Phase 4
N=115
Growth Hormone Treatment for the Prevention of Short Stature in Young Girls With Turner Syndrome Before the Age of 4 Years
Turner's Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01066052 ↗Enrolled (actual)
115
Serious AEs
13.1%
Results posted
Dec 2018
Primary outcome: Primary: Height SDS at Year 4 — -1.35; -2.44 standard deviation score
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- r-hGH (Drug)
- Age
- Pediatric
- Sex
- Female
- Sponsor
- Merck KGaA, Darmstadt, Germany
- Primary completion
- Mar 1999
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Height SDS at Year 4 |
-1.35; -2.44 | — |
| SECONDARY Number of Participants With Abnormal Glycated Hemoglobin (HbA1c) Levels |
1 | — |
| SECONDARY Difference Between Bone Age (BA) and Chronological Age (CA) (BA-CA) |
-8.9; -8.7; -8.1 | — |
| SECONDARY Number of Participants With Anti r-hGH Antibodies |
— | — |
| SECONDARY Number of Participants With Abnormal Insulin-Like Growth Factor 1 (IGF1) Levels |
46 | — |
| SECONDARY Number of Participants Who Reached Normal Height at Year 4 |
49 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT01066052). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.