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NCT Number: NCT02571205

Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy

"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.

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Key information

Conditions

Duchenne Muscular Dystrophy Congenital, Hereditary, and Neonatal Diseases and Abnormalities Endocrine System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Gonadal Disorders Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Muscular Dystrophy, Duchenne Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases Puberty, Delayed

Age range

12 year–17 year

Sex eligibility

Male

Study type

Observational

Primary location

Institute of Genetic Medicine

Newcastle upon Tyne, NE1 3BZ, United Kingdom

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Sustanon (testosterone)

Drug

Standard incremental regimen of Sustanon

Primary outcomes

  1. Total score in the Treatment Satisfaction Questionnaire for Medication (TSQM)

    Time frame: 2 years

Secondary outcomes

  1. Subject's reported effectiveness of testosterone therapy as assessed by semi-structured interviews pre and post treatment

    Time frame: 2 years

  2. Total score in Northstar Ambulatory Assessment or Performance of the Upper Limb if non-ambulant

    Time frame: 2 years

  3. 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

  4. Percentage of body mass assessed by DXA

    Time frame: 2 years

  5. Osteocalcin level, measured by blood test

    Time frame: 2 years

  6. P1NP level, measured by blood test

    Time frame: 2 years

  7. Percentage fat fraction as assessed by muscle Magnetic Resonance Imaging (MRI) of upper and lower limbs

    Time frame: 2 years

  8. Pubertal staging assessed using Tanner staging and testicular volume

    Time frame: 2 years

  9. Bone age as assessed by wrist and hand X-Ray

    Time frame: 2 years

  10. Hormonal assessment of pubertal staging using testosterone level

    Time frame: 2 years

  11. Forced vital capacity, measured by spirometry

    Time frame: 2 years

  12. Cardiac function, assessed by Electrocardiogram (ECG) and echo

    Time frame: 2 years

Sponsors and collaborators

Lead sponsor

Newcastle-upon-Tyne Hospitals NHS Trust

Other

Registry information

Official study title

Observational Study of Clinical Outcomes for Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy

Important dates

Study start
2015
Primary completion
2019
Study completion
2019
First posted
Oct 8, 2015
Registry last updated
Apr 13, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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