Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 26 April 2021
• Main ID: NCT02571205
• Date of registration: 24/08/2015
• Prospective Registration: Yes
• Primary sponsor: Newcastle-upon-Tyne Hospitals NHS Trust
• Public title: Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
• Scientific title: Observational Study of Clinical Outcomes for Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy
• Date of first enrolment: November 2015
• Target sample size: 15
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02571205
• Study type: Observational

Countries of recruitment

United Kingdom

Contacts

• Name: Michela Guglieri
• Affiliation: Newcastle University

Key inclusion & exclusion criteria

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



Age minimum: 12 Years
Age maximum: 17 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Sustanon (testosterone)

Primary Outcome(s)

Total score in the Treatment Satisfaction Questionnaire for Medication (TSQM) [Time Frame: 2 years]

Secondary Outcome(s)

Z-score from Bone mineral adjusted density of the lumbar spine and total body (minus head) using Dual Xray Absorptiometry (DXA) [Time Frame: 2 years]

Forced vital capacity, measured by spirometry [Time Frame: 2 years]

Pubertal staging assessed using Tanner staging and testicular volume [Time Frame: 2 years]

Percentage of body mass assessed by DXA [Time Frame: 2 years]

Total score in Northstar Ambulatory Assessment or Performance of the Upper Limb if non-ambulant [Time Frame: 2 years]

Bone age as assessed by wrist and hand X-Ray [Time Frame: 2 years]

Cardiac function, assessed by Electrocardiogram (ECG) and echo [Time Frame: 2 years]

Hormonal assessment of pubertal staging using testosterone level [Time Frame: 2 years]

Percentage fat fraction as assessed by muscle Magnetic Resonance Imaging (MRI) of upper and lower limbs [Time Frame: 2 years]

Subject's reported effectiveness of testosterone therapy as assessed by semi-structured interviews pre and post treatment [Time Frame: 2 years]

Osteocalcin level, measured by blood test [Time Frame: 2 years]

P1NP level, measured by blood test [Time Frame: 2 years]

Secondary ID(s)

2015-003195-68

Source(s) of Monetary Support

Please refer to primary and secondary sponsors