A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT02829268 |
Recruitment Status :
Active, not recruiting
First Posted : July 12, 2016
Last Update Posted : January 11, 2021
|
Tracking Information | |||||
---|---|---|---|---|---|
First Submitted Date ICMJE | July 6, 2016 | ||||
First Posted Date ICMJE | July 12, 2016 | ||||
Last Update Posted Date | January 11, 2021 | ||||
Actual Study Start Date ICMJE | January 2017 | ||||
Estimated Primary Completion Date | December 2021 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Number of participants with treatment-related adverse events as assessed by liver function tests [ Time Frame: 6 months with an optional extension phase up to 24 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.
|
||||
Original Primary Outcome Measures ICMJE |
Number of participants with treatment-related adverse events as assessed by liver function tests [ Time Frame: 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) and Aspartate transaminase (AST).
|
||||
Change History | |||||
Current Secondary Outcome Measures ICMJE |
|
||||
Original Secondary Outcome Measures ICMJE |
|
||||
Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome | ||||
Official Title ICMJE | A Phase 1b/2a Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome | ||||
Brief Summary | Wolfram syndrome is a rare genetic disorder characterized by juvenile-onset diabetes mellitus, diabetes insipidus, optic nerve atrophy, hearing loss, and neurodegeneration. The purpose of this study is to assess the safety and tolerability of dantrolene sodium in patients with Wolfram syndrome. In addition, we will assess the efficacy of dantrolene sodium on the cardinal manifestations of Wolfram syndrome, including visual acuity, remaining beta cell functions, and neurological functions. There is a screening period up to 56 days, a 6-month treatment period with an optional extension phase up to 24 months, and a 4-week safety follow-up period. Study assessments include medical & medication history, physical exams, neurological exams, eye exams, endocrine exams, vital signs, height, weight, electrocardiograms, blood and urine tests, pregnancy test if applicable, and questionnaires. |
||||
Detailed Description | Not Provided | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 Phase 2 |
||||
Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
||||
Condition ICMJE |
|
||||
Intervention ICMJE | Drug: dantrolene sodium
The purpose of this study is to assess the safety and tolerability of dantrolene sodium in patients with Wolfram syndrome. In addition, we will assess the efficacy of dantrolene sodium on the cardinal manifestations of Wolfram syndrome, including visual acuity, remaining beta cell functions, and neurological functions. There is a screening period up to 56 days, a 6-month treatment period with an optional extension phase up to 24 months, and a 4-week safety follow-up period. Study assessments include medical & medication history, physical exams, neurological exams, eye exams, endocrine exams, vital signs, height, weight, electrocardiograms, blood and urine tests, pregnancy test if applicable, and questionnaires. |
||||
Study Arms ICMJE |
|
||||
Publications * | Urano F. Wolfram Syndrome: Diagnosis, Management, and Treatment. Curr Diab Rep. 2016 Jan;16(1):6. doi: 10.1007/s11892-015-0702-6. Review. | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
|||||
Recruitment Information | |||||
Recruitment Status ICMJE | Active, not recruiting | ||||
Estimated Enrollment ICMJE |
50 | ||||
Original Estimated Enrollment ICMJE |
25 | ||||
Estimated Study Completion Date ICMJE | December 2021 | ||||
Estimated Primary Completion Date | December 2021 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | Inclusion Criteria: Patients must meet all of the following criteria to be eligible for enrolment:
Exclusion Criteria: Patients who meet any of the following criteria are not eligible for this study:
|
||||
Sex/Gender ICMJE |
|
||||
Ages ICMJE | 5 Years to 60 Years (Child, Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
Removed Location Countries | |||||
Administrative Information | |||||
NCT Number ICMJE | NCT02829268 | ||||
Other Study ID Numbers ICMJE | 201607006 R21DK113487 ( U.S. NIH Grant/Contract ) |
||||
Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
|
||||
IPD Sharing Statement ICMJE |
|
||||
Responsible Party | Washington University School of Medicine | ||||
Study Sponsor ICMJE | Washington University School of Medicine | ||||
Collaborators ICMJE |
|
||||
Investigators ICMJE |
|
||||
PRS Account | Washington University School of Medicine | ||||
Verification Date | January 2021 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |