An open-label, un-controlled, single-centre trial investigating the efficacy and safety of CRD007 tablets administered twice daily for 12 weeks in children with Duchenne Muscular Dystrophy (DMD) or Becker Muscular Dystrophy (BMD) or children being symptomatic carriers for DMD or BMD. - CRD007 for the treatment of DMD, BMD and symptomatic carriers

Cardoz AB0 个研究点开始时间: 2011年10月21日最近更新: 2013年5月13日

试验速览

暂无简介。

•1.Documented diagnosis of dystrophinopathy in the form of mutation analysis in the dystrophin gene
•2.Clinically documented delayed motor skills and muscle weakness
•3.Prior documented elevated creatine kinase (>5 x ULN)
•4.Age <12 years (females in the pre-pubertal stage as defined by Tanner criterion I)
•5.Body weight =14 kg
•6.Informed assent from subject before any trial related activity is carried out
•7.Written informed consent obtained from parent(s) / legal guardian(s) before any trial related activity is carried out
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 21
•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

•1.Severe functional impairment (e.g. non-walkers) resulting in inability to perform meaningful repeated assessment of functional capabilities, according to the investigator’s judgment
•2.Moderate or severe hepatic impairment or severe renal impairment, according to the investigator’s judgment
•3.Prior or ongoing medical condition or laboratory abnormality that in the investigator’s opinion could adversely affect the safety of the subject.
•4.Subject or parent(s) / legal guardian(s) not willing and able to comply with the requirements of the study
•5.Subject not able to follow trial procedures, according to the investigator’s judgement
•6.Known individual hypersensitivity to any of the ingredients/excipients of the study medication (see Section 7.1.1)
•7. Applicable for part B, only: Systemic glucocorticoid therapy within 30 days prior to start of IMP treatment
•8.Planned systemic glucocorticoid therapy during the trial period
•9.Previous exposure to idebenone
•10.Participation in any other therapeutic trial and/or intake of any investigational drug within 90 days prior to Visit 1

发起方

Cardoz AB