Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols

Rhabdomyolysis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06429982
• Other study ID #: STUDY00000710
• Status: Not yet recruiting
• Phase: Phase 1
• Start date: October 1, 2024
• Est. completion date: December 31, 2026

Study information

• Verified date: May 2024
• Source: Children's National Research Institute
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 50
• Est. completion date: December 31, 2026
• Est. primary completion date: December 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 25 Years

Eligibility

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

Related Conditions & MeSH terms

• Rhabdomyolysis

Intervention

Drug:
• Dexamethasone
Steroid five day treatment
• Placebo
Placebo control group

Locations

Country	Name	City	State
United States	Childrens National	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
Children's National Research Institute

Country where clinical trial is conducted

United States, 

References & 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. — View Citation

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. — View Citation

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Length of Stay	Number of days Length of Stay in Each Group	Primarily 5 days - 1 year
Primary	Muscle breakdown	Creatinine Kinase trend comparison between groups	Primarily 5 days - 1 year
Primary	Renal complications	Bun/ Creatinine	Primarily 5 days - 1 year
Secondary	Quantitative Pain Outcomes	EHR pain scores	14 days