Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols

Children's National

Status and phase: Not yet enrolling; Phase 1

Conditions: Rhabdomyolysis

Treatments: Drug: Dexamethasone; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT06429982

STUDY00000710

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 estimated patients

Sex: All

Ages: 6 months to 25 years old

Volunteers: No Healthy Volunteers

Inclusion and 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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50 participants in 2 patient groups, including a placebo group

Dexamethasone group

Active Comparator group

Description:

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

Treatment:

Drug: Dexamethasone

Placebo group

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo