Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
Status:
Completed
Trial end date:
2019-03-01
Target enrollment:
Participant gender:
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.