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

Catalyst Pharmaceuticals

Status and phase

Completed

Phase 3

Conditions

Lambert Eaton Myasthenic Syndrome

Treatments

Drug: Placebo

Drug: Amifampridine Phosphate

Study type

Interventional

Funder types

Industry

Identifiers

NCT01377922

LMS-002

Details and patient eligibility

About

A Phase 3 study to evaluate the efficacy and safety of Amifampridine Phosphate in patients with Lambert-Eaton Myasthenic Syndrome (LEMS).

Full 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. Data from parts 2 and 3 (the double-blind parts of the study) are presented in this record.

Enrollment

38 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion 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.