Phase 3
Completed N=92
Long-Term Treatment With rhIGF-1 in GHIS
Growth Hormone Insensitivity Syndrome
Source: ClinicalTrials.gov NCT00571727 ↗
Enrolled (actual)
92
Serious AEs
19.6%
Results posted
Jul 2023
Primary outcomePrimary: Annualized Height Velocity Up to 12 Years — 2.6; 8.0; 5.9; 5.5 centimeter per year (cm/y)
◆ Published Evidence
Highly cited
246citations · ~13 / year
Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity.
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).
Linked Publications
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Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity.
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 | — |
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) and the linked publication. 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. Informational only — not medical advice.