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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

Tracking Information
First Submitted Date  ICMJE February 5, 2010
First Posted Date  ICMJE February 10, 2010
Results First Submitted Date  ICMJE September 7, 2017
Results First Posted Date  ICMJE December 14, 2018
Last Update Posted Date December 14, 2018
Actual Study Start Date  ICMJE February 29, 1992
Actual Primary Completion Date March 31, 1999   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 12, 2018)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: February 9, 2010)
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 ]
Change History Complete list of historical versions of study NCT01066052 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: June 12, 2018)
  • 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.
  • 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.
  • Number of Participants With Anti r-hGH Antibodies [ Time Frame: Baseline up to Year 2 ]
  • 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.
  • 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.
Original Secondary Outcome Measures  ICMJE
 (submitted: February 9, 2010)
  • 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 ]
  • 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) ]
  • 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) ]
  • 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) ]
  • 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) ]
  • 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) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Turner's Syndrome
Intervention  ICMJE Drug: r-hGH
Subcutaneous administration.
Other Name: Saizen®
Study Arms  ICMJE
  • 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.
    Intervention: Drug: r-hGH
  • 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.
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 12, 2018)
115
Original Actual Enrollment  ICMJE
 (submitted: February 9, 2010)
64
Actual Study Completion Date  ICMJE August 31, 2010
Actual Primary Completion Date March 31, 1999   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Female
Ages  ICMJE up to 4 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01066052
Other Study ID Numbers  ICMJE GF 5834
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Merck KGaA, Darmstadt, Germany
Study Sponsor  ICMJE Merck KGaA, Darmstadt, Germany
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Responsible Merck KGaA, Darmstadt, Germany
PRS Account Merck KGaA, Darmstadt, Germany
Verification Date June 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP