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Completed

NCT Number: NCT01066052

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

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.

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Key information

Conditions

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

Age range

Up to 4 year

Sex eligibility

Female

Study type

Interventional

Phase

Phase 4

Primary location

Hôpital Robert Debré

Paris, 75935, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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

Exclusion criteria

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

Treatment and study plan

r-hGH

Drug

Subcutaneous administration.

Other names: Saizen®

Primary outcomes

  1. Height SDS at Year 4

    Time frame: 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.

Secondary outcomes

  1. Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels

    Time frame: Baseline up to Year 2

    HbA1c develops when hemoglobin, a protein within red blood cells that carries oxygen throughout the body, joins with glucose in the blood, becoming glycated. The higher the level of glucose in the blood, the higher the level of HbA1c is detectable on red blood cells. The normal range for HbA1c is 4 percent (%) to 5.9%. Number of participants, who had abnormal HbA1c levels any time during the assessment, were reported.

  2. Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)

    Time frame: Baseline, Year 1, Year 2

    BA was determined using left wrist and hand X-ray. CA was determined using the date of birth. Difference of BA and CA (BA-CA) was reported.

  3. Number of Participants With Anti r-hGH Antibodies

    Time frame: Baseline up to Year 2

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

    Time frame: Baseline up to Year 2

    The normal range for IGF1 levels is 45 to 117 nanogram per milliliter (ng/mL) for girls aged less than (<) 3 years and 80 to 236 ng/mL for girls aged 3 to 6 years. Values outside the normal range were considered abnormal. Number of participants, who had abnormal IGF1 levels any time during the assessment, were reported.

  5. Number of Participants Who Reached Normal Height at Year 4

    Time frame: Year 4

    Participants with normal height were those who attained a height which was within +/- 2 height SDS of reference population standard. 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.

Sponsors and collaborators

Lead sponsor

Merck KGaA, Darmstadt, Germany

Industry

Registry information

Official study title

Collaborative Study to Assess the Effects of Treatment With Recombinant Growth Hormone Saizen® in the Prevention of Short Stature in Young Girls Suffering From Turner Syndrome Before the Age of 4 Years. Original French Title: Etude Collaborative Pour apprécier Les Effets du Traitement Par l'Hormone de Croissance Recombinante SAIZEN® Dans le Retard de Croissance de la Fillette Atteinte de Syndrome de Turner Avant l'âge de 4 Ans

Important dates

Study start
1992
Primary completion
1999
Study completion
2010
First posted
Feb 10, 2010
Registry last updated
Dec 14, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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