FTD RESEARCH: The Value of Studies and Opportunities for Involvement

Transcription

1 FTD RESEARCH: The Value f Studies and Opprtunities fr Invlvement Sarah Lawrence, MS Research Prgram Manager Ann Fishman, MBA Sr. Research Crdinatr Weiyi Mu, ScM Genetic Cunselr Suzanne Dana Spuse/Caregiver Jhns Hpkins University Schl f Medicine

2 Ways t Get Invlved in FTD Research FTD Registry Research Studies Interviews and Surveys Observatinal Studies Clinical Trials Brain and Tissue Dnatin

3 FTD Registry

4 Interview studies and surveys Interviews talking abut yur experience abut FTD Surveys medical and/r psychlgical impact f FTD

5 Observatinal Studies: ARTFL and LEFFTDS

6 Observatinal Studies: T build a reliable FTD clinical research netwrk t supprt treatment and clinical trials that fcus n the preventin f FTD. T determine the clinical characteristics f spradic FTD syndrmes and the barriers t clinical trial participatin. T develp a familial FTD chrt fr clinical trials and bimarker studies. Infrmatin available at

7 Observatinal Studies: Men and wmen aged Able t walk with assistance Have a study partner Prject 1: Diagnsis f PSPS, svppa, CBS, bvftd, nfvppa, FTD/ALS Prject 2: Symptmatic r asymptmatic member f family with knwn genetic mutatin r family histry

8 Observatinal Studies: LEFFTDS Lngitudinal Evaluatin f Familial Frnttempral Dementia Subjects Primary gal: identify the mst rbust methds t track disease prgressin in familial FTD s that clinical trials f disease-mdifying therapies can be designed apprpriately Study ppulatin: Have a mutatin, r be a relative f an individual with a mutatin micrtubule assciated prtein tau (MAPT) prgranulin (PGRN; als knwn as granulin r GRN), r chrmsme 9 pen reading frame 72 (C9ORF72)

9 ARTFL and LEFFTDS Sites MGH CUMC UPENN JHU Bth ARTFL nly

10 Clinical Trials The gal f clinical research is t advance treatment, preventin, diagnsis and understanding f disease. There are many types: Preventin trials lk fr better ways t prevent a disease in peple wh have never had the disease r t prevent the disease frm returning. Better appraches may include medicines, vaccines, r lifestyle changes, amng ther things. Treatment trials test new treatments, new cmbinatins f drugs, r new appraches t surgery r radiatin therapy. Ref: NIH Clinical Trials and Yu: The Basics

11 Clinical Trials Lw-dse lithium fr behaviral symptms in bvftd Men and wmen aged Must have a diagnsis f bvftd, sv-ppa, nfv-ppa Must have a study partner r caregiver t attend study visits Must have evidence f behaviral symptms (agitatin, aggressin, disinhibitin, repetitive behavirs) There may be thers cming dwn the pipeline

12 Brain and Tissue Dnatin Obtain a definitive diagnsis Sme research can nly be dne n brain tissue Identify genetic mutatins Imprtant t plan ahead

13 Miscnceptins abut Research Participatin MYTH: Research is the same as clinical treatment FACT: Clinical treatment is patient-fcused, research has a brader utlk MYTH: Observatinal studies can replace clinical care FACT: Yu still need t see yur regular care team fr treatment management MYTH: Being seen at an academic medical center is the same as being enrlled in research FACT: Jining a research study invlves cnsent and is separate frm clinical visits

14 Miscnceptins abut Research Participatin MYTH: Anyne interested in a research study is eligible FACT: Studies ften have a specific ppulatin in mind MYTH: Everyne enrlled in a clinical trial will receive treatment FACT: Yu may receive a placeb MYTH: Research participants are treated like guinea pigs FACT: Research is bund by ethical guidelines

15 Genetic testing: research versus clinical testing An example f the bundaries between clinical and research CLINIC RESEARCH CLIA Certified? Yes N Will results be disclsed? Yes Nt Always When can I expect results? 1-2 Mnths Years Wh pays fr testing? Patient/Insurance Study What is the purpse? Fr Patient/Family Research

16 The Research Experience: What Happens When Yu Call? Participant cntacts study team Study team explains study Study team will discuss eligibility Be prepared t prvide a medicatin list, medical recrds, imaging recrds, etc. Schedule a visit Study team may send yu cnsent frm Cnsider when may be the best time f day t schedule/attend the study visit

17 The Research Experience: Advice fr a Research Day Subject signs the infrmed cnsent dcument Study team determines eligibility Review the cnsent frm, bring questins Bring legally authrized representative if needed t sign Visits may last several few hurs Be prepared: bring extra persn/snack Subject starts the study Be prepared t fllw study schedule If required, be prepared t incrprate new medicatin int daily rutine

18 (What shuld be) Frequently Asked Questins D I have t pay fr any f the study prcedures? Can transprtatin be prvided fr study visits? Is there reimbursement fr travel expenses? Can I keep any f the study infrmatin? Wh will versee my care during the study? Will I have access t the study medicatin after I finish the study? Can I cntinue t see the study dctr after I ve finished the study? Des participatin in ne study autmatically exclude me frm ther studies?

19 Where can I g t find ut abut ther studies? Resurces: AFTD.rg Clinicaltrials.gv Alz.rg - TrialMatch

20 Why Peple Participate Access t new treatments Cntribute t the greater gd Explre mre resurces Prvides a psitive curse f actin Help the next generatin

21 Thanks t ur research participants! ARTFL Observatinal Study Ann Fishman, ann.fishman@jhu.edu, Clinical Trials Sarah Lawrence, swdy1@jhmi.edu, Genetic Cunseling (clinical) Weiyi Mu, wmu2@jhmi.edu,