MD1003 in Adrenomyeloneuropathy : a Randomized Double Blind Placebo Controlled Study
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 67
- 试验地点
- 4
- 主要终点
- Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
研究概览
简要总结
The primary objective of the trial is to demonstrate the superiority of biotin at 300 mg/day over placebo in the clinical improvement (walking tests) of patients with adrenomyeloneuropathy
详细描述
AMN and progressive multiple sclerosis share some similarities including progressive spastic paraparesis and secondary energy failure leading to progressive axonal degeneration. Therefore, it was hypothesized that high doses of biotin might be efficient in patients with AMN.
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)
入排标准
- 年龄范围
- 18 Years 至 60 Years（Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •ABCD1 gene mutation identified
- •Elevated plasma VLCFA
- •Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk
- •EDSS score ≥ 3.5 and ≤ 6.5
- •Normal brain MRI or brain MRI showing :
- •abnormalities that can be observed in AMN patients without cerebral demyelination with a maximum Loes score of 4
- •and/or stable (≥6 months) cerebral demyelination without gadolinium enhancement with a Loes score ≤
- •Appropriate steroid replacement if adrenal insufficiency is present
- •Likely to be able to participate in all scheduled evaluation visits and complete all required study procedures
- •Signed and dated written informed consent to participate in the study in accordance with local regulations
- •Affiliated to a Health Insurance
排除标准
- •Brain MRI abnormalities with a Loes score > 12 or with gadolinium enhancement
- •Any progressive neurological disease other than AMN
- •Impossibility to perform the walk tests and the TUG test
- •Patients with uncontrolled hepatic disorder, renal or cardiovascular disease, or any progressive malignancy
- •Any new medication for AMN including Fampridine initiated less than 1 month prior to inclusion
- •Contra-indications for MRI procedure such as subjects with paramagnetic materials in the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants.
- •Inclusion in another therapeutic clinical trial for ALD
- •Not easily contactable by the investigator in case of emergency or not capable to call the investigator
研究组 & 干预措施
MD1003
MD1003 100mg capsules, 1 capsule tid for 24 months
干预措施: MD1003 100 mg capsule (Drug)
Placebo
Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months
干预措施: MD1003 100 mg capsule (Drug)
Placebo
Placebo capsule, 1 capsule tid for 12 months, then switch to MD1003 100mg capsule, 1 capsule tid for 12 months
干预措施: Placebo (Drug)
结局指标
主要结局
Mean change of 2 minutes walking test (2MWT) between Months 12 and baseline
时间窗: Baseline and 12 Months
次要结局
- Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20%(Baseline, 9 months, 12 months)
- Timed up and Go test (TUG)(12 Months)
- Euroqol EQ-5D questionnaire(12 months)
- Mean Change in TW25 (time to walk 25 feet)(Baseline and 12 months)
- Qualiveen Questionnaire(12 Months)
- Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20%(Baseline, 9 months, 12 months)