A Guide to Your Visits for the FAiRE LGS Study

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1 A Guide to Your Visits for the FAiRE LGS Study ( Fenfluramine Assessment in Rare Epilepsy) Thank you for enrolling in the FAiRE-LGS research study. This study will assess whether an investigational drug called ZX008 (Fenfluramine Hydrochloride, oral solution) can reduce the number of seizures that result in drops experienced by children and adults with Lennox-Gastaut syndrome (LGS) when given in combination with other antiseizure medications. This booklet tells you what you can expect to happen at each study visit. Please contact your study doctor or a member of his/her team if you have any additional questions. A Brief Reminder Following screening, participants are randomly assigned (by chance) to receive either one of two doses of the investigational study drug or placebo (a look-a-like that contains no active drug). Participants are two times more likely to receive the investigational study drug (at one of two different doses) than a placebo. Study drug or placebo are added to current medications and therefore participants should continue to take their normal antiseizure medication at a stable dose (the dose must not change) for the entire length of the study. Both the investigational study drug and the placebo are solutions taken by mouth with or without food, twice a day (morning and evening about 12 hours apart). As well as taking the assigned study medication, you will need to record in an electronic diary when you take the study medication and when seizures occur. When participation in the FAiRE-LGS study ends, participants may be able to join Part 2, a longer-term extension (follow-on) study. During the extension study, all participants will be given the investigational study drug, even if they were previously assigned a placebo.

2 Visit 1 (At the Clinic, Day -28) Visit 7 (Telephone, Day 29) Conduct informed consent conversation Review medical, neurological, and epilepsy history Ensure criteria for trial participation are met Record demographics Vital signs, height and weight Electrocardiogram (), a painless recording of the heart s electrical activity Visit 2 (Telephone, Day -15) Visit 3 (At the Clinic, Day -1) Ensure all criteria to receive study drug are met Visit 4 (Telephone, Day 4) Visit 5 (Telephone, Day 8) Visit 6 (At the Clinic, Day 15) Physical and neurological exam Echocardiogram (), a painless ultrasound of the heart Physical and neurological exam Note: Please confirm with your study doctor whether you should wait to take the morning doses of your anti-seizure medications prior to this clinic visit Visit 8 (At the Clinic, Day 43) Note: Please confirm with your study doctor whether you should wait to take the morning doses of your anti-seizure and study medication prior to this clinic visit Blood collection for pharmacokinetic (PK) assessment at 4 time points (1 hour before, and 1, 2 and 4-6 hours after the morning study drug dose) Participant symptom and treatment response Visit 9 (Telephone, Day 57) Visit 10 (At the Clinic, Day 71) Please confirm with your study doctor whether you should wait to take the morning doses of your anti-seizure and study medication prior to this clinic visit. Participant symptom and treatment response Discuss participation in Part 2, if applicable At this visit, if the study doctor feels you may be a good candidate, you may be invited to continue in Part 2 of the study, the extension part where all participants receive the investigational drug. Visit 11 (Telephone, Day 85) 2 3

3 Visit 12 (At the Clinic, Day 99) Note: If participating in Part 2 of study, Informed Consent Form must be completed by this visit Vital signs, height and weight Physical and neurological examination Return leftover study medicine; dispense transition study medicine to either discontinue or move to Part 2 of the study Post-Dosing Follow-up Visit 13 (At the Clinic, Day 113) This visit is only applicable to those who do not participate in Part 2. If participating in Part 2, the next visit after Visit 12 is Visit 15. Cardiac Safety Follow-up Visit 14 (At the Clinic) This visit is only applicable to those who do not participate in Part 2. Cardiac follow-up visit to examine the heart Return leftover transition study medicine related to cardiovascular health If you took the study drug for more than 13 weeks, you may repeat these procedures at another 1 or 2 safety follow-up visits, as necessary Welcome to FAiRE-LGS Part 2 By Visit 10, if you have tolerated the study drug, taken it as prescribed, kept up with the electronic diary, attended the study visits, and the study doctor feels you are eligible, you may be invited to enroll into Part 2, the optional open-label extension (follow-on) part of the FAiRE-LGS study. The main purpose of Part 2 is to learn about the investigational study drug s long-term safety and tolerability. The study will also continue to assess how well the investigational study drug works to decrease the frequency of seizures. Visit 15 (At the Clinic, Day 1) Ensure criteria to be included into Part 2 of the trial are met Vital signs Record demographics The following assessments will be repeated if clinically necessary or if not performed during Visit 12: Physical and neurological examinations Blood tests and urine tests Visit 16 (Telephone OR At the Clinic, Day 15) Telephone Visit: Clinic Visit: Visit 17 (At the Clinic, Day 30) Procedures Study medication collection, review and distribution All participants will receive the investigational study drug at doses that the doctor thinks are appropriate. Both the doctor and the participant will know what the dose is and the study doctor will have the ability to change it as appropriate. As in Part 1, the investigational study drug will be added to your regular epilepsy medications that your doctor has prescribed. You are not required to enter this part of the study. If you wish to enter this part of the study, you must sign the separate Informed Consent Form no later than Visit

4 Visit 18 (At the Clinic, Day 60) Visit 21 (At the Clinic, Day 270) Visit 19 (At the Clinic, Day 90) Visit 20 (At the Clinic, Day 180) Questionnaire: Visit 22 (At the Clinic, Day 365) Review study medication usage Physical and neurological examination (2) Quality of life (caregiver, [2]) Post-Dosing Follow-up Visit 23 (At the Clinic, Day 379) Collect weight Review study medication usage Post-Dosing Follow-up Visit 23 (At the Clinic, Day 379) related to cardiovascular health You may repeat these procedures at additional safety follow-ups as necessary. 6 7

5 Thank you! We appreciate your participation in the FAiRE-LGS research study. Study Doctor: Phone: Study Coordinator: Phone: Notes ZX _Study Visit Guide_v1.0_USA_12Mar2018_English