Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols

NCT06429982 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: STUDY00000710
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

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.

Conditions

Rhabdomyolysis

Outcome Measures

Primary Outcomes (3)

• 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 Outcomes (1)

• Quantitative Pain Outcomes

  14 days

Study Arms (2)

Dexamethasone group

ACTIVE COMPARATOR

Dexamethasone five days with 0.6 mg/ kg dose per day max 16 mg dose. Standard care will also be provided.

Drug: Dexamethasone

Placebo group

PLACEBO COMPARATOR

Placebo for five days with one dose per day placebo oral dosing. Standard care will also be provided.

Drug: Placebo

Interventions

Dexamethasone DRUG

Steroid five day treatment

Also known as: DexPak

Dexamethasone group

Placebo DRUG

Placebo control group

Also known as: Sugar Pill

Placebo group

Eligibility Criteria

• Age: 6 Months - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Diagnosis of rhabdomyolysis defined as creatine kinase\> 5000 with trauma excluded
• Ability of parents/patients to understand and the willingness to sign a written informed consent document.
• Patients ages 12 and older will sign written assent

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's National Research Institute: lead

Study Sites (1)

Childrens National

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Related Publications (3)

• 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.

  PMID: 27301374 BACKGROUND

• 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.

  PMID: 33300687 BACKGROUND

• Szugye HS. Pediatric Rhabdomyolysis. Pediatr Rev. 2020 Jun;41(6):265-275. doi: 10.1542/pir.2018-0300.

  PMID: 32482689 BACKGROUND

MeSH Terms

Conditions

Rhabdomyolysis

Interventions

Dexamethasone; Sugars

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Steroids, Fluorinated; Carbohydrates

Study Officials

• Natasha Shur, MD

  Children's National Research Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Natasha Shur, MD

CONTACT

202-476-5000; nshur2@childrensnational.org

Natasha Shur, MD

CONTACT

nshur2@childrensnational.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: This study will be double-blinded. The pharmacy will determine which patients are in the study group.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, Principal Investigator, Professor

Model Details: There will be a 2: 1 treatment ratio of dexamethasone treated group versus placebo.

Study Record Dates

• First Submitted: August 31, 2023
• First Posted: May 28, 2024
• Study Start: October 1, 2024
• Primary Completion: December 1, 2025
• Study Completion (Estimated): December 31, 2026
• Last Updated: July 3, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)