Trial · NCT07914049
Registered outcomes18
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
1 to 18 of 18
Download this page as JSON| 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. |
| Secondary | Muscle thickness by ultrasound | Enrollment to 2 years | Standardized muscle thickness at standard positions on the left side measured by ultrasound and summed (biceps, vastus lateralis, and tibialis anterior) |
| Secondary | Muscle ultrasound grayscale intensity | Enrollment to 2 years | Mean ultrasound grayscale intensity at selected muscles measured in pixel brightness within a manual region of interest |
| Secondary | Muscle ultrasound grayscale variability | Enrollment to 2 years | Standard deviation of pixel grayscale intensity within a manually selected region of interest at selected muscles. |
| Secondary | FVC | Enrollment to 2 years | Forced vital capacity as percent predicted |
| Secondary | 4SC | Enrollment to 2 years | Timed 4 stair climb velocity |
| Secondary | Electrophysiology - Compound Motor Action Potential (CMAP) | Enrollment to 2 years | Compound Muscle Action Potential (CMAP) at standard sites: A noninvasive electrophysiologic measure of the muscle's electrical response following nerve stimulation. CMAP amplitude and area are used to assess the integrity of motor nerve pathways and overall muscle activation. Reported as amplitude (millivolts \[mV\]) and, when assessed, area (millivolt-milliseconds \[mV·ms\]). Higher values generally indicate better neuromuscular function; there is no fixed range for this assessment (no minimum or maximum). |
| Secondary | Electrophysiology - Motor Unit Number Index (MUNIX) | Enrollment to 2 years | Motor Unit Number Index (MUNIX) at standard sites: A noninvasive electrophysiologic measure that estimates the number of functioning motor units within a muscle using nerve conduction and surface electromyography data. Calculated from CMAP and surface electromyography (EMG) recordings and reported as a unitless index score. Higher values generally indicate greater preservation of motor units; there is no fixed range for this assessment (no minimum or maximum). |
| Secondary | Electrophysiology - Motor Unit Size Index (MUSIX) | Enrollment to 2 years | Motor Unit Size Index (MUSIX): An electrophysiologic measure derived from CMAP and MUNIX results that estimates the average size of functioning motor units. Changes in MUSIX may reflect compensatory enlargement of remaining motor units associated with neuromuscular disease progression. Derived from CMAP and MUNIX measurements and reported as a unitless index score representing estimated average motor unit size. There is no minimum or maximum value. |
Source ClinicalTrials.govtrials-2026-11-12-0.5.0
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 list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/trials/v1/NCT07914049/registered