Introducing the FirST-4-LEMS Study
December 20, 2016
Your participation can help change the lives of people with Lambert-Eaton Myasthenic Syndrome (LEMS.) As someone living with LEMS and taking amifampridine phosphate, you have the opportunity to help EXPAND DRUG ACCESS FOR ALL PEOPLE WITH LEMS BY PARTICIPATING IN THE FirST-4-LEMS (Firdapse Strength Trial for LEMS) study.
- Your participation in the FirST-4-LEMS study could help others by participating in medical research. In fact, every prescription medicine you’ve taken has been investigated in studies that were only possible because of volunteer participants.
- You’ll have the reassurance of your support person accompanying you and healthcare personnel monitoring you in a safe, comfortable environment throughout the study.
A Summary of the FirST-4-LEMS Study
Medical condition under study: Lambert-Eaton Myasthenic Syndrome (LEMS); purpose of the study is to determine the degree of LEMS symptom control with amifampridine phosphate treatment
Eligibility criteria: Adult patients with a confirmed diagnosis of LEMS who are currently taking amifampridine phosphate
Participant benefits: All treatment and participation costs, (travel, hotel, meals, etc) are covered; no cost health examination, short term study (5 – 12 days); 24/7 access to expert medical care for the duration of the study
Time commitment: The FirST-4-LEMS study will be continuous for at least 5 and up to 12 days plus travel to the study site.
Study locations: Miami, FL and Los Angeles, CA
Why is the study being conducted?
The study is an additional clinical requirement that the FDA has asked Catalyst Pharmaceuticals to perform in order to further demonstrate that the medication you are taking is effective in treating LEMS symptoms. That’s why your participation is so important, because when the study is completed, the FDA may have the information needed to approve the medication so that it can be made available to all those living with LEMS in the United States.
What can I expect during the FirST-4-LEMS study ?
This is a short study (from 5 to 12 days, plus travel to/from the site), where you would stay at a hotel and have 3 to 4 required clinical evaluations at the medical center. Dates are flexible to work around your schedule and availability. The study procedures are limited, and all travel and hotel details will be arranged.
This is a “wash out” study, inwhichyou may be be off amifampridine phosphate for only 4 consecutive days. Half of the participants will be taken off their medication and given a placebo that looks like your medication. Your health is our primary concern, so you will be monitored closely throughout thestudy.
What happens in the clinical evaluation?
THE RESULTS OF THE FirST-4-LEMS STUDY ARE A NECESSARY PART OF TRYING TO OBTAIN FDA APPROVAL AND MAINTAIN ACCESS
TO EFFECTIVE THERAPY FOR THE LEMS COMMUNITY
The following will occur:
- Strength and walkingtests
- Questions about how youare feeling
• Medical history
• Optional screening for lungcancer
Will I need to sign an Informed Consent Document in order
Yes, this document is necessary for participation in all clinical trials. You’ll receive it from the study physician and have time to review it and ask questions. Your physician will also help answer any questions you may have.
Can I bring my spouse, a friend, or a support person?
Yes, you may bring a support person as part of the study, to help assist you. Their travel, hotel, and meal costs will also be provided.
Will I be compensated for my time participating in this study?
There may also be compensation available to offset financial hardships resulting from study participation, so please ask about possible availability.
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