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

NCT02970162 | Completed Phase 3 Results

• 1 other identifier: LMS-003
• Study Type: interventional
• Target: 26
• Locations: 1 country
• Sites: 1

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.

Conditions

Lambert-Eaton Myasthenic Syndrome

Keywords

LEMS

Outcome Measures

Primary Outcomes (2)

• Quantitative Myasthenia Gravis (QMG) Score

  The QMG is a physician-rated test including 13 assessments such as facial strength, swallowing, grip strength, and duration of time that limbs can be maintained in outstretched positions. Each assessment is graded as 0 (none), 1 (mild), 2 (moderate), or 3 (severe), for a total range of 0-39. A higher total score indicates a worse outcome.

  change from baseline in QMG score at end of day 4

• Subject Global Impression (SGI) Score

  The SGI is a 7-point scale on which the patient rates their global impression of the effects of a study treatment (1=terrible to 7=delighted). The SGI was assessed by the patient or the patient's parent/guardian/caregiver if the patient was unable to complete the SGI. The SGI has demonstrated concordance with the physician's assessment of improvement.

  change from baseline in SGI score at end of day 4

Secondary Outcomes (1)

• Change in Clinician's Global Impression of Improvement (CGI-I) at Day 4 Compared to Baseline

  change from baseline in CGI-I score at end of day 4

Other Outcomes (1)

• Triple Timed Up and Go Walk Test (3TUG)

  change from baseline in 3TUG at end of day 4

Study Arms (2)

amifampridine phosphate

EXPERIMENTAL

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 (for amifampridine phosphate)

PLACEBO COMPARATOR

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

Drug: Placebo Oral Tablet

Interventions

Amifampridine Phosphate DRUG

amifampridine phosphate

Placebo Oral Tablet DRUG

placebo (for amifampridine phosphate)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female ≥18 years of age and currently receiving amifampridine phosphate for LEMS.
• Diagnosis of LEMS by antibody testing or electromyography (EMG).
• Completion of anti-cancer treatment at least 3 months (90 days) prior to Screening.
• 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.
• 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.
• Female patients of childbearing potential must practice an effective, reliable contraceptive regimen during the study.
• Able to perform all study procedures and assessments.
• Willing and able to travel to study site and attend all clinic study visits.
• Willing and able to provide written informed consent.

You may not qualify if:

• Clinically significant long corrected QT (QTc) interval on ECG in previous 12 months.
• Seizure disorder.
• Active brain metastases.
• Unable to ambulate.
• Pregnant or lactating females.
• 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.

Sponsors & Collaborators

• Catalyst Pharmaceuticals, Inc.: lead

Study Sites (1)

UCLA

Los Angeles, California, 90095, United States

Location

MeSH Terms

Conditions

Lambert-Eaton Myasthenic Syndrome

Interventions

Amifampridine

Condition Hierarchy (Ancestors)

Myasthenia Gravis; 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

Intervention Hierarchy (Ancestors)

4-Aminopyridine; Aminopyridines; Amines; Organic Chemicals; Pyridines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Results Point of Contact

• Title: Gary Ingenito
• Organization: Catalyst Pharmaceuticals, Inc.

gingenito@catalystpharma.com

305-420-3200

Study Officials

• Perry Shieh, MD, PhD

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR
• Expanded Access: Yes

Study Record Dates

• First Submitted: November 16, 2016
• First Posted: November 21, 2016
• Study Start: November 1, 2016
• Primary Completion: October 1, 2017
• Study Completion: October 1, 2017
• Last Updated: December 24, 2018
• Results First Posted: December 24, 2018

Record last verified: 2018-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)