A Multi-Center, Double-Blind, Pilot Study of Minocycline in Huntington's Disease
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 114
- 试验地点
- 12
- 主要终点
- Change From Baseline to Month 18 in the Total Functional Capacity (TFC) Scale [LOCF Imputation Method]
研究概览
简要总结
This study is being conducted to assess the impact of minocycline on the progression of symptoms of HD. The study will also assess whether it is reasonable to continue with further study of minocycline in HD. We will measure the effect of minocycline on HD by measuring the change in Huntington's disease symptoms.
详细描述
The DOMINO study is a randomized, double-blind, multi-center, futility study of minocycline in patients with HD. Subjects will be randomized (3:1) to one of the two study arms: (1) the group that receives active minocycline (100 mg po b.i.d.), and (2) the group that receives placebo. Subjects will be enrolled over an approximate six-month period and remain on blinded study drug for 18 months. The primary analysis will involve a comparison of the change over time in TFC between the minocycline group and a fixed value determined from historical control data. A placebo group will also be included to facilitate blinding and to permit a descriptive assessment of the validity of the assumed change over time in historical controls.
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
- Double (Participant, Investigator)
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
- •Age 18 years or older
- •Clinical features of HD and a confirmatory family history of HD; and/or genetically confirmed HD
- •Independently walking and fully self-sufficient in activities of daily living (eating, dressing, bathing)
- •Able to take medication (capsules) by mouth
排除标准
- •History of known hypersensitivity or intolerability to minocycline or known allergy to any tetracycline
- •History of vestibular disease
- •Subjects with underlying hematologic, hepatic or renal disease
- •History of systemic lupus erythematosus (SLE)
研究组 & 干预措施
Minocycline
Minocycline (3:1 randomization) 100 mg capsules taken by mouth twice daily, 200 mg per day total for 18 months treatment duration.
干预措施: minocycline (Drug)
Matching placebo
Sugar pill manufactured to mimic minocycline, 1 capsule taken by mouth twice daily for 18 months treatment duration.
干预措施: Matching placebo (Drug)
结局指标
主要结局
Change From Baseline to Month 18 in the Total Functional Capacity (TFC) Scale [LOCF Imputation Method]
时间窗: Baseline to 18 months
Establish preliminary estimate of minocycline's impact on progression of HD (measured by the change in Total Functional Capacity (TFC) score of Unified Huntington's Disease Rating Scale \[UHDRS\] between baseline \& Month 18), and to assess futility of further study of minocycline. TFC consists of five ordinally scaled items assessing a person's capacity with: 1. occupation 2. financial affairs 3. domestic responsibilities 4. activities of daily living and 5. independent living. Total score ranges from zero (worst) to 13 (best).
次要结局
- Change From Baseline to Month 18 in the Total Functional Capacity (TFC) Scale [Regression Based Multiple Imputation Method](Baseline to 18 months)
研究者
Merit Cudkowicz
Principal Investigator
Huntington Study Group