Growth Hormone Insensitivity Syndrome (GHIS), Laron Syndrome
Conditions
Brief 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.
Interventions
Sponsors
Insmed Incorporated
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Inclusion criteria
1. A diagnosis of GHIS such as Laron syndrome, 2. 2 - 18 years of age, 3. Height less than or equal to -3SD for age, 4. Pre-pubertal, defined as Tanner breast stage 1 or testis volume \<4mL
Exclusion criteria
1. Children in puberty, 2. Diagnosed malignancy, 3. A diagnosis of diabetes mellitus
Countries
Argentina, Australia, Brazil, China, Egypt, Germany, Israel, Italy, Norway, Peru, Slovakia, Turkey (Türkiye), United Kingdom, United States
Outcome results
None listed