Duchenne Muscular DystrophyNCT02571205
Testosterone Therapy for Pubertal Delay in Duchenne Muscular Dystrophy
An observational study of Duchenne Muscular Dystrophy, run by Newcastle-upon-Tyne Hospitals NHS Trust at 1 site in United Kingdom.
Trial 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… Read more about this study
- Who is it for?
- Ages 12 to 17 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Sustanon (testosterone)
- How many people
- 15 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- Nov 2015to Mar 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 17 years
- Men only
- No healthy volunteers
- Child
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.
Who may be able to join
- 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)
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
United Kingdom1 site
Institute of Genetic Medicine
Newcastle upon Tyne, NE1 3BZ
About this study
Official title: Observational Study of Clinical Outcomes for Testosterone Treatment of 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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Sustanon (testosterone)
- Keywords
- testosterone, pubertal delay
- Medical subject headings
- Muscular Dystrophy, Duchenne, Puberty, Delayed
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Sustanon (testosterone)Drug
Standard incremental regimen of Sustanon
What is being measured
The primary outcome is the main question the study is designed to answer.
Total score in the Treatment Satisfaction Questionnaire for Medication (TSQM)
Measured: 2 years
Secondary outcomes (12)
Subject's reported effectiveness of testosterone therapy as assessed by semi-structured interviews pre and post treatment
Measured: 2 years
Total score in Northstar Ambulatory Assessment or Performance of the Upper Limb if non-ambulant
Measured: 2 years
Z-score from Bone mineral adjusted density of the lumbar spine and total body (minus head) using Dual Xray Absorptiometry (DXA)
Measured: 2 years
Percentage of body mass assessed by DXA
Measured: 2 years
Osteocalcin level, measured by blood test
Measured: 2 years
P1NP level, measured by blood test
Measured: 2 years
Percentage fat fraction as assessed by muscle Magnetic Resonance Imaging (MRI) of upper and lower limbs
Measured: 2 years
Pubertal staging assessed using Tanner staging and testicular volume
Measured: 2 years
Bone age as assessed by wrist and hand X-Ray
Measured: 2 years
Hormonal assessment of pubertal staging using testosterone level
Measured: 2 years
Forced vital capacity, measured by spirometry
Measured: 2 years
Cardiac function, assessed by Electrocardiogram (ECG) and echo
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Oct 2015 (estimated)
- Study startNov 2015
- Primary completionFeb 2019
- Study completionMar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Michela Guglieri, Principal investigator, Newcastle University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02571205
- Sponsor's protocol ID
- 2015-003195-68
- Other IDs
- 2015
- Status last verified
- Apr 2021
- First submitted
- 24 Aug 2015
- Last updated
- 13 Apr 2021
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No: Data available on request
Publications
Wood CL, Cheetham TD, Hollingsworth KG, Guglieri M, Ailins-Sahun Y, Punniyakodi S, Mayhew A, Straub V. Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne muscular dystrophy. BMC Pediatr. 2019 Apr 25;19(1):131. doi: 10.1186/s12887-019-1503-x.
PubMed 31023296
Study at a glance
From the registry record, last updated by the sponsor on 13 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational Study of Clinical Outcomes for Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02571205
- Acronym / sponsor ID
- 2015-003195-68
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- testosterone, pubertal delay
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Newcastle-upon-Tyne Hospitals NHS TrustUniversity, hospital or other
- Study start
- Nov 2015 (actual)
- Primary completion
- Feb 2019 (actual)
- Study completion
- Mar 2019 (actual)
- First posted
- 8 Oct 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
"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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Ages
- 12 to 17 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
- 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.
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (9 listed)
- 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.)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Sustanon (testosterone)Standard incremental regimen of Sustanon | Drug | – |
Primary outcome
Total score in the Treatment Satisfaction Questionnaire for Medication (TSQM)
Time frame: 2 years
12 secondary outcomes
Subject's reported effectiveness of testosterone therapy as assessed by semi-structured interviews pre and post treatment
Time frame: 2 years
Total score in Northstar Ambulatory Assessment or Performance of the Upper Limb if non-ambulant
Time frame: 2 years
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
Percentage of body mass assessed by DXA
Time frame: 2 years
Osteocalcin level, measured by blood test
Time frame: 2 years
P1NP level, measured by blood test
Time frame: 2 years
Percentage fat fraction as assessed by muscle Magnetic Resonance Imaging (MRI) of upper and lower limbs
Time frame: 2 years
Pubertal staging assessed using Tanner staging and testicular volume
Time frame: 2 years
Bone age as assessed by wrist and hand X-Ray
Time frame: 2 years
Hormonal assessment of pubertal staging using testosterone level
Time frame: 2 years
Forced vital capacity, measured by spirometry
Time frame: 2 years
Cardiac function, assessed by Electrocardiogram (ECG) and echo
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Apr 2021.
- Derived from this study: PubMed 31023296Wood CL, Cheetham TD, Hollingsworth KG, Guglieri M, Ailins-Sahun Y, Punniyakodi S, Mayhew A, Straub V. Observational study of clinical outcomes for testosterone treatment of pubertal delay in Duchenne muscular dystrophy. BMC Pediatr. 2019 Apr 25;19(1):131. doi: 10.1186/s12887-019-1503-x.
- Individual participant dataSharing planned: No. Data available on request
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Michela Guglieri principal investigator
Newcastle University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by Newcastle-upon-Tyne Hospitals NHS Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total score in the Treatment Satisfaction Questionnaire for Medication (TSQM) (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02571205, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Apr 2021.