Trial · NCT03819660
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
NCT03819660, Phase 2, Terminated, Catalyst Pharmaceuticals, Inc.; results first posted 2023-11-30
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3. Trials, record NCT03819660. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03819660
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
- NCT number
- NCT03819660
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 13 participants, actual
- Lead sponsor
- Catalyst Pharmaceuticals, Inc.
- Conditions
- Spinal Muscular Atrophy Type 3
- Interventions
- Amifampridine Phosphate 10 MG Oral Tablet
- First posted
- 2019-01-28
- Last updated
- 2023-11-30
- Results first posted
- 2023-11-30
- Limitations and caveats
The results of efficacy are limited to the assessment of quality of life using the INQoL. The premature interruption of the study determined that few patients had available data of INQoL from Month 6 onwards.
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Long-term Safety and Tolerability of Amifampridine | 18 months | Number of subjects with treatment emergent adverse events (TEAE). |
| Secondary | To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL). | Screening to end of study. | Quality of life (QoL): the Individualized Quality of Life for neuromuscular disease (INQoL) or the Pediatric Quality of Life (PEDSQLTM) was used for adult or pediatric patients, respectively. Since there were no pediatric patients, none of the patients completed the PEDSQL. The INQol evaluated weakness, pain, fatigue, double vision, muscle locking, droopy eyelids, swallowing difficulties, activities, social relationships, emotions and body image. Each of these areas were measured in four categories as follows: 1- Incidence (0= No, 1 =Yes), 2-Severity (0= None to 7 = extreme), 3- Impact - (0= None to 6 = extreme) and 4-Importance (0= None to 6 = extreme). The numbers were summative and are input to the QOL calculation, which is a percentage of severity on a scale of 0-100. The mean value was taken across this population. The higher scores were a worse outcome. |
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 | Long-term Safety and Tolerability of Amifampridine | 18 months | TEAE = any AE started at or after the first dose of drug for the current Long-Term Study. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL). | Screening to end of study. | 11 patients completed the screening Quality of Life Score and only 9 patients completed the EOS Quality of Life Score . | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group3
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Long-term Safety and Tolerability of Amifampridine | Amifampridine Phosphate | 5 | Participants | ||
| To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL). | Amifampridine Phosphate | screening | 33.737 | Standard Deviation 17.677 | score on a scale |
| To Assess the Clinical Efficacy of Amifampridine Phosphate Over Time in Patients With SMA Type 3 Based on Changes in Quality of Life (QoL). | Amifampridine Phosphate | End of Study | 39.012 | Standard Deviation 14.337 | score on a scale |
Source ClinicalTrials.gov
Participant flow3
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Amifampridine Phosphate | 13 | |
| Overall Study | COMPLETED | Amifampridine Phosphate | 13 | |
| Overall Study | NOT COMPLETED | Amifampridine Phosphate | 0 |
Source ClinicalTrials.gov
Adverse events14
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 | Paraesthesia oral | Gastrointestinal disorders | Amifampridine Phosphate | 3 | 13 | 3 |
| other | ABDOMINAL PAIN UPPER | Gastrointestinal disorders | Amifampridine Phosphate | 2 | 13 | 2 |
| other | HYPERPYREXIA | General disorders | Amifampridine Phosphate | 2 | 13 | 2 |
| other | FEELING HOT | General disorders | Amifampridine Phosphate | 1 | 13 | 1 |
| other | INFLUENZA LIKE ILLNESS | General disorders | Amifampridine Phosphate | 1 | 13 | 1 |
| other | MALAISE | General disorders | Amifampridine Phosphate | 1 | 13 | 1 |
| other | FALL | Injury, poisoning and procedural complications | Amifampridine Phosphate | 1 | 13 | 1 |
| other | POST LUMBAR PUNCTURE SYNDROME | General disorders | Amifampridine Phosphate | 1 | 13 | 1 |
| other | HYPOKALAEMIA | Metabolism and nutrition disorders | Amifampridine Phosphate | 1 | 13 | 1 |
| other | Paraesthesia | Nervous system disorders | Amifampridine Phosphate | 1 | 13 | 1 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Amifampridine Phosphate | 0 | 13 |
| Total, other adverse events | Amifampridine Phosphate | 5 | 13 |
| Total, serious adverse events | Amifampridine Phosphate | 1 | 13 |
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/NCT03819660
GET https://conductscience.org/api/trials/v1/NCT03819660/registered