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Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02571205
Recruitment Status : Active, not recruiting
First Posted : October 8, 2015
Last Update Posted : September 28, 2018
Sponsor:
Information provided by (Responsible Party):
Newcastle-upon-Tyne Hospitals NHS Trust

Brief 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.

Condition or disease Intervention/treatment
Duchenne Muscular Dystrophy Drug: Sustanon (testosterone)

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Study Type : Observational
Actual Enrollment : 15 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Observational Study of Clinical Outcomes for Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy
Study Start Date : November 2015
Estimated Primary Completion Date : February 2019
Estimated Study Completion Date : March 2019



Intervention Details:
  • Drug: Sustanon (testosterone)
    Standard incremental regimen of Sustanon


Primary Outcome Measures :
  1. Total score in the Treatment Satisfaction Questionnaire for Medication (TSQM) [ Time Frame: 2 years ]

Secondary Outcome Measures :
  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 ]

Biospecimen Retention:   Samples With DNA
Samples to be collected as part of Biobank


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Ages Eligible for Study:   12 Years to 17 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
This is a single-centre, prospective, observational, study on clinical outcomes of testosterone replacement therapy in adolescents with DMD and delayed puberty. Being an observational study of routine care, there will be no specified end point. We will aim to recruit any adolescent with DMD and delayed puberty who is reviewed by the muscle team at the John Walton Muscular Dystrophy Research Centre from the study approval date.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02571205


Locations
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United Kingdom
Institute of Genetic Medicine
Newcastle Upon Tyne, United Kingdom, NE1 3BZ
Sponsors and Collaborators
Newcastle-upon-Tyne Hospitals NHS Trust
Investigators
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Principal Investigator: Michela Guglieri Newcastle University

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Responsible Party: Newcastle-upon-Tyne Hospitals NHS Trust
ClinicalTrials.gov Identifier: NCT02571205     History of Changes
Other Study ID Numbers: 2015-003195-68
First Posted: October 8, 2015    Key Record Dates
Last Update Posted: September 28, 2018
Last Verified: September 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Data available on request
Keywords provided by Newcastle-upon-Tyne Hospitals NHS Trust:
testosterone
pubertal delay
Additional relevant MeSH terms:
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Muscular Dystrophy, Duchenne
Muscular Dystrophies
Puberty, Delayed
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Gonadal Disorders
Endocrine System Diseases
Methyltestosterone
Testosterone
Testosterone undecanoate
Testosterone enanthate
Testosterone 17 beta-cypionate
Androgens
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Antineoplastic Agents, Hormonal
Antineoplastic Agents
Anabolic Agents