Clinical trial NCT00125164: Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency
Official title: Recombinant Human Insulin-Like Growth Factor (rhIGF-1) Treatment of Prepubertal Children With Growth Failure Associated With Primary IGF-1 Deficiency: A Phase 3, Randomized, Open Label, Observation-Controlled, Multicenter, Parallel-Dose Comparison Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Growth Disorders
Insulin-Like Growth Factor-1 Deficiency
Keywords: Primary IGF-1 Deficiency
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2005-07-29
Enrollment: 137
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.

Detailed description: Prepubertal growth failure associated with primary IGF-1 deficiency (IGFD). Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action. In this protocol, primary IGFD is defined as short stature (height standard deviation score\[SDS\]\<-2 below the mean for age and gender), low serum IGF-1 (IGF-1 SDS \<-2 below the mean for age and gender), and levels of growth hormone (GH) that are normal (≥7ng/mL) after a GH stimulation test. Primary IGFD is believed to result from a lower than normal ability to produce IGF-1 when exposed to normal levels of GH, i.e., a type of GH insensitivity or GH resistance.

This trial is one year, randomized, open label, observation-controlled, parallel-dose comparison efficacy and safety study conducted in approximately 40 centers across the United States.

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.

Primary outcomes:
measure: Height Velocity During the First Year - Intent to Treat (ITT)Population
timeFrame: Measured at baseline and at one year
Secondary outcomes:
measure: Change From Baseline in Height Standard Deviation (SD) Score at One Year - ITT Population
timeFrame: Measured at baseline and at one year
measure: Changes in Bone Age From Baseline to One Year
timeFrame: Measured at baseline and at one year
measure: Percent Changes From Baseline in Serum Concentrations of IGF-1 at One Year
timeFrame: Measured at baseline and at one year
measure: Percent Changes From Baseline in Serum Concentrations of IGF-2 at One Year
timeFrame: Measured at baseline and at one year
measure: Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-2 (IGFBP-2) at One Year
timeFrame: Measured at baseline and at one year
measure: Percent Changes From Baseline in Serum Concentrations of Insulin-like Growth Factor Binding Protein-3 (IGFBP-3) at One Year
timeFrame: Measured at baseline and at one year
measure: IGF Generation Test: Change of Serum IGF-1 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)
timeFrame: Study Day 1 and Day 7
measure: IGF Generation Test: Change of Serum IGFBP-3 After 7 Days Exposure to Recombinant Human Growth Hormone (rhGH)
timeFrame: Study Day 1 and Day 7
Other outcomes:
Locations:
city: Brisbane
country: United States
facility: Ipsen (formerly Tercica)
state: California
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: