Klinefelter Syndrome and Testosterone Treatment in Puberty (TiPY)

March 5, 2024 updated by: Lise Aksglæde

Klinefelter Syndrome - the Effect of Testosterone Treatment In Puberty. A Randomized, Double-blind Placebo-controlled Intervention Study: 'The TiPY Study'

The goal of this randomized clinical trial is to study the effect of testosterone replacement therapy during puberty in boys with Klinefelter syndrome (KS, 47,XXY).

The main questions to answer are how treatment with testosterone will affect body fat mass, lipid and glucose metabolism, growth and body proportions, bone mineralization as well as effects on neurocognitive development and emotional and social difficulties.

Participants will be randomized to two years treatment with testosterone or placebo.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Klinefelter syndrome (KS, 47,XXY) is the most frequent sex chromosome disorder with a prevalence of 1:660 boys. Patients with KS are hypogonadal due to a progressive testicular destruction starting already in childhood. Consequently, the adult male with KS is characterized by small testes, signs of incomplete virilization (e.g. lack of voice deepening, sparse face and body hair, gynecomastia, low muscle mass, reduced penile length), hypergonadotropic hypogonadism, infertility and increased risk of metabolic syndrome, diabetes, cardiovascular disease, osteoporosis and psychosocial and neurodevelopmental challenges. Adults with KS have a poor health and a prevention of the major co-morbidities associated with KS and thereby an improvement in the general health would have an enormous impact on the life of a large cohort of males worldwide.

Sufficient testosterone is not only important in the adult but also during puberty and adolescence for a normal virilization and to improve body composition and body proportions, as well as to maximize peak bone mass acquisition. It has therefore been internationally accepted and makes biological sense to consider testosterone replacement therapy (TRT) during puberty in KS. However, there are no evidence based recommendations, and during recent years TRT in puberty has been questioned and is no longer recommended in some countries. There is a need on an international level for evaluating the effect of this treatment. We therefore aim at evaluating the effect of 2 years TRT during early puberty in boys with KS aged 10 to 14 years in this national, multi-center, randomized, double-blind, placebo-controlled intervention study. The primary endpoint is to evaluate the effect on body fat mass. The secondary endpoints are to evaluate effects on lipid and glucose metabolism, growth and body proportions, bone mineralization as well as effects on neurocognitive development and emotional and social difficulties.

Study Type

Interventional

Enrollment (Estimated)

32

Phase

  • Not Applicable

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • 47,XXY Klinefelter syndrome
  • Age 10-14 years at inclusion
  • Luteinizing Hormone > +2 standard deviations (SD) by ultrasensitive luteinizing hormone assay
  • Free Testosterone<+2 standard deviations
  • Signed consent from parents

Exclusion Criteria:

  • Previous or ongoing T treatment except for TRT because of micropenis
  • Contraindications to testosterone treatment known hypersensitivity to testosterone or to any other constituent of the gel known or suspected prostatic cancer or breast carcinoma
  • Participation in any other clinical trial

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Active Comparator: Testosterone
Testosterone gel applied to the skin
Drug: Testosterone gel
Two years treatment with testosterone
Other Names:
  • Testosterone
Placebo Comparator: Placebo
Placebo gel applied to the skin
Other: Placebo
Two years treatment with placebo
Other Names:
  • Testosterone

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes in body fat mass
Time Frame: Baseline and 1 and two years
Evaluation of body fat percentage by whole body dual energy x-ray absorptiometry (DEXA) scan
Baseline and 1 and two years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pubertal development and virilization
Time Frame: Every three months for two years
Tanner staging
Every three months for two years
Height
Time Frame: Every three months for two years
Measurement of height (cm)
Every three months for two years
Weight
Time Frame: Every three months for two years
Measurement of weight (kg)
Every three months for two years
Bone mineralization
Time Frame: Baseline and 1 and two years
Evaluated by DEXA scan
Baseline and 1 and two years
Neuropsychological evaluation
Time Frame: Baseline and two years
Wechsler Intelligence Scale for Children® Fifth Edition (WISC®-V)
Baseline and two years
Neuropsychological evaluation,
Time Frame: Baseline and two years
The test of variables of attention (TOVA)
Baseline and two years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Lise Aksglæde

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

April 1, 2024

Primary Completion (Estimated)

December 31, 2029

Study Completion (Estimated)

December 31, 2029

Study Registration Dates

First Submitted

February 21, 2024

First Submitted That Met QC Criteria

March 5, 2024

First Posted (Estimated)

March 6, 2024

Study Record Updates

Last Update Posted (Estimated)

March 6, 2024

Last Update Submitted That Met QC Criteria

March 5, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2023-505854-16-00

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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