This trial is active, not recruiting.

Condition klinefelter syndrome
Treatments androgen oxandrolone, placebo
Phase phase 2
Sponsor Thomas Jefferson University
Collaborator National Institute of Neurological Disorders and Stroke (NINDS)
Start date July 2006
End date April 2016
Trial size 150 participants
Trial identifier NCT00348946, R01NS050597-01A2


The purpose of this study is to evaluate the effects of low-dose androgen on the motor and cognitive development of boys with Klinefelter syndrome.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification safety study
Intervention model parallel assignment
Masking double blind (subject, caregiver, investigator)
Primary purpose treatment
(Active Comparator)
androgen oxandrolone
Oxandrolone or placebo capsule, .06 >mg/kg/day, orally, for 2 years
(Placebo Comparator)
an inactive substance

Primary Outcomes

Evaluation of several aspects of motor function including muscle strength, motor response speed, simple repetitive movement, and complex nonrepetitive motor action, previously shown to be impaired in boys with Klinefelter syndrome.
time frame: 2 years per subject

Eligibility Criteria

Male participants from 4 years up to 12 years old.

Inclusion Criteria: - Karyotype diagnosis of Klinefelter syndrome - Chronological age of 4-12 years - No treatment with androgen in the past year Exclusion Criteria: - Major liver, kidney or other systemic disease - Variant karyotypes including 47,XYY males - Evidence of spontaneous onset of puberty, defined as testicular size > 4ml

Additional Information

Official title Androgen Effect on Motor/Cognitive Outcome in Klinefelter Syndrome
Principal investigator Judith L. Ross, M.D.
Description Klinefelter syndrome (KS), a genetic disorder that affects males only, is characterized by having an extra X chromosome. The phenotype — or physical and learning features — includes testicular failure, tall stature, and specific cognitive and behavioral attributes such as diminished motor function, language-based learning difficulties, poor self-image, and shyness. The KS phenotype may be the result of androgen deficiency in utero, infancy, and childhood. For individuals with KS, androgen replacement is standard treatment in adolescence and adulthood but has not been used earlier in childhood or included in the standard medical care of KS children ages 4 to 12. The purpose of this study is to examine the effects of androgen on learning and development in boys with KS. Researchers also want to determine if low-dose androgen replacement at an early age will improve some of the learning difficulties associated with the disorder. The overall goal of this study is to address questions regarding the relationship of early androgen deficiency to learning and motor function. Participants in the study will be randomized to one of two treatment groups, receiving either oxandrolone (low-dose androgen) or placebo, for two years. All participants will be evaluated for safety at the beginning of the study and at 3, 6, 12, 18, and 24 months. Also at the beginning of the study and every 3 to 6 months thereafter (for a total of 6 visits), the researchers will perform a careful history and physical examination and a bone age X-ray, and obtain a blood sample. Participation in the trial will last two years and includes 6 clinic visits.
Trial information was received from ClinicalTrials.gov and was last updated in April 2015.
Information provided to ClinicalTrials.gov by Thomas Jefferson University.