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
|Condition or disease||Intervention/treatment||Phase|
|Turner's Syndrome||Drug: r-hGH||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||115 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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|
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.
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.
- 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.
- 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.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01066052
|Hôpital Robert Debré|
|Paris, France, 75935|
|Study Director:||Medical Responsible||Merck KGaA, Darmstadt, Germany|