Tamoxifen in Duchenne muscular dystrophy - TAMDMD: A multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trial The study will be extended to an open label study with the following title (OLE: Open Label Extension): Tamoxifen in Duchenne muscular dystrophy - TAMDMD: A 48 week open label extension of a multi centre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy trial - TAMDMD

KBB University of Basel Children's Hospital/Division of Neuropediatrics0 个研究点目标入组 18 人开始时间: 待定最近更新: 2024年4月15日

试验速览

阶段: 3 期
状态: 已完成
发起方
入组人数: 18

研究概览入排标准研究者记录与标识符相似试验

研究概览

简要总结

暂无简介。

研究设计

研究类型: Interventional

入排标准

年龄范围: 2 至 15（—）

入选标准

•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 >20kg
•- 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
•- Patients harbouring a nonsense mutation treatable with the approved drug
•ataluren should be under stable ataluren treatment for at least 3 months or in
•case of non tolerance being off ataluren treatment for at least 3 months before
•screening 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
•requirements7S8S9S
•Open label Extension: Recent participation and completion of TAMDMD study

排除标准

•- Known individual hypersensitivity or allergy to tamoxifen or other
•ingredients /excipients of IMP
•- 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
•- 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 (apart from
•glucocorticoids), platelet aggregation inhibitors and coumarin-type
•anti-coagulants
•- Use of drugs metabolized by CYP2C9, such as phenprocoumon, phenytoin,
•warfarin, celecobix, fluvastatin, ginko biloba, st. John's wort and
•sulfamethoxazol.
•- Galactosemia (lack of galactose-1-phosphat-uridylyltransferase or
•UDP-galactose-4-epimerase or galactokinase; Fanconi-Bickel-syndrome);
•congenital lack of lactase; glucose-galactose malabsorption.
•- Presence of one or more of the following eye disorders: cataract,
•retinopathia, optic neuropathy, alteration of the cornea.
•Presence of one or more of the following laboratory abnormalities: anaemia,
•thrombocytopenia, leukopenia, neutropenia or agranulocytosis.Group A:
•- Glucocorticoid naïve patients
•- Start of glucocorticoid treatment or change in dosage <6 month prior to
•screening (dosing adaptations according to weight change are allowed)Group B:
•- Glucocorticoid treated patients or patients that stopped steroid treatment <6
•month prior to screening
•- Participation in any other interventional trial

研究者

发起方

KBB University of Basel Children's Hospital/Division of Neuropediatrics

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