EUCTR2015-002530-50-FR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.
EspeRare0 个研究点目标入组 20 人开始时间: 2015年9月11日最近更新:
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
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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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