Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndrome (CMS), or Downbeat Nystagmus Patients (EAP-001)
The primary objective of the study is:
• To provide patients with LEMS/CMS/downbeat nystagmus access to amifampridine phosphate therapy until the product becomes commercially available.
The secondary objective of the study is:
• To assess the long-term safety of amifampridine phosphate in patients with LEMS/CMS/downbeat nystagmus
|Lambert-Eaton Myasthenic Syndrome Congenital Myasthenic Syndrome Downbeat Nystagmus||Drug: Amifampridine Phosphate|
|Study Type:||Expanded Access What is Expanded Access?|
|Official Title:||An Open-Label, Expanded Access Protocol for Amifampridine Phosphate Treatment in Patients With Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndrome (CMS) and Downbeat Nystagmus|
Drug: Amifampridine Phosphate
- 3,4-Diaminopyridine Phosphate
- 3,4 DAP
Dosage form: tablets containing the equivalent of 10 mg amifampridine base per tablet.
Amifampridine Phosphate is taken with food and each patient is given an individualized dose based on Investigator assessment of optimal neuromuscular benefit. Doses range from 30 mg to 80 mg, divided into doses taken 3 to 4 times per day, with a maximum single dose of 20 mg.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02189720
|Contact: Jonathan Rubine, MD||305-420-3200 ext firstname.lastname@example.org|
|United States, California|
|Los Angeles, California, United States, 90095|
|Contact: Clara Sam 310-825-3264 email@example.com|
|Contact: Gary Ingenito, MD, PhD 305-420-3200 ext 123 firstname.lastname@example.org|
|Principal Investigator:||Perry Shieh, MD, PhD||University of California, Los Angeles|