Trial · NCT02829268
A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
NCT02829268, Phase 1, Phase 2, Completed, Washington University School of Medicine; results first posted 2024-03-13
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome. Trials, record NCT02829268. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT02829268
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
- NCT number
- NCT02829268
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 21 participants, actual
- Lead sponsor
- Washington University School of Medicine
- Conditions
- Wolfram Syndrome, Diabetes Mellitus, Optic Nerve Atrophy, Ataxia
- Interventions
- dantrolene sodium
- First posted
- 2016-07-12
- Last updated
- 2024-03-13
- Results first posted
- 2024-03-13
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests | 6 months | The investigators assess the safety and tolerability of dantrolene sodium administered orally at upper end of therapeutic dose range for 6 months in patients with Wolfram syndrome. More specifically, the investigators perform liver function tests to check the levels of certain enzymes and proteins in participants' blood. Levels that are higher or lower than normal can indicate liver problems. The liver function tests include: Alanine transaminase (ALT), Aspartate transaminase (AST), Alkaline Phosphatase (AP), and bilirubin. |
| Secondary | Changes in C-peptide Levels in Participants Assessed by the ELISA Assay | 6 months | The investigators determine the effect of dantrolene sodium on residual beta cell functions. The investigators monitor base-line C-peptide levels in participants' blood. The investigators also monitor C-peptide levels in participant's blood during the oral mixed meal tolerance test. The night before the oral mixed meal tolerance test, the participants will turn their insulin pump basal rate to 50% of the normal rate at midnight or take half of their evening dose of Lantus insulin and fasted from midnight until the test at 8 a.m. The mixed meal consists of 6 ml/kg (maximum 360 ml) of Boost Original (Société des Produits Nestlé S.A., Vevey, Switzerland). Blood for glucose and C-peptide measurement will be drawn at time 0 (fasting) and 30 minutes after the Boost. If a subject's fasting glucose exceeds 11.1 mmol/l, the test will not be performed, but fasting glucose and C-peptide will be obtained. |
| Secondary | Changes in Visual Functioning in Participants Assessed by Visual Functioning Questionnaire-25. | 6 months | Changes in Visual Functioning in participants assessed by Visual Functioning Questionnaire-25. The Visual Functioning Questionnaire-25 (VFQ-25) is divided into several subdomains, each assessing a specific aspect of visual functioning and its impact on an individual's life. There are a total of 11 subdomains in the VFQ-25. To calculate the total score on the VFQ-25, we follow these steps: 1. Calculate Subdomain Scores, 2. Weighted Sum 3. Calculate Total Score VFQ-25 provides scores that range from 0 to 100. The total score represents the overall impact of visual functioning on the individual's quality of life, with higher scores indicating better quality of life and less impact from vision problems. |
| Secondary | Changes in Best-corrected Visual Acuity in Participants Measured by LogMar Score | 6 months | Best-corrected visual acuity is assessed using the Snellen optotype and then converted into LogMar Scores (Minimum: -0.30, Maximum: 3.0). A higher LogMar score signifies poorer vision. |
| Secondary | Changes in Neurological Functions in Participants Assessed by the Wolfram Unified Rating Scale (WURS) | 6 months | Neurological functions are assessed by the Wolfram Unified Rating Scale (WURS). The WURS is divided into the following subscales: Physical Assessment and Behavioral Assessment. Physical Assessment (34 items rated on a scale from 0 = no symptoms to 4 = highest severity, minimum: 0, Maximum: 136) and Behavioral Assessment (9 items rated on frequency and severity from 0 = normal behavior to 3 = highest severity, Minimum: 0, Maximum: 27). Subscale scores are summed to calculate the total scores (minimum: 0, Maximum: 163). Higher total scores indicate more severe neurological manifestations. |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests | 6 months | Participants | COUNT_OF_PARTICIPANTS | |
| SECONDARY | Changes in C-peptide Levels in Participants Assessed by the ELISA Assay | 6 months | ng/mL | MEAN | |
| SECONDARY | Changes in Visual Functioning in Participants Assessed by Visual Functioning Questionnaire-25. | 6 months | units on a scale | MEAN | |
| SECONDARY | Changes in Best-corrected Visual Acuity in Participants Measured by LogMar Score | 6 months | Patient 012 was excluded from analysis due to LogMar of 3 (No Light Perception) | LogMAR | MEAN |
| SECONDARY | Changes in Neurological Functions in Participants Assessed by the Wolfram Unified Rating Scale (WURS) | 6 months | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group20
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests | Pediatric | Baseline | 0 | Participants | |
| Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests | Adult | Baseline | 0 | Participants | |
| Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests | Pediatric | 6 months | 0 | Participants | |
| Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests | Adult | 6 months | 2 | Participants | |
| Changes in C-peptide Levels in Participants Assessed by the ELISA Assay | Pediatric | Baseline | 0.34 | Standard Deviation 0.07 | ng/mL |
| Changes in C-peptide Levels in Participants Assessed by the ELISA Assay | Adult | Baseline | 0.19 | Standard Deviation 0.05 | ng/mL |
| Changes in C-peptide Levels in Participants Assessed by the ELISA Assay | Pediatric | 6 months | 0.47 | Standard Deviation 0.13 | ng/mL |
| Changes in C-peptide Levels in Participants Assessed by the ELISA Assay | Adult | 6 months | 0.30 | Standard Deviation 0.09 | ng/mL |
| Changes in Visual Functioning in Participants Assessed by Visual Functioning Questionnaire-25. | Pediatric | Baseline | 68 | Standard Error 2.3 | units on a scale |
| Changes in Visual Functioning in Participants Assessed by Visual Functioning Questionnaire-25. | Adult | Baseline | 74.3 | Standard Error 1.3 | units on a scale |
1 to 10 of 20Source ClinicalTrials.gov
See all 20 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Pediatric | 8 | |
| Overall Study | STARTED | Adult | 13 | |
| Overall Study | COMPLETED | Pediatric | 8 | |
| Overall Study | COMPLETED | Adult | 11 | |
| Overall Study | NOT COMPLETED | Pediatric | 0 | |
| Overall Study | NOT COMPLETED | Adult | 2 |
Source ClinicalTrials.gov
Adverse events42
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 | Diarrhea | Gastrointestinal disorders | Pediatric | 2 | 8 | |
| other | Diarrhea | Gastrointestinal disorders | Adult | 3 | 11 | |
| other | Weakness | Nervous system disorders | Pediatric | 3 | 8 | |
| other | Weakness | Nervous system disorders | Adult | 1 | 11 | |
| other | Dizziness | Nervous system disorders | Pediatric | 2 | 8 | |
| other | Dizziness | Nervous system disorders | Adult | 1 | 11 | |
| other | Elevated Hepatic enzymes | Hepatobiliary disorders | Pediatric | 0 | 8 | |
| other | Elevated Hepatic enzymes | Hepatobiliary disorders | Adult | 2 | 11 | |
| other | Nausea | Gastrointestinal disorders | Pediatric | 0 | 8 | |
| other | Nausea | Gastrointestinal disorders | Adult | 2 | 11 |
1 to 10 of 42Source ClinicalTrials.gov
See all 42 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pediatric | 0 | 8 |
| Total, all-cause mortality | Adult | 0 | 11 |
| Total, other adverse events | Pediatric | 8 | 8 |
| Total, other adverse events | Adult | 11 | 11 |
| Total, serious adverse events | Pediatric | 0 | 8 |
| Total, serious adverse events | Adult | 0 | 11 |
Source ClinicalTrials.gov
Papers2
The papers the trial lists on ClinicalTrials.gov.
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| R21DK113487 | CLINICAL TRIAL OF DANTROLENE SODIUM IN PATIENTS WITH WOLFRAM SYNDROME | URANO, FUMIHIKO | WASHINGTON UNIVERSITY | NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES | 2017 to 2018 |
Source NIH RePORTER
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Pediatric vs Adult: deaths from any cause | Trial comparisons |
| Pediatric vs Adult: serious adverse events | Trial comparisons |
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/NCT02829268
GET https://conductscience.org/api/trials/v1/NCT02829268/events?page=2