A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)

Descriptive Information
Brief Title † A Phase 3 Study of Amifampridine Phosphate in Patients With Lambert Eaton Myasthenic Syndrome (LEMS)
Official Title † A Phase 3, Double-blind, Placebo-controlled, Randomized Discontinuation Study Followed by Open-label Extension Evaluating Efficacy and Safety of Amifampridine Phosphate in Patients With Lambert-Eaton Myasthenic Syndrome (LEMS)
Brief Summary A Phase 3 study to evaluate the efficacy and safety of Amifampridine Phosphate in patients with Lambert-Eaton Myasthenic Syndrome (LEMS).
Detailed Description This multicenter, double-blind, placebo-controlled, randomized (1:1) discontinuation study is a 4 part study designed to evaluate the efficacy and safety of multiple dose administration of amifampridine phosphate in patients with LEMS.
Study Phase Phase 3
Study Type † Interventional
Study Design † Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment
Primary Outcome Measure † Change from Baseline Quantitative Myasthenia Gravis (QMG)at 14 days
Secondary Outcome Measure † Change from Baseline Timed 25 Foot Walking Test (T25FW)at 14 days
Condition † Lambert Eaton Myasthenic Syndrome
Intervention † DrugAmifampridine Phosphate
Study Arms / Comparison Groups Placebo Matching placebo tablets administered 3-4 times a day over 2 weeks. Amifampridine Phosphate Matching amifampridine phosphate tablets, 10 mg, administered 3-4 times a day for 2 weeks.
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 † 36
Start Date † June 2011
Completion Date August 2016
Primary Completion Date September 2014
Eligibility Criteria † Inclusion Criteria: Individuals eligible to participate in this study must meet all of the following inclusion criteria: - ≥18 years of age - Confirmed diagnosis of LEMS - Normal respiratory function - Normal swallowing function - If receiving peripherally acting cholinesterase inhibitors a stable dose is required for at least 7 days prior to Screening. - If receiving oral immunosuppressants a stable dose is required for at least 90 days prior to Screening. - Negative pregnancy test for females of childbearing potential - If sexually active, willing to use 2 acceptable methods of contraception - Willing to perform all study procedures as physically possible. - Willing and able to provide written informed consent after the nature of the study has been explained and prior to the start of any research-related procedures. Exclusion Criteria: Individuals who meet any of the following exclusion criteria are not eligible to participate in the study: - History of epilepsy or seizure. - Known active brain metastasis. - Use of Fampridine (4-aminopyridine), and any form of 3,4-diaminopyridine other than the IP provided, such as amifampridine base or Firdapse, during the study. - Use of medications known to lower the epileptic threshold within 7 days or 5 half-lives. - Use of medications which inhibit neuromuscular junction function within 7 days or 5 half-lives. - Use of IVIG, plasmapheresis (plasma exchange), or immunoadsorption within 90 days - Use of guanidine hydrochloride within 7 days - Use of rituximab within 12 months - History of drug allergy to any pyridine-containing substances or any amifampridine phosphate excipient(s). - Use of any other investigational productwithin 30 days - Treatment with a concomitant medication that prolongs the QT/QTc interval within 7 days or 5 half-lives. - Treatment with sultopride (4-amino-N-[(1-ethylpyrrolidin-2-yl)methyl]-5-ethylsulfonyl-2-methoxybenzamide) within 7 days. - An abnormal electrocardiogram (ECG). - Documented history of arrhythmias. - History of additional risk factors for torsade de pointes. - Breastfeeding or pregnant or planning to become pregnant (self or partner) at any time during the study. - Likely or expected to require treatment for cancer within 3 months (90 days) after entering. - History of severe renal impairment or evidence of severe renal impairment - Any condition that places the patient at high risk of poor treatment compliance or of not completing the study. - History of uncontrolled asthma.
Gender Both
Ages 18 Years - N/A
Accepts Healthy Volunteers No
Contacts ††
Location Countries † United States
Administrative Information
NCT ID † NCT01377922
Organization ID LMS-002
Secondary IDs ††
Responsible Party Sponsor
Study Sponsor † Catalyst Pharmaceutical Partners, Inc
Collaborators ††
Investigators † Study Director: Charles W Gorodetzky, MD, PhD, Chief Medical Officer
Information Provided By
Verification Date August 2014
First Received Date † June 17, 2011
Last Updated Date August 4, 2014
† Required WHO trial registration data element.
†† WHO trial registration data element that is required only if it exists.
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