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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
|
| Placebo group | Placebo Comparator | Placebo for five days with one dose per day placebo oral dosing. Standard care will also be provided. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Dexamethasone | Drug | Steroid five day treatment |
|
|
| Measure | Description | Time Frame |
|---|---|---|
| Length of Stay | Number of days Length of Stay in Each Group | Primarily 5 days - 1 year |
| Muscle breakdown | Creatinine Kinase trend comparison between groups | Primarily 5 days - 1 year |
| Renal complications | Bun/ Creatinine | Primarily 5 days - 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| Quantitative Pain Outcomes | EHR pain scores | 14 days |
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Inclusion Criteria
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
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Natasha Shur, MD | Contact | 202-476-5000 | nshur2@childrensnational.org | |
| Natasha Shur, MD | Contact | nshur2@childrensnational.org |
| Name | Affiliation | Role |
|---|---|---|
| Natasha Shur, MD | Children's National Research Institute | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Childrens National | Recruiting | Washington D.C. | District of Columbia | 20010 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 27301374 | Background | 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. | |
| 33300687 | Background | 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. |
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| ID | Term |
|---|---|
| D012206 | Rhabdomyolysis |
| ID | Term |
|---|---|
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| D003907 | Dexamethasone |
| D000073893 | Sugars |
| ID | Term |
|---|---|
| D011246 | Pregnadienetriols |
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
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There will be a 2: 1 treatment ratio of dexamethasone treated group versus placebo.
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This study will be double-blinded. The pharmacy will determine which patients are in the study group.
| Placebo | Drug | Placebo control group |
|
|
| 32482689 | Background | Szugye HS. Pediatric Rhabdomyolysis. Pediatr Rev. 2020 Jun;41(6):265-275. doi: 10.1542/pir.2018-0300. |
| D000072473 |
| Fused-Ring Compounds |
| D011083 | Polycyclic Compounds |
| D013259 | Steroids, Fluorinated |
| D002241 | Carbohydrates |