Trial · NCT03717909
Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
NCT03717909, Phase 2, Completed, University of Birmingham
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome. Trials, record NCT03717909. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03717909
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
- NCT number
- NCT03717909
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 63 participants, actual
- Lead sponsor
- University of Birmingham
- Conditions
- Wolfram Syndrome
- Interventions
- Sodium Valproate 200Mg E/C Tablet, Sodium Valproate matched placebo
- First posted
- 2018-10-24
- Last updated
- 2026-05-05
Source ClinicalTrials.gov
Registered outcomes34
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Visual acuity (VA) | 36 months | Visual acuity (VA) is measured on the logMAR scale by sight tests in clinic using Early treatment diabetic retinopathy study (ETDRS) charts. Values are taken for each eye separately, both uncorrected, and corrected with glasses or contact lenses, and can range from 0, which represents perfect vision i.e. 20/20 (values of -0.1 and -0.2 are also possible representing better than perfect vision), to +2 which represents near blindness i.e. 20/2000. Increases in logMAR represent deterioration. |
| Secondary | Safety - adverse events | 37 months | measured by adverse events frequency, type and grade according to CTCAE v4 |
| Secondary | Tolerability - highest treatment dose | 36 months | measured by dose achieved |
| Secondary | Tolerability - duration of treatment | 36 months | measured by days of treatment |
| Secondary | Tolerability - dosing modifcation | 36 months | measured by treatment-related dose reductions and discontinuations |
| Secondary | Pons Volume | 37 months (+/- 6 months) | a surrogate marker for neurodegeneration, measured and recorded in mm3 by standardised analysis of MRI images of the Pons, and brain substructure volumes. |
| Secondary | Brainstem volume | 37 months (+/- 6 months) | measured by MRI as with Pons Volume |
| Secondary | Retinal nerve thickness | 37 months | measured by Optical Coherence Tomography |
| Secondary | Colour vision | 37 months | measured by Hardy Rand and Rittler test |
| Secondary | Visual fields | 37 months | measured by the local centre standard process (the same technique must be used throughout the patient participation to the study) |
1 to 10 of 34Source ClinicalTrials.gov
See all 34 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.
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 40010822 | DERIVED |
Source PubMed
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/NCT03717909
GET https://conductscience.org/api/trials/v1/NCT03717909/registered?page=2