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

• Status: Completed
• Trial end date: 2016-07-01
• Target enrollment: 0
• Participant gender: All

Summary

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Catalyst Pharmaceuticals, Inc.
• Treatments: 3,4-diaminopyridine; Amifampridine

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.