Trial · NCT04940572
Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
NCT04940572, Phase 2, Unknown, Centre d'Etude des Cellules Souches
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome. Trials, record NCT04940572. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04940572
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy Study of Daily Administration of VPA in Patients Affected by Wolfram Syndrome
- NCT number
- NCT04940572
- Status
- Unknown
- Phases
- Phase 2
- Enrollment
- 23 participants, estimated
- Lead sponsor
- Centre d'Etude des Cellules Souches
- Conditions
- Wolfram Syndrome
- Interventions
- Depakine
- First posted
- 2021-06-25
- Last updated
- 2023-11-28
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Preservation of auditory function. | Baseline - Week 156 | Preservation of auditory function defined as no decrease higher than 5 dB in hearing at 8 kHz in high frequency average (HFA) over three years in patients with Wolfram syndrome with a deficit of at least 20d dB at 8 kHz treated with VPA at optimal dose corresponding to the plasma level between 40 and 100 mg/l (ie, 300 to 700 micro mol/l). |
| Secondary | Safety endpoint | Baseline - Week 156 | Overall incidence of adverse events and serious adverse events as well as laboratory assessments will be evaluated for each group and for the study as a whole. |
| Secondary | Ventral Pons Volume measure | Baseline - Week 156 | Ventral Pons Volume measured and recorded in mm3 by standardised analysis of MRI at baseline, at visit 8 (Week 52) and at the final visit. |
| Secondary | Insulin and or desmopressin requirements | Baseline - Week 156 | Insulin and or desmopressin requirements will be assessed whenever the patient is under one or both treatments in order to document potential benefit from VPA on diabetes mellitus or diabetes insipidus |
| Secondary | Visual acuity assessment | Baseline - Week 156 | Visual acuity will be assessed using standard ETDRS measures and visual field recording at baseline, every six months during the first year of follow-up and at the final visit |
| Secondary | Retinal nerve thikness measure | Baseline - Week 156 | Retinal nerve thikness measured by OCT measures at baseline, every six months during the first year of follow-up and at the final visit |
| Secondary | Balance measured by Mini-BESTest | Baseline - Week 156 | Balance, measured by Mini-BESTest (Appendix 1) at baseline, at visit 8 (Week 52) and at the final visit. |
| Secondary | Quality of sleep assessment on pediatric population - Qualitative questionnaire (no scale) | Baseline - Week 156 | Sleep will be investigated by Pediatric Sleep Questionnaire at baseline, at visit 8 (Week 52) and at final visit. |
| Secondary | Quality of sleep assessment on adult population - Qualitative questionnaire (no scale) | Baseline - Week 156 | Sleep will be investigated by Pittsburg Sleep Quality Index Self-Report on adult population at baseline, at visit 8 (Week 52) and at final visit. |
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/NCT04940572
GET https://conductscience.org/api/trials/v1/NCT04940572/registered