IGF-I/IGFBP-3 Therapy in Children and Adolescents With Growth Hormone Insenitivity Syndrome (GHIS) Such as Laron Syndrome
Summary
STUDY OBJECTIVE To evaluate the safety, tolerability, and efficacy, as growth velocity (statural growth), of rhIGF-I/rhIGFBP-3 administered for 12 months in pre-pubertal children and adolescents with GHIS. STUDY DESIGN This study is an open-label, multi-center clinical trial to evaluate the safety and effectiveness of rhIGF-I/rhIGFBP-3 to increase rate of growth when administered once daily for 12 months in children and adolescents with growth hormone insensitivity syndrome (GHIS) such as Laron Syndrome. At the end of the initial twelve-month treatment period, additional safety and long-term efficacy data will be assessed in a second 12 month treatment period.
Timeline
- Start
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- Primary completion
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- Completion
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Outcome
Met primary endpoint
release “evaluating SomatoKine(R) (rhIGF-I/rhIGFBP-3) in children with severe short stature due to growth hormone insensitivity syndrome (GHIS). The six-month data analysis showed a statistically significant increase” insmed.com ↗
release “GH insensitivity syndrome (GHIS). The primary endpoint of change in height velocity after 6 months on treatment was achieved and was highly statistically significant” insmed.com ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | rhIGF-I/rhIGFBP-3 | Unknown | — | — |