Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial

niversity of Basel Children's Hospital, Division of Neuropediatrics0 个研究点目标入组 100 人开始时间: 2018年10月2日最近更新: 2021年1月5日

适应症

试验速览

暂无简介。

•Group A (ambulant patients)
•-Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
•-Stable treatment with glucocorticoids >6 months (no significant change in dosage (>0.2mg/kg)) at screening; dosing adaptations according to weight change are allowed
•-Male gender
•-6.5 to 12 years of age at time of screening
•-weight >15kg
•-ambulant patients
•-able to walk at least 350 meters in 6 minute walking distance test without assistance at screening
•-MFM D1 subdomain of the MFM scale >40% at screening
•-Ability to provide informed consent and to comply with study requirements
•Group B (non-ambulant patients)
•-Documented diagnosis of DMD by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e. absent or <5% of normal) on Western blot or immunostaining
•-not using glucocorticoids for >6 months
•-Male gender
•-non-ambulant patients (walking distance less than 10 meters)
•-10 to 16 years of age at time of screening
•-Ability to provide informed consent and to comply with study requirements
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 100
•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

•-Known individual hypersensitivity or allergy to tamoxifen
•-Female gender
•-Use of tamoxifen or testosterone within the last 3 months
•-Known or suspected malignancy
•-Other chronic disease or clinically relevant limitation of renal, liver or heart function (as judged by the Investigator)
•-Known or suspected non-compliance
•-Any injury which may impact functional testing, e.g. upper or lower limb fracture
•-Planned or expected spinal fusion surgery during the study period (as judged by the Investigator; i.e. due to rapid progressing scoliosis), previous spinal fusion surgery is allowed if it took place more than 6 month prior to screening.
•-Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders of the participant/parents (as judged by the investigator)
•-Concomitant participation in any other interventional trial (and up to 3 months prior to screening)
•- Use of CYP2D6 inhibitors or of CYP3A4 inducers, platelet aggregation inhibitors and coumarin-type anti-coagulants
•-Glucocorticoid naïve patients
•-Start of glucocorticoid treatment or change in dosage <6 month prior to screening (dosing adaptations according to weight change are allowed)
•-Glucocorticoid treated patients or patients that stopped steroid treatment <6 month prior to screening
•-Participation in any other interventional trial
•-Assisted ventilation of any kind necessary

发起方

niversity of Basel Children's Hospital, Division of Neuropediatrics