Back to resultsGrowth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
Primary Purpose
Turner's Syndrome
Status
Completed
Phase
Phase 4
Locations
France
Study Type
Interventional
Intervention
r-hGH
Sponsored by
About this trial
This is an interventional treatment trial for Turner's Syndrome focused on measuring Growth hormone, Turner syndrome, r-hGH, early treatment
Eligibility 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
Sites / Locations
- Hôpital Robert Debré
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
No Intervention
Arm Label
r-hGH
Historical Control
Arm Description
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.
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.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels
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)
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
Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels
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
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.
Full Information
NCT ID
NCT01066052
First Posted
February 5, 2010
Last Updated
June 12, 2018
Sponsor
Merck KGaA, Darmstadt, Germany
1. Study Identification
Unique Protocol Identification Number
NCT01066052
Brief Title
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
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
Study Type
Interventional
2. Study Status
Record Verification Date
June 2018
Overall Recruitment Status
Completed
Study Start Date
February 29, 1992 (Actual)
Primary Completion Date
March 31, 1999 (Actual)
Study Completion Date
August 31, 2010 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Merck KGaA, Darmstadt, Germany
4. Oversight
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Turner's Syndrome
Keywords
Growth hormone, Turner syndrome, r-hGH, early treatment
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
115 (Actual)
8. Arms, Groups, and Interventions
Arm Title
r-hGH
Arm Type
Experimental
Arm Description
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.
Arm Title
Historical Control
Arm Type
No Intervention
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
r-hGH
Other Intervention Name(s)
Saizen®
Intervention Description
Subcutaneous administration.
Primary Outcome Measure Information:
Title
Height SDS at Year 4
Description
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.
Time Frame
Year 4
Secondary Outcome Measure Information:
Title
Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels
Description
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.
Time Frame
Baseline up to Year 2
Title
Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)
Description
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.
Time Frame
Baseline, Year 1, Year 2
Title
Number of Participants With Anti r-hGH Antibodies
Time Frame
Baseline up to Year 2
Title
Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels
Description
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.
Time Frame
Baseline up to Year 2
Title
Number of Participants Who Reached Normal Height at Year 4
Description
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.
Time Frame
Year 4
10. Eligibility
Sex
Female
Maximum Age & Unit of Time
4 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Responsible
Organizational Affiliation
Merck KGaA, Darmstadt, Germany
Official's Role
Study Director
Facility Information:
Facility Name
Hôpital Robert Debré
City
Paris
ZIP/Postal Code
75935
Country
France
12. IPD Sharing Statement
Citations:
PubMed Identifier
21398400
Citation
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.
Results Reference
result
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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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