Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congeni… (NCT02189720) | Clinical Trial Compass
APPROVED_FOR_MARKETINGNot Applicable
Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome
United States
Plain-language summary
Primary:
The primary objective of this study under the original protocol was to provide neuromuscular specialists and neurologists access to amifampridine phosphate therapy for their patients with LEMS, CMS or downbeat nystagmus until the product became commercially available.
Secondary:
The secondary objective of this study under the original protocol was to provide additional long-term safety data on amifampridine phosphate in patients.
Primary The primary objective of this study after its fifth amendment was to provide access to amifampridine phosphate therapy to pediatric patients with LEMS, and pediatric and adult patients with CMS until the product became commercially available for these indications or development of the product for the indication was terminated.
Secondary:
The secondary objective of this study after its fifth amendment was to assess the long-term safety of amifampridine phosphate in pediatric patients with LEMS, and pediatric and adult patients with CMS.
Who can participate
Age range
2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria - Amendment 5 v6 - most recent version
* Male or female:
* 2 years of age
* Confirmed physician diagnosis of LEMS in pediatric patients or CMS in either pediatric or adult patients.
* Completion of anti-cancer treatment at least 3 months (90 days) before treatment.
* Negative urine pregnancy test for females of childbearing potential at Screening.
* If sexually active and of childbearing potential, willing to use 2 acceptable methods of contraception from screening visit until 3 months after the last dose of investigational product. No adequate clinical data on exposed pregnancies are available for amifampridine. No nonclinical safety data are available regarding the effects of amifampridine on reproductive function. Amifampridine phosphate should not be used during pregnancy. It is unknown whether amifampridine is excreted in human breast milk. The excretion of amifampridine in milk has not been studied in animals. Amifampridine phosphate should not be used during breastfeeding.
* Any subject currently participating in study CMS 001 is immediately eligible for enrollment into study EAP-001, as long as inclusion/exclusion criteria are still met.
* Willing and able to provide written informed consent after the nature of the study has been explained and before the start of any research-related procedures.
Exclusion Criteria- Amendment 5 v6
* History of epilepsy and on medication/treatment for the same.
* CMS subtypes including slow-channel syndrome, LRP…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since amifampridine phosphate has already been approved for marketing for Lambert-Eaton Myasthenic Syndrome, can you help me understand whether that approval covers my specific diagnosis — including whether it extends to Congenital Myasthenic Syndrome or acquired nystagmus — and what that means for my treatment options?
2Given that this was an expanded access study rather than a standard Phase 1, 2, or 3 trial, what do we actually know about the safety and effectiveness of amifampridine phosphate specifically for my condition, and are there any gaps in that evidence I should be aware of?
3Now that this drug is commercially approved, would prescribing it as a standard treatment make more sense for me than participating in any ongoing research program, and what would that look like in practice?
4Are there patients with my specific type of Congenital Myasthenic Syndrome or nystagmus who tend to respond better or worse to amifampridine phosphate, and how would you assess whether I'm likely to be one of them?
5What are the most important side effects or monitoring requirements associated with amifampridine phosphate that I should plan for if we decide this is the right path for me?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.