Adding dexamethasone to standard care for children and young adults with rhabdomyolysis
A Clinical Trial to Assess Whether Dexamethasone Addition to Standard Protocols for Non-Traumatic Rhabdomyolysis of Unknown or Genetic Etiologies Improves Patient Outcomes
This study will test whether giving dexamethasone alongside usual care helps children and young adults (6 months–25 years) with rhabdomyolysis have less pain, shorter hospital stays, and fewer kidney problems.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 6 Months to 25 Years |
| Sex | All |
| Sponsor | Children's National Research Institute Academic / other |
| Locations | 1 site (Washington D.C., District of Columbia) |
| Trial ID | NCT06429982 on ClinicalTrials.gov |
What this trial studies
This is a single-center, randomized, double-blind Phase 1 trial enrolling up to 50 patients aged 6 months to 25 years with rhabdomyolysis (CK >5000) not due to trauma. Participants are randomized 2:1 to receive oral dexamethasone or placebo once daily for five days in addition to standard care. Pain surveys and chart reviews will be used to track symptoms, length of stay, and kidney-related outcomes during and after the five-day treatment period. The study is planned to run two years at a single pediatric center.
Who should consider this trial
Good fit: Ideal candidates are patients aged 6 months to 25 years diagnosed with rhabdomyolysis (CK >5000) not caused by trauma, able to take oral medications, and not already on systemic steroids.
Not a fit: Patients already taking systemic steroids, unable to take oral medications, pregnant women, or those with relevant antifungal allergies are unlikely to be eligible or to benefit from this protocol.
Why it matters
Potential benefit: If successful, adding dexamethasone could reduce pain, shorten hospital stays, and lower the risk of kidney complications for pediatric and young adult patients with rhabdomyolysis.
How similar studies have performed: Steroids have been used off-label for rhabdomyolysis, but prospective randomized trials testing dexamethasone in this setting are lacking.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria 1. Diagnosis of rhabdomyolysis defined as creatine kinase\> 5000 with trauma excluded 2. Ability of parents/patients to understand and the willingness to sign a written informed consent document. 3. Patients ages 12 and older will sign written assent Exclusion Criteria: * Already taking systemic steroids. * Inability to comply with study instructions. * Uncontrolled intercurrent illness or psychiatric illness/social situations that would limit compliance with study requirements. * Pregnant women. o A urine pregnancy test will be performed for women of child-bearing potential. * Below gestational age of 40 weeks * Allergy to fluconazole, clotrimazole or nystatin. * Cannot tolerate PO medications
Where this trial is running
Washington D.C., District of Columbia
- Childrens National — Washington D.C., District of Columbia, United States (Recruiting)
Study contacts
- Principal investigator: Natasha Shur, MD — Children's National Research Institute
- Study coordinator: Natasha Shur, MD
- Email: nshur2@childrensnational.org
- Phone: 202-476-5000
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.