A Clinical Trial to Assess Whether Dexamethasone Addition to Standard Protocols for Non-Traumatic Rhabdomyolysis of Unknown or Genetic Etiologies Improves Patient Outcomes
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Length of Stay
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
This study will be double-blinded. The pharmacy will determine which patients are in the study group.
入排标准
- 年龄范围
- 6 Months 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •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
研究组 & 干预措施
Dexamethasone group
Dexamethasone five days with 0.6 mg/ kg dose per day max 16 mg dose. Standard care will also be provided.
干预措施: Dexamethasone (Drug)
Placebo group
Placebo for five days with one dose per day placebo oral dosing. Standard care will also be provided.
干预措施: Placebo (Drug)
结局指标
主要结局
Length of Stay
时间窗: Primarily 5 days - 1 year
Number of days Length of Stay in Each Group
Muscle breakdown
时间窗: Primarily 5 days - 1 year
Creatinine Kinase trend comparison between groups
Renal complications
时间窗: Primarily 5 days - 1 year
Bun/ Creatinine
次要结局
- Quantitative Pain Outcomes(14 days)
研究者
Natasha Shur
MD, Principal Investigator, Professor
Children's National Research Institute