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

Trial ID or NCT#

NCT01377922

Status

not recruiting iconNOT RECRUITING

Purpose

A Phase 3 study to evaluate the efficacy and safety 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)

Eligibility Criteria

Ages Eligible for Study: Older than 18 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. 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:
  1. 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.
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Investigator(s)

Sarada Sakamuri, MD
Sarada Sakamuri, MD
Neuromuscular neurologist, General neurologist, Neurophysiologist, Neurologist
Clinical Associate Professor, Neurology & Neurological Sciences Clinical Associate Professor (By courtesy), Neurosurgery

View on ClinicalTrials.gov