Neoplasms

Nervous System Diseases

Immune System Diseases

All Conditions

Symptoms and General Pathology

Rare Diseases

Myasthenia Gravis

Syndrome

Paraneoplastic Syndromes

Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

Autoimmune Diseases

Autoimmune Diseases of the Nervous System

Neurodegenerative Diseases

Neuromuscular Junction Diseases

Neuromuscular Diseases

All Drugs and Chemicals

Channel Blockers

Antibodies

Immunoglobulins

Amifampridine

Potassium Channel Blockers

tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

Amifampridine Phosphate

tablets matching amifampridine phosphate, 3 to 4 times a day

Placebo Oral Tablet

Renato Mantegazza, MD

Efficacy and safety of amifampridine phosphate in improving the activities of daily living for patients with antibody positive MuSK myasthenia gravis.

Randomized, double-blind, placebo-controlled, parallel group study is designed to evaluate the safety, tolerability and efficacy of amifampridine phosphate in patients with MuSK-MG. In addition, a sample of AChR-MG patients will be assess for efficacy and safety of amifampridine phosphate. Planned duration of participation for each patient is at least 38 days, excluding the screening period. Eligible patients will be titrated to an efficacious dose of amifampridine phosphate and those who demonstrate improvement will be randomized to either placebo or amifampridine, in a double-blind fashion, for 10 days.

Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

A Randomized, Placebo-control, Parallel Group Study to Evaluate the Effect of Amifampridine Phosphate in Patients With MuSK Antibody Positive Myasthenia Gravis, and a Sample of AChR Antibody Positive Myasthenia Gravis Patients

Myasthenia Gravis-Activities of Daily Living (MG-ADL) is a self-report scale to assess the patient's MG symptoms and functional performance of activities of daily living. The eight items are scored on a scale of 0-3 with 3 representing the most severe symptoms or impaired performance and 0 representing no symptoms or impaired performance. Each item was assessed by the patient at the last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total MG-ADL score was calculated as the sum of each item score, with a maximum score of 24 (most severe symptoms/impairment) and minimum score of 0 (least severe symptoms/impairment). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnos

Quantitative Myasthenia Gravis (QMG) assesses the patient's general body strength and fatigability. Each test item is scored on a scale of 0-3 with 3 representing the most severe symptom results and 0 representing no symptom results. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period and at the post-treatment visit. The post-treatment result will be the result obtained on Day 10. If the patient discontinued treatment early, the post-treatment result may be obtained at an earlier time point. The total QMG score was calculated as the sum of each item score, with a maximum score of 39 (most severe symptoms) and minimum score of 0 (least severe symptoms). The change from baseline (CFB) at Day 10 was assessed. A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.

Catalyst Pharmaceuticals, Inc.

Serious classification based on the FDA regulatory definition of a serious AE.

Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day.

The AEs reported within this treatment arm are those experienced by all participants who received Amifampridine Phosphate at the time of onset of the AE. Because this study has a run-in period where all subjects received Amifampridine Phosphate, the number of at-risk subjects in this treatment arm is identical to the overall number of at-risk participants. That is, the at-risk count includes those randomized to the Amifampridine Phosphate-Placebo sequence, Placebo - Amifampridine Phosphate sequence and those that received Amifampridine Phosphate during the open-label Run-in period but were not randomized for the crossover portion of the study.

Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day

The AEs reported within this treatment arm are those experienced by participants who received Placebo at the time of onset of the AE. This includes those randomized to the Placebo - Amifampridine Phosphate sequence.

Placebo

Includes all participants who received study drug, including Amifampridine Phosphate during the open-label Run-in period or double-blind treatment period or Placebo during the double-blind treatment period.

Overall

Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day

Patients receiving Amifampridine during the open-label Run-in period but were not randomized for the crossover portion of the study.

Amifampridine Phosphate Only

Total

Age, Continuous

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

Weight

Height

The analysis of baseline characteristics included all patients in the Safety population, who are those patients who enrolled and received at least one dose of amifampridine. Patients who began the run-in period belong to the Safety population whether they were randomized to treatment or not.

Gary Ingenito

A Wilcoxon-Mann-Whitney Rank Sum Test of equality of change from baseline distributions between subjects diagnosed with MuSK-MG treated with amifampridine and placebo was conducted.

Baseline (Day 0) MG-ADL Total Raw Score

Post-Baseline(Day 10 or time at which a patient discontinued treatment) MG-ADL Total Raw Score

CFB MG-ADL Total Score

Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

Patients randomized to receive Amifampridine and diagnosed with MuSK-MG.

Amifampridine Phosphate - MuSK

Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day

Patients randomized to receive Placebo and diagnosed with MuSK-MG.

Placebo - MuSK

Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

Patients randomized to receive Amifampridine and diagnosed with AChR-MG.

Amifampridine Phosphate - AChR

Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day

Patients randomized to receive Placebo and diagnosed with AChR-MG.

Placebo - AChR

The analysis of primary outcome data was based on the Full Analysis Set population, which included all randomized patients who received at least one dose of study medication (amifampridine or placebo post randomization) and had at least one post-treatment efficacy assessment. Patients who discontinued with no post-randomization data (no Day 0 and no Day 10 data) were excluded from all efficacy analyses but were included in the safety analyses.

Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results

Baseline (Day 0) QMG Total Raw Score

Post-Baseline (day 10 or time at which a patient discontinued treatment early) QMG Total Raw Score

CFB QMG Total Score

The analysis of secondary outcome data was based on the Full Analysis Set population, which included all randomized patients who received at least one dose of study medication (amifampridine or placebo post randomization) and had at least one post-treatment efficacy assessment. Patients who discontinued with no post-randomization data (no Day 0 and no Day 10 data) were excluded from all efficacy analyses but were included in the safety analyses.

Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results

Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

MuSK - patients diagnosed with MuSK-MG

Amifampridine Phosphate: tablets equivalent to 10mg amifampridine, titrated to an efficacious and tolerable dose, 3 to 4 times a day

AChR-MG- patients diagnosed with AChR-MG

Amifampridine Phosphate - AChR-MG

Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day

MuSK - patients diagnosed with MuSK-MG

Placebo - MuSk

Placebo Oral Tablet: tablets matching amifampridine phosphate, 3 to 4 times a day

AChR-MG- patients diagnosed with AChR-MG

Placebo - AChR-MG

Patients receiving amifampridine during the open-label run-in period but were not randomized for the crossover portion of the study and diagnosed with MuSK-MG.

Amifampridine Phosphate Only - MuSK

Patients receiving amifampridine during the open-label run-in period but were not randomized for the crossover portion of the study and diagnosed with AChR-MG.

Spots Global Cancer Trial Database for Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG

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