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
Overview
- Phase
- Phase 4
- Intervention
- r-hGH
- Conditions
- Turner's Syndrome
- Sponsor
- Merck KGaA, Darmstadt, Germany
- Enrollment
- 115
- Locations
- 1
- Primary Endpoint
- Height SDS at Year 4
- Status
- Completed
- Last Updated
- 7 years ago
Overview
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.
Investigators
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
Arms & Interventions
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: r-hGH
Outcomes
Primary Outcomes
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 Outcomes
- Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA)(Baseline, Year 1, Year 2)
- Number of Participants With Anti r-hGH Antibodies(Baseline up to Year 2)
- Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels(Baseline up to Year 2)
- Number of Participants Who Reached Normal Height at Year 4(Year 4)
- Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels(Baseline up to Year 2)