## Protocol Section
### Identification Module
**NCT ID:** NCT06429982

**Brief Title:** 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

#### Organization Study ID Info
**ID:** STUDY00000710
#### Organization
**Class:** OTHER
**Full Name:** Children's National Research Institute
### Status Module
#### Completion Date
**Date:** 2026-12-31

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-28

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-23

**Overall Status:** NOT_YET_RECRUITING

#### Primary Completion Date
**Date:** 2025-12-01

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-10-01

**Type:** ESTIMATED

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-28
**Type:** ACTUAL
**Study First Submit Date:** 2023-08-31
**Study First Submit QC Date:** 2024-05-23
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** OTHER

**Name:** Children's National Research Institute

#### Responsible Party

**Investigator Affiliation:** Children's National Research Institute

**Investigator Full Name:** Natasha Shur

**Investigator Title:** MD, Principal Investigator, Professor

**Type:** PRINCIPAL_INVESTIGATOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** True

**Oversight Has DMC:** True

### Description Module

**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.

### Conditions Module

**Conditions:**

- Rhabdomyolysis

### Design Module

#### Design Info

**Allocation:** RANDOMIZED

**Intervention Model:** PARALLEL

**Intervention Model Description:** There will be a 2: 1 treatment ratio of dexamethasone treated group versus placebo.

##### Masking Info

**Masking:** QUADRUPLE

**Masking Description:** This study will be double-blinded. The pharmacy will determine which patients are in the study group.

**Who Masked:**

- PARTICIPANT

- CARE_PROVIDER

- INVESTIGATOR

- OUTCOMES_ASSESSOR

**Primary Purpose:** TREATMENT

#### Enrollment Info

**Count:** 50

**Type:** ESTIMATED

**Phases:**

- PHASE1

**Study Type:** INTERVENTIONAL

### Arms Interventions Module

#### Arm Group 1

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

**Intervention Names:**

- Drug: Dexamethasone

**Label:** Dexamethasone group

**Type:** ACTIVE_COMPARATOR

#### Arm Group 2

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

**Intervention Names:**

- Drug: Placebo

**Label:** Placebo group

**Type:** PLACEBO_COMPARATOR

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Dexamethasone group

**Description:** Steroid five day treatment

**Name:** Dexamethasone

**Other Names:**

- DexPak

**Type:** DRUG

#### Intervention 2

**Arm Group Labels:**

- Placebo group

**Description:** Placebo control group

**Name:** Placebo

**Other Names:**

- Sugar Pill

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** Number of days Length of Stay in Each Group

**Measure:** Length of Stay

**Time Frame:** Primarily 5 days - 1 year

**Description:** Creatinine Kinase trend comparison between groups

**Measure:** Muscle breakdown

**Time Frame:** Primarily 5 days - 1 year

**Description:** Bun/ Creatinine

**Measure:** Renal complications

**Time Frame:** Primarily 5 days - 1 year

#### Secondary Outcomes

**Description:** EHR pain scores

**Measure:** Quantitative Pain Outcomes

**Time Frame:** 14 days

### Eligibility Module

**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

**Maximum Age:** 25 Years

**Minimum Age:** 6 Months

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Washington

**Contacts:**

***Contact 1:***

- **Email:** nshur2@childrensnational.org

- **Name:** Natasha Shur

- **Phone:** 202-545-2515

- **Role:** CONTACT

***Contact 2:***

- **Email:** sberger@childrensnational.org

- **Name:** Seth Berger

- **Phone:** 202 476-6156

- **Role:** CONTACT

**Country:** United States

**Facility:** Childrens National

**State:** District of Columbia

**Zip:** 20010

### IPD Sharing Statement Module

**IPD Sharing:** NO

### References Module

#### 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.

**PMID:** 27301374

**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.

**PMID:** 33300687

**Citation:** Szugye HS. Pediatric Rhabdomyolysis. Pediatr Rev. 2020 Jun;41(6):265-275. doi: 10.1542/pir.2018-0300.

**PMID:** 32482689

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000009135

- Term: Muscular Diseases

- ID: D000009140

- Term: Musculoskeletal Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC05

- Name: Musculoskeletal Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC10

- Name: Nervous System Diseases

### Condition Browse Module - Browse Leaves

- ID: M15035

- Name: Rhabdomyolysis

- Relevance: HIGH

- As Found: Rhabdomyolysis

- ID: M12092

- Name: Muscular Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M12097

- Name: Musculoskeletal Diseases

- Relevance: LOW

- As Found: Unknown

### Condition Browse Module - Meshes

- ID: D000012206

- Term: Rhabdomyolysis

### Intervention Browse Module - Ancestors

- ID: D000000893

- Term: Anti-Inflammatory Agents

- ID: D000000932

- Term: Antiemetics

- ID: D000001337

- Term: Autonomic Agents

- ID: D000018373

- Term: Peripheral Nervous System Agents

- ID: D000045505

- Term: Physiological Effects of Drugs

- ID: D000005765

- Term: Gastrointestinal Agents

- ID: D000005938

- Term: Glucocorticoids

- ID: D000006728

- Term: Hormones

- ID: D000006730

- Term: Hormones, Hormone Substitutes, and Hormone Antagonists

- ID: D000018931

- Term: Antineoplastic Agents, Hormonal

- ID: D000000970

- Term: Antineoplastic Agents

### Intervention Browse Module - Browse Branches

- Abbrev: Infl

- Name: Anti-Inflammatory Agents

- Abbrev: ANeo

- Name: Antineoplastic Agents

- Abbrev: AnEm

- Name: Antiemetics

- Abbrev: Gast

- Name: Gastrointestinal Agents

- Abbrev: All

- Name: All Drugs and Chemicals

### Intervention Browse Module - Browse Leaves

- ID: M7102

- Name: Dexamethasone

- Relevance: HIGH

- As Found: Children

- ID: M235549

- Name: Dexamethasone acetate

- Relevance: LOW

- As Found: Unknown

- ID: M199152

- Name: BB 1101

- Relevance: LOW

- As Found: Unknown

- ID: M4217

- Name: Anti-Inflammatory Agents

- Relevance: LOW

- As Found: Unknown

- ID: M4251

- Name: Antiemetics

- Relevance: LOW

- As Found: Unknown

- ID: M8881

- Name: Gastrointestinal Agents

- Relevance: LOW

- As Found: Unknown

- ID: M9047

- Name: Glucocorticoids

- Relevance: LOW

- As Found: Unknown

- ID: M9789

- Name: Hormones

- Relevance: LOW

- As Found: Unknown

- ID: M9788

- Name: Hormone Antagonists

- Relevance: LOW

- As Found: Unknown

- ID: M20966

- Name: Antineoplastic Agents, Hormonal

- Relevance: LOW

- As Found: Unknown

### Intervention Browse Module - Meshes

- ID: D000003907

- Term: Dexamethasone

### Misc Info Module

- Version Holder: 2024-05-31
