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

NCT ID: NCT01377922

Last Updated: 2018-01-04

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 38 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-06-30
• Study Completion Date: 2016-07-31

Brief Summary

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

Detailed Description

This multicenter, double-blind, placebo-controlled, randomized (1:1) discontinuation study is a 4-part study designed to evaluate the efficacy and safety of multiple dose administration of amifampridine phosphate in patients with LEMS. Data from parts 2 and 3 (the double-blind parts of the study) are presented in this record.

Conditions

Lambert Eaton Myasthenic Syndrome

Study Design

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

Study Groups

Placebo

Matching placebo tablets administered 3-4 times a day (to the individual patient's tablet count of active at baseline) over 2 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Matching placebo tablets administered 3-4 times a day (to the individual patient's tablet count of active at baseline) over 2 weeks.

Amifampridine Phosphate

Amifampridine, 30-80 mg given 3-4 times per day with a maximum single dose of 20 mg (2 x 10 mg tablets), for 2 weeks.

Group Type: EXPERIMENTAL

Amifampridine Phosphate

Intervention Type: DRUG

Amifampridine, 30-80 mg given 3-4 times per day with a maximum single dose of 20 mg (2 x 10 mg tablets), for 2 weeks.

Interventions

Amifampridine Phosphate

Amifampridine, 30-80 mg given 3-4 times per day with a maximum single dose of 20 mg (2 x 10 mg tablets), for 2 weeks.

Intervention Type: DRUG

Placebo

Matching placebo tablets administered 3-4 times a day (to the individual patient's tablet count of active at baseline) over 2 weeks.

Intervention Type: DRUG

Other Intervention Names

3,4-diaminopyridine phosphate; 3,4-DAP phosphate

Eligibility Criteria

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

• 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.

• 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

Charles W Gorodetzky, MD, PhD

Role: STUDY_DIRECTOR

Chief Medical Officer

Locations

Birmingham, Alabama, United States

Scottsdale, Arizona, United States

Los Angeles, California, United States

Palo Alto, California, United States

Kansas City, Kansas, United States

New York, New York, United States

Lyon, , France

Munich, Bavaria, Germany

Berlin, , Germany

Pécs, , Hungary

Warsaw, , Poland

Moscow, , Russia

Belgrade, , Serbia

Madrid, , Spain

Countries

United States; France; Germany; Hungary; Poland; Russia; Serbia; Spain

Other Identifiers

LMS-002

Identifier Type: -

Identifier Source: org_study_id