lariana, Ospedale S. Anna, Como, Italy Improvement of mineral and bone metabolism
More information23-Aug-2011 Lixisenatide (AVE0010) - EFC6014 Version number: 1 (electronic 1.0)
SYNOPSIS Title of the study: A randomized, double-blind, placebo-controlled, parallel-group, multicenter 24-week study followed by an extension assessing the efficacy and safety of AVE0010 on top of metformin
More informationPreclinical Requirements for Therapeutic Studies in Humans with Advanced Cancer
Preclinical Requirements for Therapeutic Studies in Humans with Advanced Cancer Scott Laurie MD, FRCPC The Ottawa Hospital Cancer Centre Associate Professor, University of Ottawa Disclosures I have no
More informationHTX-011, a Proprietary, Unique, Long-Acting Local Anesthetic, Reduces Acute Postoperative Pain Intensity and Opioid Consumption Following Bunionectomy
HTX-011, a Proprietary, Unique, Long-Acting Local Anesthetic, Reduces Acute Postoperative Pain Intensity and Opioid Consumption Following Bunionectomy Eugene Viscusi, 1 Oscar DeLeon-Casasola, 2 TJ Gan,
More informationCorporate Medical Policy Chelation Therapy
Corporate Medical Policy Chelation Therapy File Name: Origination: Last CAP Review: Next CAP Review: Last Review: chelation_therapy 12/1995 2/2015 2/2016 2/2015 Description of Procedure or Service Chelation
More informationDiscovery. Hepcidin Today. Hepcidin: discovery June 2000: Man: Plasma ultrafiltrate Liver Expressed Antimicrobial Peptide
Hepcidin Today Rachel van Swelm 10 05 2018 ISLH www.radboud ironcenter.com; www.hepcidinanalysis.com Discovery Hepcidin: discovery June 2000: Man: Plasma ultrafiltrate Liver Expressed Antimicrobial Peptide
More informationRonald Goldwater 1 Azra Hussaini
Clin Pharmacokinet (217) 56:83 813 DOI 1.17/s4262-17-536-2 ORIGINAL RESEARCH ARTICLE Comparison of a Novel Formulation of Abiraterone Acetate vs. the Originator Formulation in Healthy Male Subjects: Two
More informationISSN Asian Journal of Medical and Pharmaceutical Researches Asian J. Med. Pharm. Res. 3(3): 93-97, 2013
\\\\ 2013, Scienceline Publication www.science-line.com ISSN 2322-4789 Asian Journal of Medical and Pharmaceutical Researches Asian J. Med. Pharm. Res. 3(3): 93-97, 2013 AJMPR Comparison of Therapeutic
More informationAbstract 1 INTRODUCTION RESEARCH ARTICLE
Received: 19 August 2016 Revised: 23 January 2017 Accepted: 26 January 2017 DOI 10.1002/ajh.24668 RESEARCH ARTICLE New film-coated tablet formulation of deferasirox is well tolerated in patients with thalassemia
More informationCorporate Medical Policy
Corporate Medical Policy Chelation Therapy File Name: Origination: Last CAP Review: Next CAP Review: Last Review: chelation_therapy 12/1995 2/2018 2/2019 2/2018 Description of Procedure or Service Chelation
More information