Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years

NCT01066052 | Completed Phase 4 Results

• 1 other identifier: GF 5834
• Study Type: interventional
• Target: 115
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this study is to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in girls with Turner Syndrome under the age of 4 years. After 4 years of treatment, height in these girls will be compared with an historical control group of untreated girls with Turner Syndrome, matched for age and height at baseline.

Conditions

Turner's Syndrome

Keywords

Growth hormone; Turner syndrome; r-hGH; early treatment

Outcome Measures

Primary Outcomes (1)

• Height SDS at Year 4

  Height SDS was calculated as height minus reference mean height divided by standard deviation of the reference population. Height SDS reflects the height relative to a reference population of the same age and gender.

  Year 4

Secondary Outcomes (5)

• Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels

  Baseline up to Year 2

• Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)

  Baseline, Year 1, Year 2

• Number of Participants With Anti r-hGH Antibodies

  Baseline up to Year 2

• Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels

  Baseline up to Year 2

• Number of Participants Who Reached Normal Height at Year 4

  Year 4

Study Arms (2)

r-hGH

EXPERIMENTAL

Participants (girls) will receive r-hGH as a subcutaneous injection administered by a parent in the evening. During Years 1-2, the dose of r-hGH received will depend on participants' baseline height standard deviation score (SDS) relative to the general population standard: participants with a height SDS of -2 standard deviation (SD) or lower will receive 0.05 milligrams per kilogram (mg/kg) per day r-hGH and those with a height SDS between -1 and -2 SD will receive 0.035 mg/kg per day r-hGH. After 2 years of treatment, all participants will receive a fixed dose of 0.05 mg/kg per day for a further 2 years.

Drug: r-hGH

Historical Control

NO INTERVENTION

This arm will include matching (age and height) historical control participants (girls) with turner syndrome, who were born between 1961 and 1990 and were untreated.

Interventions

r-hGH DRUG

Subcutaneous administration.

Also known as: Saizen®

r-hGH

Eligibility Criteria

• Age: Up to 4 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Young girls with turner syndrome proved by karyotype
• Growth hormone secretion confirmed with ornithin stimulation test
• Normal glucidic metabolism confirmed by assessment of HbA1c
• None associated severe pathology which could have impact on growth (i.e. renal insufficiency, decompensated heart failure)
• No previous or associated treatment with anabolic or sexual steroids
• Known parental height

You may not qualify if:

• Severe associated pathology with impact on growth
• Concomitant treatment with impact on growth
• Previous or associated treatment with anabolic steroids
• Associated growth hormone deficiency

Sponsors & Collaborators

• Merck KGaA, Darmstadt, Germany: lead

Study Sites (1)

Hôpital Robert Debré

Paris, 75935, France

Location

Related Publications (1)

• Linglart A, Cabrol S, Berlier P, Stuckens C, Wagner K, de Kerdanet M, Limoni C, Carel JC, Chaussain JL; French Collaborative Young Turner Study Group. Growth hormone treatment before the age of 4 years prevents short stature in young girls with Turner syndrome. Eur J Endocrinol. 2011 Jun;164(6):891-7. doi: 10.1530/EJE-10-1048. Epub 2011 Mar 11.

  PMID: 21398400 RESULT

MeSH Terms

Conditions

Turner Syndrome

Interventions

Human Growth Hormone

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Growth Hormone; Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Merck KGaA Communication Center,
• Organization: Merck Healthcare, a business of Merck KGaA, Darmstadt, Germany

service@merckgroup.com

496151725200

Study Officials

• Medical Responsible

  Merck KGaA, Darmstadt, Germany

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 5, 2010
• First Posted: February 10, 2010
• Study Start: February 29, 1992
• Primary Completion: March 31, 1999
• Study Completion: August 31, 2010
• Last Updated: December 14, 2018
• Results First Posted: December 14, 2018

Record last verified: 2018-06

Locations

FR (1)