Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols

Phase 1

Not yet recruiting

• Conditions: Rhabdomyolysis
• Interventions: Drug: Placebo; Drug: Dexamethasone

• Registration Number: NCT06429982
• Lead Sponsor: Children's National Research Institute

Brief Summary

There is a significant unmet need for optimized treatment in rhabdomyolysis. There are few prospective interventional studies on treatment for rhabdomyolysis, a condition which affects diverse and underrepresented populations at a higher rate. While steroids are often used off-label, a systematic study has not yet been initiated, and steroids have not been yet considered in as a consideration to standard care guidelines.

The hypothesis is that patients who receive dexamethasone in addition to standard care versus placebo and standard care will have improvement in pain, length of hospital stay, and decrease in kidney complications.

Detailed Description

Study Objective:

This is a single center, 2-year, blinded prospective randomized study for those diagnosed with rhabdomyolysis age six months-25 years using dexamethasone versus placebo treatment in addition to standard care in up to 50 patients.

Study Design:

Patients will be enrolled with a 2:1 ratio of treatment of dexamethasone for 5 days versus placebo treatment for 5 days in addition to receiving standard care. The treatment period for each subject will be five days treatment with oral treatment once per day with study drug (dexamethasone versus placebo). All patients will also receive standard care. Patients and their parent/ caregiver will have the option to complete surveys before and after treatment in order to assess pain level and treatment improvement. Chart review will be performed on all patients throughout and after the five-day study treatment period. There will be no additional interventions.

Study Timeline

• Study First Submitted: 2023-08-31
• Status Verified: 2024-05
• Study First Submitted QC: 2024-05-23
• Last Update Submitted: 2024-05-23
• Study First Posted: 2024-05-28
• Last Update Posted: 2024-05-28
• Study Start: 2024-10-01
• Primary Completion: 2025-12-01
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Not provided

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo group	Placebo	Placebo for five days with one dose per day placebo oral dosing. Standard care will also be provided.
Dexamethasone group	Dexamethasone	Dexamethasone five days with 0.6 mg/ kg dose per day max 16 mg dose. Standard care will also be provided.

Primary Outcome Measures

Name	Time	Method
Length of Stay	Primarily 5 days - 1 year	Number of days Length of Stay in Each Group
Muscle breakdown	Primarily 5 days - 1 year	Creatinine Kinase trend comparison between groups
Renal complications	Primarily 5 days - 1 year	Bun/ Creatinine

Secondary Outcome Measures

Name	Time	Method
Quantitative Pain Outcomes	14 days	EHR pain scores

Trial Locations

Locations (1)

Childrens National; 🇺🇸 Washington, District of Columbia, United States