RhabdomyolysisPhase 1NCT06429982
Dexamethasone Use in Pediatric Rhabdomyolysis Patients in Addition to Standard Protocols
A Phase 1 study testing Dexamethasone in people with Rhabdomyolysis, run by Children's National Research Institute at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 6 months to 25 years
- Conditions the study is for
- Rhabdomyolysis
- What is being tested?
- Dexamethasone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 50 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Oct 2024to 31 Dec 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Natasha Shur, MD contact
United States1 site, 1 recruiting
- Recruiting
Childrens National
Washington D.C., District of Columbia, 20010
Natasha Shur, 202 545-2515, nshur2@childrensnational.org
Seth Berger, 202 476-6156, sberger@childrensnational.org
About this study
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
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.
Read the 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.
- Conditions
- Rhabdomyolysis
- Being tested
- Dexamethasone, Placebo
- Medical subject headings
- Rhabdomyolysis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DexamethasoneDrug
Also called DexPak
Steroid five day treatment
PlaceboDrug
Also called Sugar Pill
Placebo control group
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Dexamethasone |
| Placebo group | Placebo comparator | Placebo for five days with one dose per day placebo oral dosing. Standard care will also be provided. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Length of Stay
Number of days Length of Stay in Each Group
Measured: Primarily 5 days - 1 year
Muscle breakdown
Creatinine Kinase trend comparison between groups
Measured: Primarily 5 days - 1 year
Renal complications
Bun/ Creatinine
Measured: Primarily 5 days - 1 year
Secondary outcomes (1)
Quantitative Pain Outcomes
EHR pain scores
Measured: 14 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 May 2024
- Study start1 Oct 2024
- Primary completion1 Dec 2025 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for RhabdomyolysisStudies of the same condition, open and under way.
- More trials run by Children's National Research InstituteThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- There will be a 2: 1 treatment ratio of dexamethasone treated group versus placebo.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's National Research Institute
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Natasha Shur, Children's National Research Institute
- Investigators
- Natasha Shur, MD, Principal investigator, Children's National Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06429982
- Sponsor's protocol ID
- STUDY00000710
- Status last verified
- Jul 2025
- First submitted
- 31 Aug 2023
- Last updated
- 3 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 27301374Summerlin 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.
PubMed 33300687Szugye HS. Pediatric Rhabdomyolysis. Pediatr Rev. 2020 Jun;41(6):265-275. doi: 10.1542/pir.2018-0300.
PubMed 32482689
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06429982
- Acronym / sponsor ID
- STUDY00000710
- Conditions
- Rhabdomyolysis
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Children's National Research InstituteUniversity, hospital or other
- Study start
- 1 Oct 2024 (actual)
- Primary completion
- 1 Dec 2025 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 28 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- There will be a 2: 1 treatment ratio of dexamethasone treated group versus placebo.
| Arm | Type | Receives |
|---|---|---|
| Dexamethasone group | Active comparator | Drug: DexamethasoneDexamethasone five days with 0.6 mg/ kg dose per day max 16 mg dose. Standard care will also be provided. |
| Placebo group | Placebo comparator | Drug: PlaceboPlacebo for five days with one dose per day placebo oral dosing. Standard care will also be provided. |
Who may qualify
- Ages
- 6 months to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DexamethasoneAlso: DexPakSteroid five day treatment | Drug | Dexamethasone group |
| PlaceboAlso: Sugar PillPlacebo control group | Drug | Placebo group |
Primary outcome
Length of Stay
Time frame: Primarily 5 days - 1 year
Number of days Length of Stay in Each Group
Muscle breakdown
Time frame: Primarily 5 days - 1 year
Creatinine Kinase trend comparison between groups
Renal complications
Time frame: Primarily 5 days - 1 year
Bun/ Creatinine
1 secondary outcome
Quantitative Pain Outcomes
Time frame: 14 days
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2025.
- Background: PubMed 27301374Chavez 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.
- Background: PubMed 33300687Summerlin 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.
- Background: PubMed 32482689Szugye HS. Pediatric Rhabdomyolysis. Pediatr Rev. 2020 Jun;41(6):265-275. doi: 10.1542/pir.2018-0300.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Natasha Shur, MD contact
Natasha Shur, MD principal investigator
Children's National Research Institute
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Dexamethasone in people with Rhabdomyolysis, run by Children's National Research Institute at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 3 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Natasha Shur, MD (202-476-5000, nshur2@childrensnational.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Length of Stay (time frame: Primarily 5 days - 1 year). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06429982, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jul 2025.