Safety and Tolerability of Phenylbutyrate in Inclusion Body Myositis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events
研究概览
简要总结
This is a pilot study (phase 1 clinical trial) to evaluate the safety and tolerability of phenylbutyrate in IBM. In this open label study, 10 patients with sporadic inclusion body myositis will be treated with phenylbutyrate (3 gm twice daily) for 3 months. There will be a run-in period, during which certain biomarkers will be measured at baseline and at the end of the run-in period in addition to final measurement at the end of the treatment period.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Fulfill ENMC 2011 diagnostic criteria for IBM
- •Age > 18 years
- •Women must be post-menopausal (no menses in >12 months) or status post hysterectomy
- •Able to give informed consent
排除标准
- •Presence of any one of the following medical conditions: chronic infection; chronic renal insufficiency; cancer other than skin cancer less than five years prior; multiple sclerosis or prior episode of central nervous system demyelination; or other chronic serious medical illnesses
- •Presence of any of the following on routine blood screening: WBC<3000; Platelets < 100,000; hematocrit < 30%; BUN > 30 mg %; creatine > 1.5 mg%; liver disease with serum albumin < 3 G/DL
- •Women who are pregnant or lactating
- •History of non-compliance with other therapies
- •Coexistence of other muscular disease
- •Drug or alcohol abuse within past three months
- •Known bleeding disorder
- •Known liver disease
- •Known congestive heart failure
- •Known hypernatremia
- •Inability to give informed consent
研究组 & 干预措施
Phenylbutyrate
Open-label phenylbutyrate
干预措施: Phenylbutyrate Oral Tablet (Drug)
结局指标
主要结局
Safety and Tolerability of Phenylbutyrate Measured by Incidence of Adverse Events
时间窗: Month 3 - Month 6
Since this is a Phase I pilot trial, the primary outcome will be testing the safety and tolerability of phenylbutyrate in patients with Inclusion Body Myositis. This will be measured by the incidence of adverse events reported in subjects throughout the treatment period of the trial.
次要结局
- Inclusion Body Myositis Functional Rating Scale (IBMFRS)(Month 6)