Trial · NCT05219487
Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
NCT05219487, Completed, Bakri Elsheikh
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration. Trials, record NCT05219487. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05219487
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
- NCT number
- NCT05219487
- Status
- Completed
- Enrollment
- 18 participants, actual
- Lead sponsor
- Bakri Elsheikh
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Risdiplam
- First posted
- 2022-02-02
- Last updated
- 2026-02-23
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Decrement on repetitive nerve stimulation | Baseline | Change in the percent decrement of the compound muscle action potential amplitude assessed using 3 Hz repetitive nerve stimulation (RNS) of the spinal accessory nerve compared to historical controls. If CMAP amplitude is absent at spinal accessory nerve, SMA, ulnar CMAP will be used to assess NMJ transmission. |
| Secondary | Six Minute Walk Test (6MWT) Total Distance | Baseline | The score difference between groups stratified using a 10% decrement cutoff |
| Secondary | Six Minute Walk Test Difference in Distance Between the First and Final minute | Baseline | The score difference between groups stratified using a 10% decrement cutoff |
| Secondary | Modified Spinal Muscular Atrophy Function Rating Scale (SMA-FRS) | Baseline | The score difference between groups stratified using a 10% decrement cutoff (scale 0-40, higher score is better). |
| Secondary | Revised Upper Limb Module (RULM) | Baseline | The score difference between groups stratified using a 10% decrement cutoff (scale 0-37, a higher score is better) |
| Secondary | Hammersmith Functional Rating Scale (HFMSE) | Baseline | The score difference between groups stratified using a 10% decrement cutoff (0-66, a higher score is better |
| Secondary | Fatigue Severity Scale (FSS) | Baseline | The score difference between groups stratified using a 10% decrement cutoff (9-63, the higher the score the higher the fatigue) |
| Secondary | Children's Hospital of Philadelphia - Adult Test of Neuromuscular Disorders (CHOP-ATEND) | Baseline | The score difference between groups stratified using a 10% decrement cutoff analyses (0-52, a higher score is better) |
| Secondary | Strength Measure using Hand Held Dynamometry | Baseline | Strength measurements of of bilateral shoulder abduction, elbow flexion, elbow extension, hip flexion, hip abduction, hip adduction, knee flexion and knee extension. The score difference between groups stratified using a 10% decrement cutoff analyses. |
| Secondary | Ulnar Compound Muscle Action Potential (Ulnar -CMAP) | Baseline | The difference in amplitudes between groups stratified using a 10% decrement cutoff |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes0
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
None in this release.
Results by group0
Each measurement the trial posted, group by group, in the words and numbers it posted.
None in this release.
Participant flow0
How many started, completed or left each group, period by period.
None in this release.
Adverse events0
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
None in this release.
Adverse event totals0
The totals the trial posted for each group.
None in this release.
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/NCT05219487
GET https://conductscience.org/api/trials/v1/NCT05219487/registered