Trial · NCT03304054
Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG
NCT03304054, Phase 3, Completed, Catalyst Pharmaceuticals, Inc.; results first posted 2021-08-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG. Trials, record NCT03304054. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03304054
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study to Evaluate Amifampridine Phosphate in Patients With MuSK-MG
- NCT number
- NCT03304054
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 93 participants, actual
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.
- Conditions
- Myasthenia Gravis, Generalized
- Interventions
- Amifampridine Phosphate, Placebo Oral Tablet
- First posted
- 2017-10-06
- Last updated
- 2024-03-12
- Results first posted
- 2021-08-13
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early). | 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 |
| Secondary | Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time at which a patient discontinued treatment early). | 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. |
Source ClinicalTrials.gov
Outcomes2
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time point at which a patient discontinued treatment early). | 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. | Score on a Scale | MEAN |
| SECONDARY | Quantitative Myasthenia Gravis (QMG) Total Score Summary Statistics by Time Point and MG Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Last day (Day 0) of the Run-in period and at the post-treatment visit (i.e., day 10 or the time at which a patient discontinued treatment early). | 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. | Score on a Scale | MEAN |
Source ClinicalTrials.gov
Results by group24
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Amifampridine Phosphate - MuSK | Baseline (Day 0) MG-ADL Total Raw Score | 4.96 | Standard Deviation 2.915 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Placebo - MuSK | Baseline (Day 0) MG-ADL Total Raw Score | 3.86 | Standard Deviation 2.103 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Amifampridine Phosphate - AChR | Baseline (Day 0) MG-ADL Total Raw Score | 6.14 | Standard Deviation 3.388 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Placebo - AChR | Baseline (Day 0) MG-ADL Total Raw Score | 7.00 | Standard Deviation 4.175 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Amifampridine Phosphate - MuSK | Post-Baseline(Day 10 or time at which a patient discontinued treatment) MG-ADL Total Raw Score | 6.00 | Standard Deviation 3.658 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Placebo - MuSK | Post-Baseline(Day 10 or time at which a patient discontinued treatment) MG-ADL Total Raw Score | 6.11 | Standard Deviation 3.19 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Amifampridine Phosphate - AChR | Post-Baseline(Day 10 or time at which a patient discontinued treatment) MG-ADL Total Raw Score | 4.71 | Standard Deviation 3.147 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Placebo - AChR | Post-Baseline(Day 10 or time at which a patient discontinued treatment) MG-ADL Total Raw Score | 10.38 | Standard Deviation 3.583 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Amifampridine Phosphate - MuSK | CFB MG-ADL Total Score | 1.04 | Standard Deviation 2.980 | Score on a Scale |
| Myasthenia Gravis-Activities of Daily Living (MG-ADL) Summary by Time Point and Myasthenia Gravis Type: Wilcoxon-Mann-Whitney Rank Sum Test Results | Placebo - MuSK | CFB MG-ADL Total Score | 2.25 | Standard Deviation 3.658 | Score on a Scale |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Amifampridine Phosphate - MuSK | 27 | |
| Overall Study | STARTED | Amifampridine Phosphate - AChR-MG | 7 | |
| Overall Study | STARTED | Placebo - MuSk | 28 | |
| Overall Study | STARTED | Placebo - AChR-MG | 8 | |
| Overall Study | STARTED | Amifampridine Phosphate Only - MuSK | 17 | |
| Overall Study | STARTED | Amifampridine Phosphate Only- AChR-MG | 6 | |
| Overall Study | COMPLETED | Amifampridine Phosphate - MuSK | 27 | |
| Overall Study | COMPLETED | Amifampridine Phosphate - AChR-MG | 7 | |
| Overall Study | COMPLETED | Placebo - MuSk | 27 | |
| Overall Study | COMPLETED | Placebo - AChR-MG | 8 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events135
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal discomfort | Gastrointestinal disorders | Amifampridine Phosphate | 5 | 86 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Placebo | 0 | 36 | |
| other | Abdominal discomfort | Gastrointestinal disorders | Overall | 5 | 86 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Amifampridine Phosphate | 7 | 86 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 0 | 36 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Overall | 7 | 86 | |
| other | Diarrhoea | Gastrointestinal disorders | Amifampridine Phosphate | 12 | 86 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 0 | 36 | |
| other | Diarrhoea | Gastrointestinal disorders | Overall | 12 | 86 | |
| other | Dyspepsia | Gastrointestinal disorders | Amifampridine Phosphate | 7 | 86 |
1 to 10 of 135Source ClinicalTrials.gov
See all 135 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Amifampridine Phosphate | 0 | 86 |
| Total, all-cause mortality | Placebo | 0 | 36 |
| Total, all-cause mortality | Overall | 0 | 86 |
| Total, other adverse events | Amifampridine Phosphate | 80 | 86 |
| Total, other adverse events | Placebo | 7 | 36 |
| Total, other adverse events | Overall | 80 | 86 |
| Total, serious adverse events | Amifampridine Phosphate | 2 | 86 |
| Total, serious adverse events | Placebo | 0 | 36 |
| Total, serious adverse events | Overall | 2 | 86 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT03304054
GET https://conductscience.org/api/trials/v1/NCT03304054/results?page=2