Trial · NCT02562066
Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
NCT02562066, Phase 3, Completed, Catalyst Pharmaceuticals, Inc.; results first posted 2021-04-02
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes. Trials, record NCT02562066. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02562066
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
- NCT number
- NCT02562066
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 20 participants, actual
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.
- Conditions
- Myasthenic Syndromes, Congenital
- Interventions
- amifampridine phosphate, Placebo
- First posted
- 2015-09-29
- Last updated
- 2021-04-02
- Results first posted
- 2021-04-02
Source ClinicalTrials.gov
Registered outcomes3
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis | Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29 | Subject Global Impression (SGI) rates the subject's impression of the effects of the study medication during the preceding week with max score =7 (most satisfied) and min score =1 (least satisfied). It will be completed at D0 (baseline for Study Period 1), D8, D21 (baseline for Study Period 2), and D29. Change from baseline (CFB) will be assessed for Study Period 1 (difference in SGI score from D0 to D8), Study Period 2 (difference in SGI score from D21 to D29) and the change in CFB from Study Period 1 to Study Period 2 (difference between CFB during Study Period 1 and CFB during Study Period 2). A Mann-Whitney-Wilcoxon test for equality of the CFB results in the two treatments in Period 1 and for the equality of the CFB results in the two treatment sequences will be conducted. A mixed effects liner model will be fit with the SGI raw scores as a response and study arm, treatment, period, age group and mutation type as fixed effect terms and patient as a random effect. |
| Secondary | Motor Function Measure 32 (MFM-32) Score Summary: Mann-Whitney Main Effect Test Results, MFM-32 Score Mixed Model Analysis | Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29 | Motor Function Measure 32 (MFM-32) evaluates the severity and progression of motor function in patients 7 years of age or older with max total score = 96 (best score) and min total score =0 (worst score). It will be completed at D0 (baseline for Study Period 1), D8, D21 (baseline for Study Period 2) and D29. CFB will be assessed for Study Period 1 (difference in MFM-32 from D0 to D8), Study Period 2 (difference in MFM-32 from D21 to D28) and the change in CFB from Study Period 1 to Study Period 2 (difference between CFB during Study Period 1 and CFB during Study Period 2). A Mann-Whitney-Wilcoxon test for equality of CFB results in the two treatments in Period 1 and for the equality of the CFB results in the two treatment sequences will be conducted. A mixed effects linear model will be fit to the overall MFM-32 score as the response variable and fixed effect terms for treatment, period, age group, genetic mutation type, and sequence\*period and a random effect for patient. |
| Secondary | Motor Function Measure 20 (MFM-20) Score Mixed Model Analysis, MFM-20 Score Mixed Model Analysis | Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29 | Motor Function Measure 20 (MFM-20) evaluates the severity and progression of motor function in patients 2 to 6 years of age with max total score = 60 (best score) and min total score =0 (worst score). It will be completed at D0 (baseline for Study Period 1), D8, D21 (baseline for Study Period 2) and D29. CFB will be assessed for Study Period 1 (difference in MFM-20 from D0 to D8), Study Period 2 (difference in MFM-20 from D21 to D28) and the change in CFB from Study Period 1 to Study Period 2 (difference between CFB during Study Period 1 and CFB during Study Period 2). A Mann-Whitney-Wilcoxon test for equality of CFB results in the two treatments in Period 1 and for the equality of the CFB results in the two treatment sequences will be conducted. A mixed effects linear model will be fit to the overall MFM-32 score as the response variable and fixed effect terms for treatment, period, age group, genetic mutation type, and sequence\*period and a random effect for patient. |
Source ClinicalTrials.gov
Outcomes3
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis | Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29 | The Full Analysis Set consists of all randomized patients who receive at least one dose of study medication and have at least one post-treatment efficacy assessment. | Scores on a scale | MEAN |
| SECONDARY | Motor Function Measure 32 (MFM-32) Score Summary: Mann-Whitney Main Effect Test Results, MFM-32 Score Mixed Model Analysis | Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29 | The Full Analysis Set consists of all randomized patients who receive at least one dose of study medication and have at least one post-treatment efficacy assessment. | Score on a Scale | MEAN |
| SECONDARY | Motor Function Measure 20 (MFM-20) Score Mixed Model Analysis, MFM-20 Score Mixed Model Analysis | Study Period 1: Baseline (Day 0), Day 8; Study Period 2: Baseline (Day 21), Day 29 | The Full Analysis Set consists of all randomized patients who receive at least one dose of study medication and have at least one post-treatment efficacy assessment. | Score on a Scale | MEAN |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis | Amifampridine Phosphate - Placebo | Study Period 1: CFB in SGI Score at Day 8 | -0.13 | Standard Deviation 1.81 | Scores on a scale |
| Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis | Placebo - Amifampridine Phosphate | Study Period 1: CFB in SGI Score at Day 8 | -1.75 | Standard Deviation 2.12 | Scores on a scale |
| Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis | Amifampridine Phosphate - Placebo | Study Period 2 : CFB in SGI Score at Day 29 | -0.75 | Standard Deviation 1.75 | Scores on a scale |
| Subject Global Impression (SGI) Score Summary: Mann-Whitney Main Effects Test Results; SGI Score Mixed Model Analysis | Placebo - Amifampridine Phosphate | Study Period 2 : CFB in SGI Score at Day 29 | -1.14 | Standard Deviation 1.68 | Scores on a scale |
| Motor Function Measure 32 (MFM-32) Score Summary: Mann-Whitney Main Effect Test Results, MFM-32 Score Mixed Model Analysis | Amifampridine Phosphate - Placebo | Study Period 1: CFB in Overall MFM-32 Score at Day 8 | 3.00 | Standard Deviation 3.92 | Score on a Scale |
| Motor Function Measure 32 (MFM-32) Score Summary: Mann-Whitney Main Effect Test Results, MFM-32 Score Mixed Model Analysis | Placebo - Amifampridine Phosphate | Study Period 1: CFB in Overall MFM-32 Score at Day 8 | -0.17 | Standard Deviation 4.58 | Score on a Scale |
| Motor Function Measure 32 (MFM-32) Score Summary: Mann-Whitney Main Effect Test Results, MFM-32 Score Mixed Model Analysis | Amifampridine Phosphate - Placebo | Study Period 2: CFB in Overall MFM-32 Score at Day 29 | 0.75 | Standard Deviation 2.22 | Score on a Scale |
| Motor Function Measure 32 (MFM-32) Score Summary: Mann-Whitney Main Effect Test Results, MFM-32 Score Mixed Model Analysis | Placebo - Amifampridine Phosphate | Study Period 2: CFB in Overall MFM-32 Score at Day 29 | -0.60 | Standard Deviation 0.89 | Score on a Scale |
| Motor Function Measure 20 (MFM-20) Score Mixed Model Analysis, MFM-20 Score Mixed Model Analysis | Amifampridine Phosphate - Placebo | Study Period 1: CFB in Overall MFM-20 Score at Day 8 | -1.50 | Standard Deviation 4.20 | Score on a Scale |
| Motor Function Measure 20 (MFM-20) Score Mixed Model Analysis, MFM-20 Score Mixed Model Analysis | Placebo - Amifampridine Phosphate | Study Period 1: CFB in Overall MFM-20 Score at Day 8 | -0.50 | Standard Deviation 6.36 | Score on a Scale |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Amifampridine Phosphate - Placebo | 8 | |
| Overall Study | STARTED | Placebo - Amifampridine Phosphate | 8 | |
| Overall Study | STARTED | Amifampridine Phosphate Only | 4 | |
| Overall Study | COMPLETED | Amifampridine Phosphate - Placebo | 7 | |
| Overall Study | COMPLETED | Placebo - Amifampridine Phosphate | 7 | |
| Overall Study | COMPLETED | Amifampridine Phosphate Only | 0 | |
| Overall Study | NOT COMPLETED | Amifampridine Phosphate - Placebo | 1 | |
| Overall Study | NOT COMPLETED | Placebo - Amifampridine Phosphate | 1 | |
| Overall Study | NOT COMPLETED | Amifampridine Phosphate Only | 4 |
Source ClinicalTrials.gov
Adverse events58
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 | Ear haemorrhage | Ear and labyrinth disorders | Amifampridine Phosphate | 1 | 20 | |
| other | Ear haemorrhage | Ear and labyrinth disorders | Placebo | 0 | 20 | |
| other | Ear pain | Ear and labyrinth disorders | Amifampridine Phosphate | 0 | 20 | |
| other | Ear pain | Ear and labyrinth disorders | Placebo | 1 | 20 | |
| other | Diarrhoea | Gastrointestinal disorders | Amifampridine Phosphate | 1 | 20 | |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 0 | 20 | |
| other | Food poisoning | Gastrointestinal disorders | Amifampridine Phosphate | 1 | 20 | |
| other | Food poisoning | Gastrointestinal disorders | Placebo | 0 | 20 | |
| other | Nausea | Gastrointestinal disorders | Amifampridine Phosphate | 1 | 20 | |
| other | Nausea | Gastrointestinal disorders | Placebo | 1 | 20 |
1 to 10 of 58Source ClinicalTrials.gov
See all 58 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Amifampridine Phosphate | 0 | 20 |
| Total, all-cause mortality | Placebo | 0 | 20 |
| Total, other adverse events | Amifampridine Phosphate | 11 | 20 |
| Total, other adverse events | Placebo | 6 | 20 |
| Total, serious adverse events | Amifampridine Phosphate | 1 | 20 |
| Total, serious adverse events | Placebo | 1 | 20 |
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Amifampridine Phosphate vs Placebo: deaths from any cause | Trial comparisons |
| Amifampridine Phosphate vs Placebo: serious adverse events | Trial comparisons |
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/NCT02562066
GET https://conductscience.org/api/trials/v1/NCT02562066/events?page=2