NCT03231878已完成2 期
A Randomized, Double-blind, Placebo-controlled, Multinational, Multicenter Study With Open-label Treatment Extension to Assess the Effect of MIN-102 (IMP) on the Progression of Adrenomyeloneuropathy in Male Patients With X-linked Adrenoleukodystrophy
Minoryx Therapeutics, S.L.10 个研究点 分布在 8 个国家目标入组 105 人开始时间: 2017年12月8日最近更新:
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 105
- 试验地点
- 10
- 主要终点
- To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.
研究概览
简要总结
This is a Phase II/III, randomized, double-blind, placebo-controlled, multicenter, two parallel-group study in male patients with the AMN phenotype of X-linked adrenoleukodystrophy (X-ALD) to assess the efficacy and safety of MIN-102 treatment. Study sites will consist of specialist referral centers experienced in the management of adrenoleukodystrophy (ALD).
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Male and between 18-65 years of age.
- •Diagnosed with X-linked adrenoleukodystrophy (X-ALD) based on elevated VLCFA and genetic testing.
- •Clinical evidence of spinal cord involvement.
排除标准
- •Any other chronic neurological disease with signs of spastic paraplegia, such as hereditary spastic paraplegia, multiple sclerosis, etc.
- •Presence of inflammatory (Gd-enhancing) MRI lesions or any abnormality other than those mentioned in the inclusion criteria.
- •Known type 1 or type 2 diabetes.
- •Known intolerance to pioglitazone or any other thiazolidinedione.
- •Taking or have taken honokiol, pioglitazone or other thiazolidinediones within the 6 months prior to screening.
- •Previous bone marrow transplantation.
- •Previous or current history of cancer (other than treated basal cell carcinoma).
- •Previous or current history of congestive heart failure.
研究组 & 干预措施
Active
Active Comparator
干预措施: MIN-102 (Drug)
Placebo
Placebo Comparator
干预措施: Placebos (Drug)
结局指标
主要结局
To evaluate the efficacy of MIN-102 on the progression of adrenomyeloneuropathy (AMN) in male patients as determined by a motor function test.
时间窗: in 96 weeks
次要结局
- To evaluate the efficacy of MIN-102 in terms of patient reported outcomes.(in 96 weeks)
- SSPROM (Severity Score System for Progressive Myelopathy )(in 96 weeks)
- Quality of life scales (Euroqol)(in 96 weeks)
- Incidence of cerebral inflammatory lesions(in 96 weeks)
- EDSS (Expanded Disability Status Scale )(in 96 weeks)
研究者
研究点 (10)
Loading locations...
相似试验
已完成
2 期
A Study to Evaluate the Efficacy and Safety of TNX-1900 in Patients With Chronic MigraineChronic MigraineChronic Migraine, HeadacheChronic Migraine Without AuraAura MigraineNCT05679908Tonix Pharmaceuticals, Inc.88
已完成
2 期
A Study to Evaluate the Efficacy, Safety and Tolerability of MIV-711 in Osteoarthritis PatientsOsteoarthritis, KneeNCT02705625Medivir244
终止
2 期
A Study of RO7123520 to Evaluate the Safety and Efficacy in Participants With Moderately To Severely Active Rheumatoid Arthritis (RA) Who Are Inadequately Responding to Anti-Tumor Necrosis Factor (TNF)-Alpha TherapyRheumatoid ArthritisNCT03001219Hoffmann-La Roche109
已完成
2 期
A Study to Evaluate the Safety and Efficacy of Rituximab in Adults With Primary Progressive Multiple SclerosisMultiple SclerosisNCT00087529Genentech, Inc.439
招募中
2 期
A Study of CM310 in Subjects With Moderate to Severe AsthmaModerate to Severe AsthmaNCT05761028CSPC ZhongQi Pharmaceutical Technology Co., Ltd.600