Phase 4
N=458
First Year Growth Response Associated Genetic Markers Validation Phase IV Open-label Study in Growth Hormone Deficient and Turner Syndrome Pre-pubertal Children: the PREDICT Pharmacogenetics Validation Study
Idiopathic Growth Hormone Deficiency · Turner Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT01419249 ↗Enrolled (actual)
458
Serious AEs
0.0%
Results posted
Jan 2014
Primary outcome: Primary: Change From Baseline in Height at Year 1 — 103.6; 103.5; 9.8; 8.6 centimeter
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Blood sampling (Other)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Merck KGaA, Darmstadt, Germany
- Primary completion
- Oct 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Height at Year 1 |
103.6; 103.5; 9.8; 8.6 | — |
| PRIMARY Change From Baseline in Height Standard Deviation Score (SDS) at Year 1 |
-2.60; -2.17; 0.98; 0.71 | — |
| PRIMARY Height Velocity Standard Deviation Score (SDS) at Year 1 |
4.18; 2.59 | — |
| SECONDARY Evaluation of the Contribution of Validated Genetic Markers to the Amplitude of First Year Growth Response to r-hGH Therapy in IGHD Children Using Growth Hormone Deficiency Kabi-Pharmacia International Growth Study (GHD KIGS) Predictive Model |
— | — |
| SECONDARY Evaluation of the Contribution of Validated Genetic Markers to the Amplitude of First Year Growth Response to r-hGH Therapy in TS Girls Using Turner Syndrome Kabi-Pharmacia International Growth Study (TS KIGS) Predictive Model |
— | — |
Summary
PREDICT Validation is a validation pharmacogenetic trial. The purpose of this study is to confirm that some genes can be used to predict how well a subject diagnosed with idiopathic growth hormone deficiency (IGHD) or turner syndrome (TS) will respond to a treatment with recombinant human growth hormone (r-hGH).
Eligibility Criteria
Inclusion Criteria
- Pre-established diagnosis of IGHD or TS based on classical criteria with at least 1 year of r-hGH therapy and with Tanner stage 1 at treatment start
- Retrospective availability of a complete set of clinical, auxological and biological parameters necessary for building the predictive model
- Other protocol defined inclusion criteria could apply
Exclusion Criteria
- Acquired growth hormone deficiency (GHD)
- Any drug or disease that could affect growth during the first year of r-hGH treatment
- Other protocol defined exclusion criteria could apply
Data sourced from ClinicalTrials.gov (NCT01419249). 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.