Phase 3
N=26
Phase 3 Study to Evaluate Efficacy of Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS)
Lambert-Eaton Myasthenic Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT02970162 ↗Enrolled (actual)
26
Serious AEs
0.0%
Results posted
Dec 2018
Primary outcome: Primary: Quantitative Myasthenia Gravis (QMG) Score — 7.8; 8.5; 7.9; 15.0 scores on a scale — p=0.0004
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Amifampridine Phosphate (Drug); Placebo Oral Tablet (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Catalyst Pharmaceuticals, Inc.
- Primary completion
- Oct 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Quantitative Myasthenia Gravis (QMG) Score |
7.8; 8.5; 7.9; 15.0; 0.1; 6.5 | 0.0004 sig |
| PRIMARY Subject Global Impression (SGI) Score |
6.1; 5.8; 5.3; 2.4; -0.8; -3.5 | 0.0003 sig |
| SECONDARY Change in Clinician's Global Impression of Improvement (CGI-I) at Day 4 Compared to Baseline |
3.8; 5.5 | 0.0020 sig |
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.
Eligibility Criteria
Inclusion Criteria
- 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.
Exclusion Criteria
- 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.
Data sourced from ClinicalTrials.gov (NCT02970162). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.