Testosterone in DMD follow up study

This is an observational study, designed to investigate the longer term effects of testosterone replacement therapy after treatment for delayed puberty in DMD. The aim is to continue to monitor testosterone levels, growth, muscle strength, mass and function, bone mineral density, body composition and quality of life in a cohort of young men with DMD, for two years after they have completed a previous clinical trial (NCT02571205) where they were given testosterone by injection to induce puberty. The extension to the original study will enable to investigate the longer term consequences of testosterone treatment after earlier induction of puberty.

 
Primary Outcome Measures

Evaluate the effect of previous pubertal induction on:

1. Hormonal assessment of gonadal function (measured by morning blood levels of testosterone, LH, FSH, AMH and Inhibin B)
2. Physical assessment of gonadal function (measured by testicular volume)

 

 
Secondary Outcome Measures

Evaluate the effect of previous pubertal induction on:

1. Quality of life and patient satisfaction as measured by Treatment Satisfaction Questionnaire for Medication (TSQM) and PEDSQoL questionnaires
2. Motor function as measured by standardised physiotherapy assessments (Northstar Ambulatory Assessment (NSAA), Performance of the Upper Limb (PUL) EK scale, timed tests (Time to Rise from the Floor, Time to run/walk 10 meters) and assessment of workable reach space using Kinect
3. Muscle strength as measured by hand-held myometry (knee flexion and extension, elbow flexion and extension, shoulder abduction) and grip strength
4. Bone mineral adjusted density of the lumbar spine and total body (minus head) using DXA and bone maturation using wrist x-ray.
5. Assessment of lean versus total body mass assessed by DXA
6. Bone turnover markers (bone-specific alkaline phosphatase, beta crosslaps, osteocalcin, P1NP, RANKL, OPG, sclerostin)
7. Muscle volume and fat fraction as assessed by muscle MRI of upper and lower limbs
8. Cortical and trabecular bone structure assessed by spinal x-ray
9. Trabecular bone microarchitecture assessed by bone MRI, in subset of patients
10. Forced vital capacity, forced expiratory volume and peak cough flow, measured by spirometry

 
Can I take part?

 
Inclusion Criteria

 
Exclusion Criteria