Klinefelter SyndromePhase 4NCT02408445
Body Composition in Infants With Klinefelter Syndrome and Effects of Testosterone Treatment
A Phase 4 study testing testosterone cypionate 200mg/ml in people with Klinefelter Syndrome, run by University of Colorado, Denver at 1 site in United States.
Trial summary
This research study in infant males with Klinefelter syndrome (47,XXY) will learn more about body composition (muscle and fat) and male hormones and look at the effect of testosterone shots on body composition. Read more about this study
- Who is it for?
- Ages 42 to 108 days, men only
- Conditions the study is for
- Klinefelter Syndrome
- What is being tested?
- testosterone cypionate 200mg/ml
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 8 May 2015to 1 Jan 2020
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 42 to 108 days
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male infants with 47,XXY karyotype
Who cannot join
- Gestational age at birth <36 weeks
- Birth weight <5%ile or >95% for gestational age
- History of thrombosis in a first degree relative
- Exposure to androgen therapy outside of the study protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Hospital Colorado
Aurora, Colorado, 80045
About this study
This research study in infant males with Klinefelter syndrome (47,XXY) will learn more about body composition (muscle and fat) and male hormones and look at the effect of testosterone shots on body composition. The Investigators know that older boys and men with Klinefelter syndrome often have more fat compared to muscle than adults without Klinefelter syndrome, but we do not know if this difference is present at birth or develops over time. The Investigators will learn if body composition and motor skills are improved with testosterone treatment in infants with Klinefelter syndrome.
- Conditions
- Klinefelter Syndrome
- Being tested
- testosterone cypionate 200mg/ml
- Keywords
- body composition, Klinefelter syndrome, XXY, sex chromosome variation, sex chromsome aneuploidy
- Medical subject headings
- Klinefelter Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
testosterone cypionate 200mg/mlDrug
Subjects in this group will be randomized to receive testosterone cypionate 200 mg/ml to be given intramuscularly every 4 weeks for a total of 3 doses.
| Study group | Role | What they receive |
|---|---|---|
| Testosterone treatment | Experimental | Testosterone cypionate (200 mg/ml) intramuscular injection Receives: Drug: testosterone cypionate 200mg/ml |
| No treatment | No intervention | Subjects will not receive any testosterone during the study period. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Body Fat Percent Z-score
Body fat percentage will be measured using air displacement plethysmography (PEA POD) at the beginning and end of the study period. Age and sex-normed z-scores will be calculated. The Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Change in the z-score over time, if following a normal growth curve, is 0. Positive change in z-scores indicates a gain in body fat above growth typically expected. Negative change in z-scores indicates a gain in body fat that is less than typically expected.
Measured: Baseline and 3 months
Secondary outcomes (11)
Serum Luteinizing Hormone (LH)
Serum will be collected at the first study visit prior to randomization. Ultrasensitive LH will be measured.
Measured: baseline only
Serum Follicle Stimulating Hormone (FSH)
Serum will be collected at the first study visit prior to randomization. Ultrasensitive FSH will be measured.
Measured: baseline only
Serum Total Testosterone
Serum will be collected at the first study visit prior to randomization. Total testosterone by mass spectroscopy will be measured.
Measured: baseline only
Serum Inhibin B (INHB)
Serum will be collected at the first study visit prior to randomization. Inhibin B levels will be measured.
Measured: baseline only
Serum Anti-Mullerian Hormone (AMH)
Serum will be collected at the first study visit prior to randomization. AMH levels will be measured.
Measured: baseline only
Leptin
Serum will be collected at the first study visit prior to randomization. Leptin levels will be measured.
Measured: baseline only
Change in Raw Score on the Alberta Infant Motor Scale
Muscle tone and motor development will be assessed by an occupational therapist using the standardized Alberta Infant Motor Scale (AIMS). The AIMS scale measures infant motor maturation from birth until the age of independent walking. An occupational therapists assesses 58 motor behavior items in 4 position categories: prone (21 items), supine (9 items), sitting (12 items) \& standing(16 standing). Each item receives one point (range of raw scores 0-58), with higher scores indicating more skills acquired. For change in scores, the raw score at 3 months was subtracted from the baseline raw score.
Measured: 3 months
Change in Score on the Movement Assessment of Infants (MAI)
Muscle tone and motor development will be assessed by an occupational therapist using the standardized Movement Assessment of Infants (MAI). The MAI evaluates four domains: muscle tone, primitive reflex, automatic reactions and volitional movement. All items are scored 1-5 and summed to generate a "Total Risk Score". Lower scores indicate better function, and Total Risk Scores of 8 or more indicate high risk.
Measured: 3 months
Change in Total Motor Standard Score on the Peabody Developmental Motor Scales 2
Motor development will be assessed by an occupational therapist using the standardized Peabody Developmental Motor Scales 2. Standard scores are normalized to age with a mean of 100 and standard deviation of 15. Change in standard score was calculated as the differences between the subject's standard score at 3 months minus the standard score at baseline. A positive change in standard scores would indicate greater growth on the measure relative to peers, while a negative number would indicate slower growth on the measure relative to peers.
Measured: 3 months
Change in Penile Length
Stretched penile length will be measured by a physician before randomization and at the end of the study period.
Measured: Baseline and 3 months
Change in Fat Free Mass
Fat free mass (lean mass) will be measured using air displacement plethysmography (PEA POD) at the beginning and end of the study period.
Measured: Baseline and 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2015 (estimated)
- Study start8 May 2015
- Primary completion31 Jan 2018
- Study completion1 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Klinefelter SyndromeStudies of the same condition, open and under way.
- More trials run by University of Colorado, DenverThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Colorado, Denver
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Shanlee M Davis, MD, Principal investigator, University of Colorado, Denver
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02408445
- Sponsor's protocol ID
- 14-1720
- Other IDs
- UL1TR001082
- Status last verified
- Apr 2020
- First submitted
- 31 Mar 2015
- Last updated
- 15 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Body Composition in Infants With Klinefelter Syndrome and Effects of Testosterone Treatment
- ClinicalTrials.gov ID
- NCT02408445
- Acronym / sponsor ID
- 14-1720
- Conditions
- Klinefelter Syndrome
- Keywords
- body composition, Klinefelter syndrome, XXY, sex chromosome variation, sex chromsome aneuploidy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of Colorado, DenverUniversity, hospital or other
- Study start
- 8 May 2015 (actual)
- Primary completion
- 31 Jan 2018 (actual)
- Study completion
- 1 Jan 2020 (actual)
- First posted
- 3 Apr 2015 (estimated)
- Results
- Posted 15 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study in infant males with Klinefelter syndrome (47,XXY) will learn more about body composition (muscle and fat) and male hormones and look at the effect of testosterone shots on body composition. The Investigators know that older boys and men with Klinefelter syndrome often have more fat compared to muscle than adults without Klinefelter syndrome, but we do not know if this difference is present at birth or develops over time. The Investigators will learn if body composition and motor skills are improved with testosterone treatment in infants with Klinefelter syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Testosterone treatment | Experimental | Drug: testosterone cypionate 200mg/mlTestosterone cypionate (200 mg/ml) intramuscular injection |
| No treatment | No intervention | –Subjects will not receive any testosterone during the study period. |
Who may qualify
- Ages
- 42 to 108 days
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (1 listed)
- Male infants with 47,XXY karyotype
Key exclusion criteria (4 listed)
- Gestational age at birth <36 weeks
- Birth weight <5%ile or >95% for gestational age
- History of thrombosis in a first degree relative
- Exposure to androgen therapy outside of the study protocol.
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 |
|---|---|---|
| testosterone cypionate 200mg/mlSubjects in this group will be randomized to receive testosterone cypionate 200 mg/ml to be given intramuscularly every 4 weeks for a total of 3 doses. | Drug | Testosterone treatment |
Primary outcome
Change in Body Fat Percent Z-score
Time frame: Baseline and 3 months
Body fat percentage will be measured using air displacement plethysmography (PEA POD) at the beginning and end of the study period. Age and sex-normed z-scores will be calculated. The Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Change in the z-score over time, if following a normal growth curve, is 0. Positive change in z-scores indicates a gain in body fat above growth typically expected. Negative change in z-scores indicates a gain in body fat that is less than typically expected.
11 secondary outcomes
Serum Luteinizing Hormone (LH)
Time frame: baseline only
Serum Follicle Stimulating Hormone (FSH)
Time frame: baseline only
Serum Total Testosterone
Time frame: baseline only
Serum Inhibin B (INHB)
Time frame: baseline only
Serum Anti-Mullerian Hormone (AMH)
Time frame: baseline only
Leptin
Time frame: baseline only
Change in Raw Score on the Alberta Infant Motor Scale
Time frame: 3 months
Change in Score on the Movement Assessment of Infants (MAI)
Time frame: 3 months
Change in Total Motor Standard Score on the Peabody Developmental Motor Scales 2
Time frame: 3 months
Change in Penile Length
Time frame: Baseline and 3 months
Change in Fat Free Mass
Time frame: Baseline and 3 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Shanlee M Davis, MD principal investigator
University of Colorado, Denver
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing testosterone cypionate 200mg/ml in people with Klinefelter Syndrome, run by University of Colorado, Denver at 1 site in United States.
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 1 inclusion and 4 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 15 Apr 2020. 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 States. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Apr 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Change in Body Fat Percent Z-score (time frame: Baseline and 3 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 15 Apr 2020.