EUCTR2017-004554-42-GB进行中（未招募）1 期
Tamoxifen in Duchenne muscular dystrophy - TAMDMDA multicentre, randomised, double-blind, placebo-controlled, phase 3 safety and efficacy 48-week trialTamoxifen in Duchenne muscular dystrophy: A 48-week open labelextension of a multicentre, randomised, double-blind, placebo-controlled,phase 3 safety and efficacy 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8月3日最近更新:
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 100
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
- •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 nontolerance 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
- •requirements
- •Open label extension
- •- Recent participation and completion of TAMDMD study
- •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
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
- •- 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 months 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, celecoxib, fluvastatin, ginko biloba, St. John’s
- •wort and sulfamethoxazol
- •- Galactosemia (lack of galactose-1-phosphat-uridylyltransferase or
- •UDP-galactose-4-epimerase or galactokinase; Fanconi-Bickelsyndrome);
- •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
- •- 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
- •glucocorticoid treatment <6 month prior to screening
- •- Assisted ventilation of any kind necessary
研究者
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