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

This study is currently recruiting participants.
Verified October 2017 by Catalyst Pharmaceuticals, Inc.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02970162
First Posted: November 21, 2016
Last Update Posted: October 4, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Catalyst Pharmaceuticals, Inc.
  Purpose
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 Intervention Phase
Lambert-Eaton Myasthenic Syndrome Drug: Amifampridine Phosphate Drug: Placebo Oral Tablet Phase 3

Access to an investigational treatment associated with this study is available outside the clinical trial.   More info ...

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Catalyst Pharmaceuticals, Inc.:

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

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

Estimated Enrollment: 28
Study Start Date: November 2016
Estimated Study Completion Date: October 2017
Estimated Primary Completion Date: October 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
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 ClinicalTrials.gov identifier (NCT number): NCT02970162


Contacts
Contact: Adriana Manari 305-420-3217 amanari@catalystpharma.com
Contact: Jonathan Rubine, MD 305-420-3233 jrubine@catalystpharma.com

Locations
United States, California
UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Angela Ho    310-825-3264      
Sponsors and Collaborators
Catalyst Pharmaceuticals, Inc.
Investigators
Principal Investigator: Perry Shieh, MD, PhD University of California, Los Angeles
  More Information

Responsible Party: Catalyst Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02970162     History of Changes
Other Study ID Numbers: LMS-003
First Submitted: November 16, 2016
First Posted: November 21, 2016
Last Update Posted: October 4, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Catalyst Pharmaceuticals, Inc.:
LEMS

Additional relevant MeSH terms:
Syndrome
Lambert-Eaton Myasthenic Syndrome
Disease
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
3,4-diaminopyridine
4-Aminopyridine
Potassium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action