Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols

A Clinical Trial to Assess Whether Dexamethasone Addition to Standard Protocols for Non-Traumatic Rhabdomyolysis of Unknown or Genetic Etiologies Improves Patient Outcomes

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.

Study Overview

• Status: Recruiting
• Conditions: Rhabdomyolysis
• Intervention / Treatment: Drug: Dexamethasone; Drug: Placebo

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 Type: Interventional
• Enrollment (Estimated): 50
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Natasha Shur, MD; Phone Number: 202-476-5000; Email: nshur2@childrensnational.org
• Study Contact Backup: Name: Natasha Shur, MD; Email: nshur2@childrensnational.org

Study Locations

• United States
  • District of Columbia
    • Washington DC, District of Columbia, United States, 20010
      • Recruiting
      • Childrens National
      • Contact: Seth Berger; Phone Number: 202 476-6156; Email: sberger@childrensnational.org
      • Contact: Natasha Shur; Phone Number: 202 545-2515; Email: nshur2@childrensnational.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Dexamethasone group; Dexamethasone five days with 0.6 mg/ kg dose per day max 16 mg dose. Standard care will also be provided.	Drug: Dexamethasone; Steroid five day treatment; Other Names: DexPak
Placebo Comparator: Placebo group; Placebo for five days with one dose per day placebo oral dosing. Standard care will also be provided.	Drug: Placebo; Placebo control group; Other Names: Sugar Pill

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Quantitative Pain Outcomes	EHR pain scores	14 days

Collaborators and Investigators

• Sponsor: Children's National Research Institute
• Investigators: Principal Investigator: Natasha Shur, MD, Children's National Research Institute

Publications and helpful links

General Publications

• Chavez LO, Leon M, Einav S, Varon J. Beyond muscle destruction: a systematic review of rhabdomyolysis for clinical practice. Crit Care. 2016 Jun 15;20(1):135. doi: 10.1186/s13054-016-1314-5.
• Summerlin ML, Regier DS, Fraser JL, Chapman KA, Kafashzadeh D, Billington C Jr, Kisling M, Grochowsky A, Ah Mew N, Shur N. Use of dexamethasone in idiopathic, acute pediatric rhabdomyolysis. Am J Med Genet A. 2021 Feb;185(2):500-507. doi: 10.1002/ajmg.a.62000. Epub 2020 Dec 10.
• Szugye HS. Pediatric Rhabdomyolysis. Pediatr Rev. 2020 Jun;41(6):265-275. doi: 10.1542/pir.2018-0300.

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2024
• Primary Completion (Estimated): December 1, 2025
• Study Completion (Estimated): December 31, 2026

Study Registration Dates

• First Submitted: August 31, 2023
• First Submitted That Met QC Criteria: May 23, 2024
• First Posted (Actual): May 28, 2024

Study Record Updates

• Last Update Posted (Actual): July 3, 2025
• Last Update Submitted That Met QC Criteria: July 1, 2025
• Last Verified: July 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Muscular Diseases; Rhabdomyolysis; Antineoplastic Agents; Physiological Effects of Drugs; Anti-Inflammatory Agents; Antiemetics; Autonomic Agents; Peripheral Nervous System Agents; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Dexamethasone
• Other Study ID Numbers: STUDY00000710

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No