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

NCT ID: NCT02970162

Last Updated: 2018-12-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 26 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-11-30
• Study Completion Date: 2017-10-31

Brief 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.

Detailed Description

This was a randomized (1:1), double-blind, placebo-controlled, parallel-group, withdrawal study designed to evaluate the efficacy and safety of amifampridine phosphate in patients diagnosed with LEMS. The study was planned to include approximately 28 male and female patients.

Prior to the study, patients were receiving unblinded treatment in the expanded access program (EAP-001). Patients had to be on a stable dose and frequency of amifampridine phosphate for at least 1 week prior to randomization into LMS-003. Screening and randomization (Day 0) may have been into a single visit.

Patients who met eligibility criteria were randomized 1:1 to amifampridine phosphate (at the patient's optimal dose) or placebo on Day 0.

Baseline assessments were obtained on Study Day 0, while the patient has been on open-label amifampridine phosphate and in relationship to the usual dosing schedule. Patients took blinded study medication on Day 1 through Day 3. On Day 4, a dose of blinded study medication was administered by the site study personnel. This was the same medication that the patient took on Day 1 through Day 3. The assessments listed below were performed following either the second, third, or fourth dose of medication taken on Day 4, and this should be the same dose after which Day 0 assessments were performed. For example, if the patient took their second dose of amifampridine in the clinic on Day 0 and had assessments started 40 minutes later, then on Day 4, that patient should be assessed after taking their second dose of investigational product (IP).

Beginning with the next dose after all Day 0 baseline assessments were completed, the patient received IP through Day 4, with a clinic visit on the last day (Day 4) for assessments.

The planned duration of participation for each patient was up to 12 days, including screening (up to 7 days), Day 0 assessments and randomization, and IP administration (Day 1 through Day 4).

Conditions

Lambert-Eaton Myasthenic Syndrome

Keywords

LEMS

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

amifampridine phosphate

amifampridine phosphate 10 mg (amifampridine equivalent) by mouth, 30 to 80 mg total daily dose, 3 to 4 times per day for 4 days

Group Type: EXPERIMENTAL

Amifampridine Phosphate

Intervention Type: DRUG

placebo (for amifampridine phosphate)

placebo by mouth 3 to 4 times per day for 4 days

Group Type: PLACEBO_COMPARATOR

Placebo Oral Tablet

Intervention Type: DRUG

Interventions

Amifampridine Phosphate

Intervention Type: DRUG

Placebo Oral Tablet

Intervention Type: DRUG

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Catalyst Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Perry Shieh, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University of California, Los Angeles

Locations

UCLA

Los Angeles, California, United States

Countries

United States

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

LMS-003

Identifier Type: -

Identifier Source: org_study_id