Dexamethasone for Rhabdomyolysis

Recruiting at 2 trial locations

Overseen By Natasha Shur, MD

Age: < 65

Sex: Any

Trial Phase: Phase 1

Sponsor: Children's National Research Institute

Must not be taking: Systemic steroids

Disqualifiers: Pregnancy, Uncontrolled illness, Allergy, others

Approved in 4 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 5 days

50 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether adding dexamethasone, a type of steroid, to standard care can help people with rhabdomyolysis recover faster and reduce kidney problems. Rhabdomyolysis occurs when muscle tissue breaks down, releasing creatine kinase into the blood, which can cause pain and kidney issues. The trial includes two groups: one receiving dexamethasone and the other a placebo, both alongside standard care. Suitable candidates for this trial are individuals aged 12 and up with rhabdomyolysis and creatine kinase levels above 5000, not caused by trauma. As a Phase 1 trial, this research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are already taking systemic steroids.

Is there any evidence suggesting that dexamethasone is likely to be safe for humans?

Research has shown that dexamethasone has been tested in animals for conditions like Crush syndrome, which resemble rhabdomyolysis. These studies found better outcomes without major safety issues. In humans, dexamethasone has been used safely for other conditions, but it is not yet a standard treatment for rhabdomyolysis. Some studies on children with rhabdomyolysis of unknown cause have shown encouraging results with few side effects. Since this trial is in an early stage, it primarily focuses on safety. The FDA has already approved dexamethasone for other uses, which provides some reassurance about its safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for rhabdomyolysis focus on hydration and correcting electrolyte imbalances. However, Dexamethasone is unique because it leverages its anti-inflammatory properties to potentially reduce muscle damage more directly. This steroid is used to help control inflammation, which could be crucial in managing the muscle breakdown that characterizes rhabdomyolysis. Researchers are excited because this approach might offer a faster, more targeted way to address the condition, possibly improving recovery times and outcomes compared to standard care.

What evidence suggests that dexamethasone might be an effective treatment for rhabdomyolysis?

Research shows that dexamethasone, a type of steroid, might help treat rhabdomyolysis, a condition where muscle tissue breaks down and can damage the kidneys. In this trial, participants in the dexamethasone group will receive dexamethasone for five days. Studies have found that dexamethasone can shorten the active phase of rhabdomyolysis and reduce the time patients need to stay in intensive care. This could lead to faster recovery and less time in the hospital. Although not yet a standard treatment, early results are promising and suggest that dexamethasone could improve recovery for people with rhabdomyolysis. ^{3 6 7 8 9}

Who Is on the Research Team?

Natasha Shur, MD

Principal Investigator

Children's National Research Institute

Are You a Good Fit for This Trial?

This trial is for children with rhabdomyolysis, a muscle breakdown condition. It's open to those who meet certain health criteria but details on specific inclusions or exclusions aren't provided.

Inclusion Criteria

Ability of parents/patients to understand and the willingness to sign a written informed consent document

I am 12 or older and willing to sign a consent form.

I have been diagnosed with rhabdomyolysis with a creatine kinase level over 5000, without any injury.

Exclusion Criteria

Allergy to fluconazole, clotrimazole or nystatin

Inability to comply with study instructions

Uncontrolled intercurrent illness or psychiatric illness/social situations that would limit compliance with study requirements

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive dexamethasone or placebo for 5 days in addition to standard care

5 days

Daily visits for oral dosing

Follow-up

Participants are monitored for safety and effectiveness after treatment, including pain assessment and chart review

14 days

Surveys before and after treatment

Extended Follow-up

Participants are monitored for long-term outcomes such as length of hospital stay and renal complications

Up to 1 year

What Are the Treatments Tested in This Trial?

Interventions

• Dexamethasone

Trial Overview The study tests if adding dexamethasone (a steroid) to standard treatment helps reduce pain, shorten hospital stays, and prevent kidney problems better than the usual care plus a placebo.

How Is the Trial Designed?

Group I: Dexamethasone group Active Control 1 Intervention

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

Group II: Placebo group Placebo Group 1 Intervention

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

Who Is Running the Clinical Trial?

Children's National Research Institute

Lead Sponsor

Trials

227

Recruited

258,000+

Published Research Related to This Trial

Deflazacort treatment in boys with Duchenne muscular dystrophy (DMD) significantly delayed the loss of walking ability and improved pulmonary function compared to untreated boys, with treated boys maintaining better functional vital capacity at 15 years (88% vs. 39%).

While deflazacort was associated with the development of asymptomatic cataracts in 10 out of 30 boys, other side effects like hypertension and infections were not more common than in untreated boys, indicating a relatively favorable safety profile.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Deflazacort treatment of Duchenne muscular dystrophy. Biggar, WD., Gingras, M., Fehlings, DL., et al.[2014]

In a double-blind trial involving 28 patients with Duchenne muscular dystrophy, Deflazacort (DF) showed statistically significant improvements in muscle strength and function compared to a placebo, indicating its efficacy as a treatment option.

While some mild side effects were observed, such as increased appetite and a cushingoid appearance in a few patients, no one had to stop taking the medication, suggesting that DF may have a better safety profile than traditional steroids like prednisone.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Steroids in Duchenne muscular dystrophy--deflazacort trial. Mesa, LE., Dubrovsky, AL., Corderi, J., et al.[2019]

Deflazacort, approved for Duchenne muscular dystrophy (DMD) in 2017, shows similar or slower rates of functional decline compared to prednisone/prednisolone, based on evidence from various clinical studies.

While deflazacort has a better safety profile regarding weight gain and behavioral side effects, it may have more negative impacts on bone health, growth, and cataract development compared to prednisone/prednisolone.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy. Biggar, WD., Skalsky, A., McDonald, CM.[2022]

Long-term corticosteroid treatment (over 3 years) in patients with Duchenne muscular dystrophy provides significant benefits, including prolonging ambulation by 2 to 5 years and improving overall neuromuscular function without major side effects.

Corticosteroids also reduce the need for spinal stabilization surgery, enhance cardiopulmonary function, delay the requirement for noninvasive ventilation, and improve both survival rates and quality of life for these patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Change in natural history of Duchenne muscular dystrophy with long-term corticosteroid treatment: implications for management. Moxley, RT., Pandya, S., Ciafaloni, E., et al.[2022]

Citations

1. cureswithinreach.org cureswithinreach.org/2023/06/15/using-steroids-improve-rhabdomyolysis-outcomes-pediatric-patients/

Using Steroids to Improve Rhabdomyolysis Outcomes in ... The research team found that off-label treatment with a generic, readily available steroid, dexamethasone, may be effective in potentially decreasing ...

2. researchgate.net researchgate.net/publication/369386413_Use_of_dexamethasone_in_acute_rhabdomyolysis_in_LPIN1_deficiency

Use of dexamethasone in acute rhabdomyolysis in LPIN1 ... Results: Dexamethasone administration shortened the course of active rhabdomyolysis, intensive care admission and rehabilitation. It also ...

3. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/36941958/

Use of dexamethasone in acute rhabdomyolysis in LPIN1 ... Results: Dexamethasone administration shortened the course of active rhabdomyolysis, intensive care admission and rehabilitation. It also ...

4. onlinelibrary.wiley.com onlinelibrary.wiley.com/doi/full/10.1002/jimd.12592

Systemic corticosteroids for the treatment of acute episodes ... Promising results with dexamethasone have been already described ... Use of dexamethasone in idiopathic, acute pediatric rhabdomyolysis.

5. sciencedirect.com sciencedirect.com/science/article/abs/pii/S0025619611652427

Corticosteroids in the Treatment of Alcohol-Induced ... A few case reports show effectiveness of corticosteroids in the treatment of rhabdomyolysis, but this is not considered standard practice [34,81–83].

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11432986/

Risk of Rhabdomyolysis Associated with Dexmedetomidine ... Our findings suggest dexmedetomidine poses a lower rhabdomyolysis risk than propofol, supporting its safe use for sedation in high-risk patients.

7. withpower.com withpower.com/trial/phase-1-rhabdomyolysis-9-2024-da933

Dexamethasone for Rhabdomyolysis Research on rats with Crush syndrome, which involves muscle damage similar to rhabdomyolysis, shows that dexamethasone improved clinical outcomes and had ...

8. researchgate.net researchgate.net/publication/347590053_Use_of_dexamethasone_in_idiopathic_acute_pediatric_rhabdomyolysis

Use of dexamethasone in idiopathic, acute pediatric ... Current rhabdomyolysis treatment guidelines vary based on the etiology and diagnosis, yet many cases evade conclusive diagnosis.

9. trialx.com trialx.com/clinical-trials/listings/316189/dexamethasone-use-in-pediatric-rhabdomyolysis-patients-in-addition-to-standard-protocols/

Dexamethasone Use in Pediatric Rhabdomyolysis Patients ... This is a single center, 2-year, blinded prospective randomized study for those diagnosed with rhabdomyolysis age six months-25 years using ...