Trial · NCT04226924
Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
NCT04226924, Phase 2, Withdrawn, Bioblast Pharma Ltd.
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose. Trials, record NCT04226924. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04226924
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Treatment of Oculopharyngeal Muscular Dystrophy With Trehalose
- NCT number
- NCT04226924
- Status
- Withdrawn
- Phases
- Phase 2
- Enrollment
- 0 participants, actual
- Lead sponsor
- Bioblast Pharma Ltd.
- Conditions
- Oculopharyngeal Muscular Dystrophy
- Interventions
- Trehalose
- First posted
- 2020-01-13
- Last updated
- 2020-01-13
Source ClinicalTrials.gov
Registered outcomes8
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Drinking Test Time | 24 weeks | Change from baseline in timed drinking tests with 80 cc of ice-cold water and nectar. |
| Secondary | Muscle Strength Testing | 24 weeks | Change from baseline in strength tests in selected muscle groups as measured by a handheld dynamometer |
| Secondary | Stair Climb Test | 24 weeks | Change from baseline in functional muscle testing as measured by the Stair Climb test |
| Secondary | Timed Up and Go Test | 24 weeks | Change from baseline in functional muscle testing as measured by the Timed Up and Go (TUG) test |
| Secondary | 30-Second Lift Test | 24 weeks | Change from baseline in functional muscle testing as measured by 30-Second Lift test |
| Secondary | EuroQol-5D-5L | 24 weeks | Change from baseline in health status using the EuroQol-5D-5L Questionnaire |
| Secondary | Swallowing Quality of Life | 24 weeks | Change from baseline in quality of life using modified Swallowing Quality of Life Questionnaire |
| Secondary | Sydney Swallow Questionnaire | 24 weeks | Change from baseline in quality of life using Sydney Swallow Questionnaire |
Source ClinicalTrials.gov
Outcomes0
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/NCT04226924
GET https://conductscience.org/api/trials/v1/NCT04226924/registered