Phase 3
N=92
Long-Term Treatment With rhIGF-1 in GHIS
Growth Hormone Insensitivity Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00571727 ↗Enrolled (actual)
92
Serious AEs
19.6%
Results posted
Jul 2023
Primary outcome: Primary: Annualized Height Velocity Up to 12 Years — 2.6; 8.0; 5.9; 5.5 centimeter per year (cm/y)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- mecasermin (Drug)
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Ipsen
- Primary completion
- Dec 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annualized Height Velocity Up to 12 Years |
2.6; 8.0; 5.9; 5.5; 5.2; 4.9 | — |
| PRIMARY Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment |
7 | — |
| SECONDARY Height Velocity Standard Deviation Score Up to 12 Years |
-3.4; 1.7; -0.0; -0.1; -0.2; -0.3 | — |
| SECONDARY Height Standard Deviation Score Up to 12 Years |
-6.9; -6.1; -5.6; -5.3; -5.1; -5.0 | — |
| SECONDARY Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height |
13.3 | — |
Summary
The objective of this study was to evaluate the long-term safety and effectiveness of mecasermin (the study drug) in children with growth failure due to severe Primary insulin-like growth factor-1 deficiency (Primary IGFD).
Eligibility Criteria
Inclusion Criteria
- Height <-2SD for age and gender
- IGF-1 <-2SD for age and gender
- Evidence of GH resistance
Exclusion Criteria
- closed epiphyses
- prior active malignancy
- major organ disfunction
- treatment with medications that would diminish growth
- clinically significant cardiac abnormalities
Data sourced from ClinicalTrials.gov (NCT00571727). 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.