Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy. - Testosterone therapy in Duchenne Muscular Dystrophy

The Newcastle upon Tyne NHS Hospitals Foundation Trust0 个研究点目标入组 20 人开始时间: 2015年10月26日最近更新: 2020年7月27日

适应症

试验速览

暂无简介。

•A molecular diagnosis of Duchenne Muscular Dystrophy.
•Pre-pubertal males aged between 12 and 17 years of age at time of first dosing (Tanner stage 1, testicular volume <4 mls, initial testosterone level of <2.0 nmol/l)
•Patients should receive the standard of care for DMD as recommended by the NorthStar UK and TREAT-NMD guidelines
•Patients should be capable of sitting upright in a wheelchair for at least an hour
•Patients should be stable from a respiratory point of view. Artificial ventilation with either Bipap or tracheostomy is not a contraindication to the study.
•Informed consent signed by the patient (or parent/guardian if under 16 years of age and assent)
•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 0
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range 0

•Participants with severe learning difficulties that would preclude them from cooperating with examination.
•Anticipated surgery during the study period.
•Symptomatic cardiac failure.
•Participants/families who may have emotional or psychological problems if recruited to a study
•Hypersensitivity to the active substance or to any of the excipients, including arachis oil or derivatives. Sustanon is therefore contraindicated in patients allergic to peanuts or soya.
•Any contra-indication to receiving an intramuscular injection
•Any additional chronic disease that affects androgen production
•Anti-coagulant therapy
•None of the current treatments for DMD are exclusion criteria
•If participation in the study is not recommended in the opinion of the investigators

发起方

The Newcastle upon Tyne NHS Hospitals Foundation Trust