Phase 3
N=136
Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency
Growth Disorders · Insulin-Like Growth Factor-1 Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT00125164 ↗Enrolled (actual)
136
Serious AEs
6.6%
Results posted
Apr 2010
Primary outcome: Primary: Height Velocity During the First Year - Intent to Treat (ITT)Population — 5.2; 6.9; 7.7 cm/yr — p=<0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- rhIGF-1 (mecasermin, Tercica, Inc.) (Drug)
- Age
- Pediatric · 3+ yrs
- Sex
- All
- Sponsor
- Ipsen
- Primary completion
- Jul 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Height Velocity During the First Year - Intent to Treat (ITT)Population |
5.2; 6.9; 7.7 | <0.0001 sig |
| SECONDARY Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population |
0.02; 0.38; 0.48 | < 0.0001 sig |
| SECONDARY Changes in Bone Age From Baseline to One Year |
0.8; 1.1; 1.2 | — |
| SECONDARY Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year |
64; 227; 266 | — |
| SECONDARY Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year |
14; -38; -37 | — |
| SECONDARY Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-2 (IGFBP-2) at One Year |
-16; 58; 61 | — |
| SECONDARY Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) at One Year |
13; -19; -19 | — |
| SECONDARY IGF Generation Test: Change of Serum IGF-1 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH) |
40; 53; 48 | — |
| SECONDARY IGF Generation Test: Change of Serum IGFBP-3 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH) |
744; 800; 681 | — |
Summary
This study is intended to determine whether twice daily weight based dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children with short stature associated with low IGF-1 levels but who produce sufficient growth hormone (GH). Subjects will be randomized to either an observation arm or to active treatment.
Eligibility Criteria
Inclusion Criteria
- Chronological age ≥ 3 and chronological or bone age less than or equal to 11 years inclusive in girls;
- Chronological age ≥ 3 and chronological or bone age less than or equal to 12 years inclusive for boys
- Prepubertal
- Height SD score of < -2
- IGF-1 SD score of < -2
Exclusion Criteria
- Prior treatment with rhGH, rhIGF-1, or other growth-influencing medications
- Growth failure associated with other identifiable causes (e.g., syndromes, chromosomal abnormality)
- Chronic illness such as diabetes, cystic fibrosis, etc.
Data sourced from ClinicalTrials.gov (NCT00125164). 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.