Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS)

• Status: Completed
• Trial end date: 2017-10-01
• Target enrollment: 0
• Participant gender: All

Summary

This study evaluates the effect of withdrawing amifampridine phosphate treatment from patients with LEMS. One half of the patients will continue to receive amifampridine phosphate and the other half will receive placebo, during this double-blind study.

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

Details

• Lead Sponsor: Catalyst Pharmaceuticals, Inc.
• Treatments: Amifampridine

Criteria

Inclusion Criteria:

1. Male or female ≥18 years of age and currently receiving amifampridine phosphate for LEMS.

2. Diagnosis of LEMS by antibody testing or electromyography (EMG).

3. Completion of anti-cancer treatment at least 3 months (90 days) prior to Screening.

4. If receiving peripherally acting cholinesterase inhibitors (e.g. pyridostigmine), a stable dose of cholinesterase inhibitors is required for at least 7 days prior to randomization and throughout the study.

5. If receiving permitted oral immunosuppressants (prednisone or other corticosteroid), a stable dose is required for at least 30 days prior to randomization and throughout the study.

6. Female patients of childbearing potential must practice an effective, reliable contraceptive regimen during the study.

7. Able to perform all study procedures and assessments.

8. Willing and able to travel to study site and attend all clinic study visits.

9. Willing and able to provide written informed consent.

Exclusion Criteria:

1. Clinically significant long corrected QT (QTc) interval on ECG in previous 12 months.

2. Seizure disorder.

3. Active brain metastases.

4. Unable to ambulate.

5. Pregnant or lactating females.

6. Any other condition which, in the opinion of the Investigator, might interfere with the patient's participation in the study or confound the assessment of the patient.