Trial · NCT07914049
Personalized Disease Modeling for Rare Genetic Myopathies
NCT07914049, Enrolling by invitation, Akron Children's Hospital
3 sourcestrials-2026-11-12-0.4.0
Cite this record
ConductScience Library. Personalized Disease Modeling for Rare Genetic Myopathies. Trials, record NCT07914049. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT07914049
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Personalized Disease Modeling for Rare Genetic Myopathies
- NCT number
- NCT07914049
- Status
- Enrolling by invitation
- Enrollment
- 3 participants, estimated
- Lead sponsor
- Akron Children's Hospital
- Conditions
- Congenital Myopathies, Muscular Dystrophy
- First posted
- 2026-11-03
- Last updated
- 2026-11-03
Source ClinicalTrials.gov
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Medical Research Council (MRC) sum score | Enrollment to 2 years | Sum of bilateral Medical Research Council grades for arm abduction, arm flexion, arm extension, hip flexion, knee extension, and ankle dorsiflexion. Scoring values are 0 (Minimum) to 60 (Maximum), with a higher score representing better outcomes. |
| Primary | North Star Ambulatory Assessment (NSAA) | From enrollment to 2 years | The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy (DMD). It is usually used to monitor the progression of the disease and treatment effects. The activities in the 17-itm scale are graded as follows: 2 - "Normal" - no obvious modification of activity 1 - Modified method but achieves goal independent of physical assistance from another 0 - Unable to achieve independently This scale is ordinal with 34 as the maximum score indicating fully-independent function. The higher the score, the better outcome. |
| Secondary | 10 Meter Walk/Run | Enrollment to 2 years | Best time on 10-meter walk or run |
| Secondary | Supine to stand velocity | Enrollment to 2 years | Fastest time from lying to standing |
| Secondary | Pediatric Quality of Life Inventory 4.0 Generic Core Scales (PedsQL 4.0 GCS) | Enrollment to 2 years | Pediatric Quality of Life Inventory (PedsQL) 4.0 Generic Core Scales and Duchenne Muscular Dystrophy (DMD) Module : Patient-reported outcome measures (PROM) used to assess health-related quality of life in children with DMD. The PedsQL 4.0 Generic Core contains 23 items assessing physical, emotional, social, and school functioning, while the DMD Module includes disease-specific items addressing daily activities, treatment barriers, worry, and communication. Child self-report and parent proxy versions use a 5-point Likert scale (0 = never a problem to 4 = almost always a problem; ages 5-7 use a simplified 3-point scale). Responses are reverse-scored and transformed to a 0-100 scale, with higher scores indicating better quality of life. Scores are calculated for individual domains and total summary measures. |
| Secondary | Patient Global Impression of Change (PGI-C) | Enrollment to 2 years | Patient Global Impression of Change (PGI-C) and Clinician Global Impression of Change (CGI-C): Single-item assessments completed by the patient that measure overall change in the participant's condition since the start of treatment. Scores range from 1 to 7, where 1 = very much improved, 4 = no change, and 7 = very much worse. Lower scores indicate improvement, higher scores indicate worsening, and mid-range scores indicate little or no change. |
| Secondary | Clinician Global Impression of Change (CGI-C) | Enrollment to 2 years | Clinician Global Impression of Change (CGI-C): Single-item assessments completed by the clinician (CGI-C) that measure overall change in the participant's condition since the start of treatment. Scores range from 1 to 7, where 1 = very much improved, 4 = no change, and 7 = very much worse. Lower scores indicate improvement, higher scores indicate worsening, and mid-range scores indicate little or no change. |
| Secondary | SV95C | Enrollment to 2 years | 95%ile stride velocity based on wearable biosensor |
| Secondary | Grip strength | Enrollment to 2 years | Dynamometry for grip strength, measured in kilograms taken as the best of 3 trials on the best side |
| Secondary | Muscle Fat fraction | Enrollment to 2 years | Muscle Fat Fraction: Quantitative MRI assessment of muscle composition performed using an 8-point Dixon MRI sequence of the right thigh on a 3 Tesla (3T) MRI scanner. Muscle fat fraction is reported as the percentage of fat-containing voxels within the muscle, with higher percentages indicating greater fatty infiltration of muscle tissue. |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 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.
Papers31
The papers the trial lists on ClinicalTrials.gov.
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/NCT07914049
GET https://conductscience.org/api/trials/v1/NCT07914049/papers?page=2