Trial · NCT03781479
Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients
NCT03781479, Phase 2, Completed, Catalyst Pharmaceuticals, Inc.; results first posted 2021-06-01
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients. Trials, record NCT03781479. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03781479
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients
- NCT number
- NCT03781479
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 13 participants, actual
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.
- Conditions
- Muscular Atrophy, Spinal
- Interventions
- Amifampridine Phosphate, Placebo Oral Tablet
- First posted
- 2018-12-20
- Last updated
- 2021-06-01
- Results first posted
- 2021-06-01
Source ClinicalTrials.gov
Registered outcomes1
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Hammersmith Functional Motor Scale Expanded (HFMSE) Summary Statistics and Mixed Model Analysis | Screening, the first (Day 1) and last day (Day 0) of the Run-in period, during Period 1 at Day 7 and Day 14, and during Period 2 at Day 21 and Day 28 | Hammersmith Functional Motor Scale Expanded (HFMSE) assesses motor function by functional item in order of progressive difficulty, with higher values showing higher function abilities. Each item is scored on a scale of 0-2 with 2 representing item achieved unaided and 0 representing inability to achieve item. Each item was assessed by the patient at Screening, the first (Day 1) and last day (Day 0) of the Run-in period, during Period 1 at Day 7 and Day 14, and during Period 2 at Day 21 and Day 28. The total HFMSE score was calculated as the sum of each item score, with a maximum score of 66 (all items achieved unaided) and minimum score of 0 (all items failed). Change from baseline (CFB) will be assessed from Day 0 to Day 28. A mixed effects liner model was fit with the HFMSE change from baseline (CFB) scores at Day 28 as a response and treatment, sequence, and treatment by sequence as fixed effect terms and patient as a random effect. |
Source ClinicalTrials.gov
Outcomes1
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Hammersmith Functional Motor Scale Expanded (HFMSE) Summary Statistics and Mixed Model Analysis | Screening, the first (Day 1) and last day (Day 0) of the Run-in period, during Period 1 at Day 7 and Day 14, and during Period 2 at Day 21 and Day 28 | 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) and had at least one post-treatment efficacy assessment. Patients are compared for efficacy according to the treatment to which they were randomized, regardless of the treatment actually received. | Overall Score | LEAST_SQUARES_MEAN |
Source ClinicalTrials.gov
Results by group2
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Hammersmith Functional Motor Scale Expanded (HFMSE) Summary Statistics and Mixed Model Analysis | Amifampridine Phosphate | 0.208 | Standard Error 0.326 | Overall Score | |
| Hammersmith Functional Motor Scale Expanded (HFMSE) Summary Statistics and Mixed Model Analysis | Placebo | -0.583 | Standard Error 0.326 | Overall Score |
Source ClinicalTrials.gov
Participant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Amifampridine Phosphate - Placebo | 6 | |
| Overall Study | STARTED | Placebo - Amifampridine Phosphate | 6 | |
| Overall Study | STARTED | Not Randomized | 1 | |
| Overall Study | COMPLETED | Amifampridine Phosphate - Placebo | 6 | |
| Overall Study | COMPLETED | Placebo - Amifampridine Phosphate | 6 | |
| Overall Study | COMPLETED | Not Randomized | 0 | |
| Overall Study | NOT COMPLETED | Amifampridine Phosphate - Placebo | 0 | |
| Overall Study | NOT COMPLETED | Placebo - Amifampridine Phosphate | 0 | |
| Overall Study | NOT COMPLETED | Not Randomized | 1 |
Source ClinicalTrials.gov
Adverse events24
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 | Hypoaesthesia oral | Gastrointestinal disorders | Amifampridine | 1 | 12 | |
| other | Hypoaesthesia oral | Gastrointestinal disorders | Placebo | 0 | 12 | |
| other | Paraesthesia oral | Gastrointestinal disorders | Amifampridine | 3 | 12 | |
| other | Paraesthesia oral | Gastrointestinal disorders | Placebo | 0 | 12 | |
| other | Fatigue | General disorders | Amifampridine | 0 | 12 | |
| other | Fatigue | General disorders | Placebo | 1 | 12 | |
| other | Gait disturbance | General disorders | Amifampridine | 0 | 12 | |
| other | Gait disturbance | General disorders | Placebo | 1 | 12 | |
| other | Influenza like illness | General disorders | Amifampridine | 1 | 12 | |
| other | Influenza like illness | General disorders | Placebo | 0 | 12 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Amifampridine | 0 | 12 |
| Total, all-cause mortality | Placebo | 0 | 12 |
| Total, other adverse events | Amifampridine | 5 | 12 |
| Total, other adverse events | Placebo | 3 | 12 |
| Total, serious adverse events | Amifampridine | 0 | 12 |
| Total, serious adverse events | Placebo | 0 | 12 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 35763114 | DERIVED |
Source PubMed
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 vs Placebo: deaths from any cause | Trial comparisons |
| Amifampridine 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/NCT03781479
GET https://conductscience.org/api/trials/v1/NCT03781479/events?page=2