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Muscle Relaxant

dantrolene sodium for Wolfram Syndrome

Phase 1 & 2

Waitlist Available

Research Sponsored by Washington University School of Medicine

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 6 months

Awards & highlights

Study Summary

This trial will study the safety and tolerability of dantrolene sodium in patients with Wolfram syndrome, as well as the efficacy of the drug on the cardinal manifestations of the disorder.

Eligible Conditions

Wolfram Syndrome
Ataxia
Diabetes
Optic Nerve Atrophy

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 6 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~6 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and 6 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of Participants With Treatment-related Adverse Events as Assessed by Liver Function Tests

Secondary outcome measures

Changes in Best-corrected Visual Acuity in Participants Measured by LogMar Score

Changes in C-peptide Levels in Participants Assessed by the ELISA Assay

Changes in Neurological Functions in Participants Assessed by the Wolfram Unified Rating Scale (WURS)

+1 more

Side effects data

From 2023 Phase 1 & 2 trial • 21 Patients • NCT02829268

38%

Weakness

38%

Hypoglycemia (mild)

38%

Fatigue

25%

Hyperglycemia

25%

Headaches

25%

Dizziness

25%

Diarrhea

13%

Knee Infection

13%

Knee Effusions

13%

Urinary Tract Infection

13%

Influenza

13%

hyponatremia

13%

Tics

13%

Rash

13%

Rhinovirus infection

13%

Urinary Retention

13%

Hyperkalemia

13%

Gastrointestinal upset

100%

80%

60%

40%

20%

Study treatment Arm

Pediatric

Adult

Trial Design

2Treatment groups

Experimental Treatment

Group I: PediatricExperimental Treatment1 Intervention

Pediatric patients treated with dantrolene sodium

Group II: AdultExperimental Treatment1 Intervention

Adult patients treated with dantrolene sodium

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

dantrolene sodium

2017

Completed Phase 2

~30

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Washington University School of MedicineLead Sponsor

1,930 Previous Clinical Trials

2,298,964 Total Patients Enrolled

3 Trials studying Wolfram Syndrome

516 Patients Enrolled for Wolfram Syndrome

National Institutes of Health (NIH)NIH

2,690 Previous Clinical Trials

6,950,987 Total Patients Enrolled

2 Trials studying Wolfram Syndrome

401 Patients Enrolled for Wolfram Syndrome

National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)NIH

2,352 Previous Clinical Trials

4,315,121 Total Patients Enrolled

1 Trials studying Wolfram Syndrome

300 Patients Enrolled for Wolfram Syndrome

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Who meets the eligibility requirements for participating in this research?

"To be eligible for this research, individuals must have wolfram syndrome and be between 5-60 years old. A total of 50 participants are being accepted into the trial."

Answered by AI

Could you share how many individuals are included in this experiment?

"2889 other clinical trials are actively recruiting patients with wolfram syndrome. 5 studies for dantrolene sodium are also admitting participants."

Answered by AI

Are we currently enrolling participants for this research project?

"Unfortunately, this specific study is no longer recruiting patients for participation. Originally appearing on 1/1/2017, this trial was last updated 2/16/2022. However, there are other ongoing trials that may be of interest; 2889 clinical trials involving wolfram syndrome and 5 dantrolene sodium trials are currently searching for participants."

Answered by AI

Will this experiment be testing on elderly individuals?

"The age requirements for this study are between 5 and 60 years old."

Answered by AI

What medical condition is dantrolene sodium commonly used to treat?

"dantrolene sodium is a medication used to help patients who have had cerebrovascular accidents, as well as those suffering from conditions like spinal cord damage, malignant hyperthermia, and spasticity."

Answered by AI

Are there any other ongoing or completed research trials that have used dantrolene sodium?

"Currently, 5 clinical trials are investigating dantrolene sodium. 1 of these research projects is in Phase 3. The majority of the tests for this medication are being conducted in Bethesda, Maryland; however, 6 medical centres total are running these experiments."

Answered by AI

Recent research and studies

JCI InsightJournal

A Phase 1b/2a Clinical Trial of Dantrolene Sodium in Patients with Wolfram Syndrome

Abreu, Damien, Stephen I. Stone, Toni S. Pearson, Robert C. Bucelli, Ashley N. Simpson, Stacy Hurst, Cris M. Brown, et al.. 2021. “A Phase 1b/2a Clinical Trial of Dantrolene Sodium in Patients with Wolfram Syndrome”. JCI Insight. American Society for Clinical Investigation. doi:10.1172/jci.insight.145188.

Current Diabetes ReportsJournal

Wolfram Syndrome: Diagnosis, Management, and Treatment

Urano, Fumihiko. 2016. “Wolfram Syndrome: Diagnosis, Management, and Treatment”. Current Diabetes Reports. Springer Science and Business Media LLC. doi:10.1007/s11892-015-0702-6.

Current Diabetes ReportsJournal

Wolfram Syndrome: Diagnosis, Management, and Treatment

Urano, Fumihiko. 2016. “Wolfram Syndrome: Diagnosis, Management, and Treatment”. Current Diabetes Reports. Springer Science and Business Media LLC. doi:10.1007/s11892-015-0702-6.

clinicaltrials.govStudy