Androgen Effect on Klinefelter Syndrome Motor Outcome

NCT00348946 | Completed Phase 2 Results DMC

• 1 other identifier: R01NS050597-01A2
• Study Type: interventional
• Target: 93
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Klinefelter Syndrome

Keywords

Klinefelter syndrome; androgen; androgen oxandrolone

Outcome Measures

Primary Outcomes (1)

• Motor Function/Strength

  Outcome measures were tested using the following assessments: Bruininks-Osertesky Test of Motor Proficiency (BOT) subscales of (1) Visual Motor Control, (2) Upper limb Speed, and (3) Strength, Physical and Neurological Evaluation for Soft Signs (PANESS), and Hand Strength Dynamometer. BOT assess the child's motor development and includes standard scores (mean=100, SD=15) and subtest scores and is normed for sex and age (4-14.5 years). PANESS assesses the time required to press thumb to 4 fingers 20 times for the dominant and nondominant hands and includes standard scores (mean=100, SD=15) with age-specific norms (4-18 years). Hand strength dynamometer assess hand strength in the dominant and nondominant hands and includes standard scores (mean=100, SD=15). Data is expressed as standard scores with mean of 100 and SD of 15. The minimum standard score is 50; the maximum standard score is 145. Higher scorers imply better function.

  2 years per subject

Secondary Outcomes (4)

• Cognitive Function and Language

  2 years per subject

• Working Memory/Attention

  2 years per participant

• Psychosocial and Behavior Domain

  2 years per participants

• Psychosocial and Behavior Domain

  2 years per subject

Study Arms (2)

Oxandrolone

ACTIVE COMPARATOR

Androgen oxandrolone: Oxandrolone, 0.6 \> mg/kg/day, orally, for 2 years.

Drug: androgen oxandrolone

Placebo

PLACEBO COMPARATOR

An inactive substance.

Other: placebo

Interventions

androgen oxandrolone DRUG

Oxandrolone ,06 \>mg/kg/day, orally, for 2 years

Oxandrolone

placebo OTHER

an inactive substance

Placebo

Eligibility Criteria

• Age: 4 Years - 12 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Karyotype diagnosis of Klinefelter syndrome
• Chronological age of 4-12 years
• No treatment with androgen in the past year

You may not qualify if:

• Major liver, kidney or other systemic disease
• Variant karyotypes including 47,XYY males
• Evidence of spontaneous onset of puberty, defined as testicular size \> 4ml

Sponsors & Collaborators

• Thomas Jefferson University: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (1)

Thomas Jefferson University, Department of Pediatrics, 1025 Walnut Street, Suite 726

Philadelphia, Pennsylvania, 19107, United States

Location

Related Publications (3)

• Davis SM, Lahlou N, Cox-Martin M, Kowal K, Zeitler PS, Ross JL. Oxandrolone Treatment Results in an Increased Risk of Gonadarche in Prepubertal Boys With Klinefelter Syndrome. J Clin Endocrinol Metab. 2018 Sep 1;103(9):3449-3455. doi: 10.1210/jc.2018-00682.

  PMID: 29931143 DERIVED

• Ross JL, Kushner H, Kowal K, Bardsley M, Davis S, Reiss AL, Tartaglia N, Roeltgen D. Androgen Treatment Effects on Motor Function, Cognition, and Behavior in Boys with Klinefelter Syndrome. J Pediatr. 2017 Jun;185:193-199.e4. doi: 10.1016/j.jpeds.2017.02.036. Epub 2017 Mar 10.

  PMID: 28285751 DERIVED

• Davis S, Lahlou N, Bardsley M, Temple MC, Kowal K, Pyle L, Zeitler P, Ross J. Gonadal function is associated with cardiometabolic health in pre-pubertal boys with Klinefelter syndrome. Andrology. 2016 Nov;4(6):1169-1177. doi: 10.1111/andr.12275. Epub 2016 Sep 16.

  PMID: 27637014 DERIVED

MeSH Terms

Conditions

Klinefelter Syndrome

Condition Hierarchy (Ancestors)

Sex Chromosome Disorders of Sex Development; Disorders of Sex Development; Urogenital Abnormalities; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Gonadal Disorders; Endocrine System Diseases; Hypogonadism

Results Point of Contact

• Title: Judith L. Ross, M.D.
• Organization: Thomas Jefferson University

judith.ross@jefferson.edu

215-955-1648

Study Officials

• Judith L. Ross, M.D.

  Thomas Jefferson University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 3, 2006
• First Posted: July 6, 2006
• Study Start: July 1, 2006
• Primary Completion: November 30, 2017
• Study Completion: November 30, 2017
• Last Updated: July 8, 2021
• Results First Posted: July 8, 2021

Record last verified: 2021-07

Locations

US (1)