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

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ClinicalTrials.gov Identifier: NCT01066052
Recruitment Status : Completed
First Posted : February 10, 2010
Results First Posted : December 14, 2018
Last Update Posted : December 14, 2018
Sponsor:
Information provided by (Responsible Party):
Merck KGaA, Darmstadt, Germany

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.

Condition or disease Intervention/treatment Phase
Turner's Syndrome Drug: r-hGH Phase 4

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 115 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : February 29, 1992
Actual Primary Completion Date : March 31, 1999
Actual Study Completion Date : August 31, 2010


Arm Intervention/treatment
Experimental: r-hGH
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
Subcutaneous administration.
Other Name: Saizen®

No Intervention: Historical Control
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.



Primary Outcome Measures :
  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 Outcome Measures :
  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.



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Ages Eligible for Study:   up to 4 Years   (Child)
Sexes Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01066052


Locations
France
Hôpital Robert Debré
Paris, France, 75935
Sponsors and Collaborators
Merck KGaA, Darmstadt, Germany
Investigators
Study Director: Medical Responsible Merck KGaA, Darmstadt, Germany

Publications of Results:
Responsible Party: Merck KGaA, Darmstadt, Germany
ClinicalTrials.gov Identifier: NCT01066052     History of Changes
Other Study ID Numbers: GF 5834
First Posted: February 10, 2010    Key Record Dates
Results First Posted: December 14, 2018
Last Update Posted: December 14, 2018
Last Verified: June 2018

Keywords provided by Merck KGaA, Darmstadt, Germany:
Growth hormone
Turner syndrome
r-hGH
early treatment

Additional relevant MeSH terms:
Syndrome
Turner Syndrome
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Dwarfism
Disease
Pathologic Processes
Disorders of Sex Development
Urogenital Abnormalities
Sex Chromosome Disorders of Sex Development
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Heart Diseases
Congenital Abnormalities
Sex Chromosome Disorders
Chromosome Disorders
Genetic Diseases, Inborn
Gonadal Disorders
Endocrine System Diseases
Ovarian Diseases
Adnexal Diseases
Genital Diseases, Female
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs