Testosterone therapy in DMD

Phase 1

• Conditions: Duchenne Muscular Dystrophy; MedDRA version: 18.1Level: PTClassification code 10013801Term: Duchenne muscular dystrophySystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2015-003195-68-GB
• Lead Sponsor: The Newcastle upon Tyne NHS Hospitals Foundation Trust

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2015-10-26
• Last Update Posted: 27 July 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 20

Inclusion Criteria

•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

Exclusion Criteria

•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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified