Clinical trial NCT06429982: Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols
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
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Rhabdomyolysis
Lead sponsor: Children's National Research Institute
Sponsor class: OTHER
First posted: 2024-05-28
Enrollment: 50
Sex: ALL
Minimum age (years): 6
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: There will be a 2: 1 treatment ratio of dexamethasone treated group versus placebo.
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Length of Stay
timeFrame: Primarily 5 days - 1 year
measure: Muscle breakdown
timeFrame: Primarily 5 days - 1 year
measure: Renal complications
timeFrame: Primarily 5 days - 1 year
Secondary outcomes:
measure: Quantitative Pain Outcomes
timeFrame: 14 days
Other outcomes:
Locations:
city: Washington
country: United States
facility: Childrens National
state: District of Columbia
Study officials:
References:
citation: 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.
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.
citation: Szugye HS. Pediatric Rhabdomyolysis. Pediatr Rev. 2020 Jun;41(6):265-275. doi: 10.1542/pir.2018-0300.