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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT02970162
Recruitment Status : Completed
First Posted : November 21, 2016
Last Update Posted : November 27, 2017
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.

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.

Condition or disease Intervention/treatment Phase
Lambert-Eaton Myasthenic Syndrome Drug: Amifampridine Phosphate Drug: Placebo Oral Tablet Phase 3

Expanded Access : An investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 26 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Double-Blind, Placebo-controlled, Randomized, Parallel-Group Study to Evaluate the Efficacy and Safety of Amifampridine Phosphate in Patients With Lambert-Eaton Myasthenic Syndrome
Study Start Date : November 2016
Actual Primary Completion Date : October 2017
Actual Study Completion Date : October 2017

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: 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
Drug: Amifampridine Phosphate
Placebo Comparator: placebo (for amifampridine phosphate)
placebo by mouth 3 to 4 times per day for 4 days
Drug: Placebo Oral Tablet

Primary Outcome Measures :
  1. Quantitative Myasthenia Gravis (QMG) score [ Time Frame: change from baseline in QMG score at end of day 4 ]
  2. Subject Global Impression (SGI) score [ Time Frame: change from baseline in SGI score at end of day 4 ]

Secondary Outcome Measures :
  1. Clinician's Global Impression of Improvement (CGI-I) [ Time Frame: change from baseline in CGI-I score at end of day 4 ]

Other Outcome Measures:
  1. Triple Timed Up and Go walk test (3TUG) [ Time Frame: change from baseline in time to complete test on day 4 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02970162

United States, California
Los Angeles, California, United States, 90095
Sponsors and Collaborators
Catalyst Pharmaceuticals, Inc.
Principal Investigator: Perry Shieh, MD, PhD University of California, Los Angeles

Responsible Party: Catalyst Pharmaceuticals, Inc. Identifier: NCT02970162     History of Changes
Other Study ID Numbers: LMS-003
First Posted: November 21, 2016    Key Record Dates
Last Update Posted: November 27, 2017
Last Verified: November 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Catalyst Pharmaceuticals, Inc.:

Additional relevant MeSH terms:
Lambert-Eaton Myasthenic Syndrome
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action