Trial · NCT03796637
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
NCT03796637, Phase 2, Completed, PTC Therapeutics; results first posted 2022-04-05
3 sourcestrials-2026-11-12-0.4.0
Cite this record
ConductScience Library. A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren. Trials, record NCT03796637. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03796637
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) Who Have Been Treated With Ataluren
- NCT number
- NCT03796637
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 6 participants, actual
- Lead sponsor
- PTC Therapeutics
- Conditions
- Duchenne Muscular Dystrophy
- Interventions
- Ataluren
- First posted
- 2019-01-08
- Last updated
- 2022-04-05
- Other registries' IDs
- eudract:2019-001691-11
- Results first posted
- 2022-04-05
Source ClinicalTrials.gov
Registered outcomes2
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL) | Day 1 of biopsy | The mean dystrophin protein levels were measured by ECL. Dystrophin levels are reported by muscle group (gastrocnemius, tibialis anterior, and across muscle locations). Results below the limit of quantitation were imputed as half of lower limit of quantitation (LLOQ). LLOQ = 0.5 micrograms (μg)/milliliter (mL) |
| Secondary | Dystrophin Protein Levels as Determined by Immunohistochemistry | Day 1 of biopsy | Dystrophin levels by IHC mean membrane stain density are reported by muscle group (gastrocnemius, tibialis anterior, and across muscle locations). |
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 | Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL) | Day 1 of biopsy | ITT population included all enrolled participants with a valid assessment of dystrophin level, as measured by ECL. | nanograms (ng)/mg | MEAN |
| SECONDARY | Dystrophin Protein Levels as Determined by Immunohistochemistry | Day 1 of biopsy | ITT population included all enrolled participants with a valid assessment of dystrophin level, as measured by ECL. | ng/mg | MEAN |
Source ClinicalTrials.gov
Results by group6
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL) | Ataluren | Gastrocnemius | 0.0844 | Standard Deviation 0.05874 | nanograms (ng)/mg |
| Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL) | Ataluren | Tibialis Anterior | 0.1002 | Standard Deviation 0.08060 | nanograms (ng)/mg |
| Mean Dystrophin Levels as Measured by Electrochemiluminescence (ECL) | Ataluren | Across Muscle Locations | 0.1054 | Standard Deviation 0.08300 | nanograms (ng)/mg |
| Dystrophin Protein Levels as Determined by Immunohistochemistry | Ataluren | Gastrocnemius | 0.28817 | Standard Deviation 0.220547 | ng/mg |
| Dystrophin Protein Levels as Determined by Immunohistochemistry | Ataluren | Tibialis Anterior | 0.26578 | Standard Deviation 0.226510 | ng/mg |
| Dystrophin Protein Levels as Determined by Immunohistochemistry | Ataluren | Across Muscle Locations | 0.30483 | Standard Deviation 0.218600 | ng/mg |
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 | Ataluren | 6 | |
| Overall Study | COMPLETED | Ataluren | 6 | |
| Overall Study | NOT COMPLETED | Ataluren | 0 |
Source ClinicalTrials.gov
Adverse events2
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 | Puncture site discharge | General disorders | Ataluren | 1 | 6 | |
| other | Procedural pain | Injury, poisoning and procedural complications | Ataluren | 2 | 6 |
Source ClinicalTrials.gov
Adverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Ataluren | 0 | 6 |
| Total, other adverse events | Ataluren | 2 | 6 |
| Total, serious adverse events | Ataluren | 0 | 6 |
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.
Source
- Release
- trials-2026-11-12-0.4.0
- Release manifest SHA-256
- 0f4a8d8c48aee2a7573ae0fa94ecbeab70c1adbe449bbc95c424bc7d3e97b9d4
- Registry capture
- 2026-11-12
- Results copy, AACT
- 2026-03-23
- NIH clinical studies file
- 2026-11-13
Source ClinicalTrials.gov
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNone 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/NCT03796637
GET https://conductscience.org/api/trials/v1/NCT03796637/registered