EUCTR2015-002530-50-GB进行中（未招募）1 期
A phase Ib, open label study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending oral doses of Rimeporide in patients with Duchenne Muscular Dystrophy - RIM4DMD
EspeRare0 个研究点目标入组 20 人开始时间: 2015年9月7日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 20
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1.Duchenne muscular dystrophy genetically confirmed;
- •2.Males between 6 and 14 years old;
- •3.Able to walk independently at least 75 meters;
- •4.Patients on a stable dose of corticosteroids at least 6 months prior to baseline;
- •5.Willing and able to comply with all protocol requirements and procedures;
- •6.Signed informed consents by the parent(s)/legal guardian(s);
- •7.France only: Affiliated to or a beneficiary of a social security system
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 20
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range 0
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range 0
排除标准
- •1.Patients with significant renal disease or impairment, with Glomerular Filtration Rate estimated using plasma cystatin C level using the Filler formula less than 90ml/min/1.73m2
- •2.Current or history of liver disease or impairment,
- •3.History of any significant medical disorder which may confound the interpretation of either efficacy or safety data e.g. inflammatory, coagulation disease, unstable cardiac or respiratory disease
- •4.Acute illness within 4 weeks of the first administration of study medication which may interfere with study assessments;
- •5.Significant change of dosage and/or dosing regimens for corticosteroids planned for the duration of study medication;
- •6.Use of beta blockers / and ACEI or ARB unless at stable dose for at least 3 months prior to baseline;
- •7.Use of protocol pump inhibitors unless at a stable dose for at least 3 months prior to baseline
- •8.Use of aldosterone antagonists (i.e. spironolactone, eplerenone) within 3 months prior to first administration of study medication;
- •9.Use of anticoagulants, antithrombotics or antiplatelet agents,
- •10.Previous treatment with idebenone or other forms of Coenzyme Q10 within 1 month of the first administration of study medication;
- •11.Previous treatment with investigational drugs within 4 weeks (or 7 half-life if longer than 4 weeks) of the first administration of study medication including placebo;
- •12.A baseline QTc>450msec,or history of risk factors for torsades de pointes (eg, heart failure, hypokalaemia, family history of long QT syndrome);
- •13.LVEF= 45% at screening or within the past 6 months and/or history of acute heart failure;
- •14.Ventilator dependent;
- •15.Known individual hypersensitivity to any of the ingredients/excipients of the study medication;
- •16.For patients willing to undergo MRI/MRS assessments: Patients with specific contraindication to MRI (e.g.: metallic foreign body, claustrophobia, etc.).
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