Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy (NCT02571205) | Clinical Trial Compass
CompletedNot Applicable
Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
United Kingdom15 participantsStarted 2015-11
Plain-language summary
"Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy" is a single centre observational study that aims to follow the progress of 20 adolescents with Duchenne Muscular Dystrophy (DMD) and delayed puberty who are treated by the Newcastle muscle team, as they are treated with testosterone to induce puberty. The participants will all be treated with the standard stepwise regimen of testosterone injections every 4 weeks and data will be collected to help determine the effectiveness and tolerability of the current treatment regimen. The investigators will use the data to explore the effect of testosterone on pubertal development, growth, muscle strength and function, bone mineral density and body composition and characterise any side effects. Semi-structured interviews will also be carried out to learn the boys' views on the tolerability of the regimen. The study will last up to a maximum of 27 months in total for each participant, but may be less if they are happy with pubertal development before this time. It is important to do this study because from the investigator's limited experience in this group, testosterone treatment seems to be well liked and tolerated but the best treatment regimen to use remains unknown and there is no current consensus. It is not currently part of the standard of care in DMD but it would be important to include it if this study can show that it is an effective treatment for pubertal delay.
Who can participate
Age range
12 Years – 17 Years
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* A molecular diagnosis of Duchenne Muscular Dystrophy.
* Males aged between 12 and 17 years of age at time of first dosing
* Prepubertal (Tanner stage 1, testicular volume \<4 mls, initial testosterone level of \<2.0 nmol/l)
* Subjects are receiving the standard of care for DMD as recommended by the NorthStar UK and TREAT-NMD guidelines
* Patients are capable of sitting upright in a wheelchair for at least an hour
* Patients have stable respiratory function. Artificial ventilation with either Bipap/continuous positive airways pressure (CPAP) or tracheostomy is not a contraindication to the study.
* Informed consent/assent signed by the patient (or parent/guardian if under 16 years of age)
Exclusion Criteria:
* 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 (including hypersensitivity and allergy to peanuts or soya.)
* Any contra-indication to receiving an intramuscular injection
* Any additional chronic disease that affects androgen production
* Anti-coagulant therapy
* If participation in the study is not recommended in the opinion of the investigators
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Total score in the Treatment Satisfaction Questionnaire for Medication (TSQM)