Testosterone therapy in Duchenne Muscular Dystrophy
Research type
Research Study
Full title
Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne Muscular Dystrophy.
IRAS ID
182134
Contact name
Michela Guglieri
Contact email
Sponsor organisation
The Newcastle upon Tyne NHS Hospitals Foundation Trust
Eudract number
2015-003195-68
Duration of Study in the UK
2 years, 3 months, 2 days
Research 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 and delayed puberty who are treated by the Newcastle muscle team, as they are treated with testosterone to induce puberty. They will all be treated with the standard stepwise regimen of testosterone injections every 4 weeks and data will be collected to help us determine the effectiveness and tolerability of the current treatment regimen. We 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. We will also carry out some interviews 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 our limited experience in this group, testosterone treatment seems to be well-liked and tolerated but we do not know the best treatment regimen to use and there is no current consensus. It is not currently part of the official 'standard of care' in DMD but it would be important to include it if we can show that it is an effective treatment for pubertal delay.
REC name
North East - York Research Ethics Committee
REC reference
15/NE/0332
Date of REC Opinion
15 Oct 2015
REC opinion
Further Information Favourable Opinion