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Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndrome (CMS), or Downbeat Nystagmus Patients

Descriptive Information
Brief Title † Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenic Syndrome (CMS), or Downbeat Nystagmus Patients
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
Brief Summary 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
Detailed Description
Study Phase N/A
Study Type † Expanded Access
Study Design †
Primary Outcome Measure †
Secondary Outcome Measure †
Condition † Lambert-Eaton Myasthenic Syndrome Congenital Myasthenic Syndrome Downbeat Nystagmus
Intervention † DrugAmifampridine Phosphate
Study Arms / Comparison Groups
Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information
Recruitment Status † Drug
Estimated Enrollment †
Start Date †
Completion Date
Primary Completion Date
Eligibility Criteria † Inclusion Criteria: - Male or female: - 2 years of age at 5 pediatric CMS study sites - 10 years of age at other study sites. - Confirmed physician diagnosis of LEMS, CMS or downbeat nystagmus. - 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 studies LMS-002, LMS-002EXT, or 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: - History of epilepsy. - CMS subtypes including slow-channel syndrome, LRP4 deficiency, and acetylcholinesterase deficiency. - Known active brain metastasis. Patients with treated brain metastasis (radiotherapy and/or surgery) who have completed treatment for their brain metastasis >90 days before Screening, are neurologically stable (neurological symptoms grade 1.5 × upper limit of normal (ULN). - Any condition that, in the view of the Principal Investigator, places the patient at high risk of poor treatment compliance or of not completing the study.
Gender All
Ages 10 Years - N/A
Accepts Healthy Volunteers
Contacts †† Jonathan Rubine, MD, 305-420-3200, jrubine@catalystpharma.com
Location Countries † United States
Administrative Information
NCT ID † NCT02189720
Organization ID EAP-001
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Catalyst Pharmaceuticals, Inc.
Collaborators ††
Investigators † Principal Investigator: Perry Shieh, MD, PhD, University of California, Los Angeles
Information Provided By
Verification Date January 2017
First Received Date † July 5, 2014
Last Updated Date January 31, 2017
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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