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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

This study has been completed.
Sponsor:
Information provided by:
Merck KGaA
ClinicalTrials.gov Identifier:
NCT01066052
First received: February 5, 2010
Last updated: August 12, 2011
Last verified: August 2011
History of Changes
  Purpose

This study was conducted to evaluate the efficacy and safety of recombinant human growth hormone (r-hGH) treatment in young girls with Turner Syndrome.


Condition Intervention Phase
Turner's Syndrome
 Other: 0.15 IU/kg/day GH
Other: 0.1 IU/kg/day GH
 Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: 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 "

Resource links provided by NLM:

Drug Information available for: Somatropin
U.S. FDA Resources

Further study details as provided by Merck KGaA:

Primary Outcome Measures:
  • Height gain at 2 year [ Time Frame: At least after 2 years of treatment (initial interventional study), then it was agreed to follow-up patients until end of treatment (non-interventional FU) and if feasible to collect final height ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Tolerability criteria (Clinical): general and local tolerability (collection of AEs/SAEs) [ Time Frame: Within first 2 years of treatment (initial interventional study), then it was agreed to follow-up patients until end of treatment (non-interventional FU) and to collect potential SAEs until end of treatment ] [ Designated as safety issue: Yes ]
  • Tolerability criteria (Metabolic): HBA1c [ Time Frame: Every 6 month within first 2 years of treatment (initial interventional study), then it was agreed to follow-up patients until end of treatment (non-interventional FU) ] [ Designated as safety issue: No ]
  • Tolerability criteria (Radiologic): Bone age [ Time Frame: Every 6 month within first 2 years of treatment (initial interventional study), then it was agreed to follow-up patients until end of treatment (non-interventional FU) ] [ Designated as safety issue: No ]
  • Tolerability criteria (Immunologic): Anti-GH and anti non-hGH proteins antibodies [ Time Frame: Systematically at 3, 6, 12 and 24 months and at 9, 12, 18 and 36 months if positive result(s) at 6 month then it was agreed to follow-up patients until end of treatment (non-interventional FU) ] [ Designated as safety issue: No ]
  • Tolerability criteria: IGF1 [ Time Frame: Within first 2 years of treatment (initial interventional study), then it was agreed to follow-up patients until end of treatment (non-interventional FU) ] [ Designated as safety issue: No ]
  • Efficacy criteria: Height and weight evolution [ Time Frame: Within first 2 years of treatment (initial interventional study), then it was agreed to follow-up patients until end of treatment (non-interventional FU) ] [ Designated as safety issue: No ]

Enrollment: 64
Study Start Date: February 1992
Study Completion Date: August 2010
Primary Completion Date: March 1999 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Group Q
Subjects with height <-2SD / standard height (sempé cohort) received 0.15 IU/kg/day
 Other: 0.15 IU/kg/day GH
Group Q = 0.15 IU/kg/day (was interventional at the time of protocol writing)
Group U
Subjects with height <-1 SD and >-2SD / standard height (sempé cohort) received 0.1 IU/kg/day
 Other: 0.1 IU/kg/day GH
Group U = 0.1 IU/kg/day (was interventional at the time of protocol writing)

Detailed Description:

The study started as an interventional clinical trial. Following primary completion of the trial, the sponsor together with the investigators decided that it was interesting to perform an observational follow up of the patients, who participated in the interventional part of the study, in order to retrieve more outcome results and safety information on this cohort of young turner patients for whom treatment started before the age of 4. The follow up period was then to be performed until end of GH treatment as decided by the treating physician according to usual clinical practice. More recently, it was agreed to also collect information on final adult height and weight for patients not lost to follow up.

  Eligibility

Ages Eligible for Study:   up to 4 Years
Genders Eligible for Study:   Female
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Young girls with turner syndrome proved by caryotype
  • Age < 4 years old
  • GH secretion confirmed with ornithin stimulation test
  • Normal glucidic metabolism confirmed by assessment of glycosylated haemoglobin (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
  • Signed informed consent

Exclusion Criteria:

  • Severe associated pathology with impact on growth
  • Concomitant treatment with impact on growth
  • Previous or associated treatment with anabolic steroids
  • Associated GH deficiency
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01066052

Sponsors and Collaborators
Merck KGaA
Investigators
Principal Investigator: Jean-Claude Carel, MD, Professor Endocrinologie Diabétologie Pédiatrique & INSERM U690, Université Paris 7 Denis Diderot, Hôpital Robert Debré, 48, boulevard Sérurier, 75935 Paris cedex 19, France (email id: jean-claude.carel@inserm.fr)
  More Information

Publications:

Responsible Party: Medical Responsible, Merck Serono s.a.s., France, an affiliate of Merck KGaA, Darmstadt, Germany
ClinicalTrials.gov Identifier: NCT01066052     History of Changes
Other Study ID Numbers: GF 5834
Study First Received: February 5, 2010
Last Updated: August 12, 2011
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

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

Additional relevant MeSH terms:
Dwarfism
Turner Syndrome
Gonadal Dysgenesis
Primary Ovarian Insufficiency
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Endocrine System Diseases
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
Gonadal Disorders
Ovarian Diseases
Adnexal Diseases
Genital Diseases, Female
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 16, 2013