Prepubertal Children With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency

Condition(s) targeted: Growth Disorders; Insulin-Like Growth Factor-1 Deficiency

Intervention: rhIGF-1 (mecasermin, Tercica, Inc.) for a period of 1 year (Drug)

Phase: Phase 3

Status: Active, not recruiting

Sponsored by: Tercica

Official(s) and/or principal investigator(s):
George Bright, M.D., Study Director, Affiliation: Tercica, Inc.

This study is intended to determine whether dosing with recombinant human insulin-like growth factor (rhIGF-1) will safely and effectively increase the growth of prepubertal children 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.

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 design: Treatment, Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety/Efficacy Study

Primary outcome:

Height velocity during the first year

Change in height standard deviation (SD) score

Secondary outcome:

Changes in serum concentrations of IGF-1, IGF-2, insulin-like growth factor binding protein-2 (IGFBP-2) and insulin-like growth factor binding protein-3 (IGFBP-3) during the course of the study

Changes in bone age over one year

IGF generation test: serum IGF-1 and IGFBP-3 changes after 7 days exposure to recombinant human growth hormone (rhGH)

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 (<-2 standard deviations [SDs] below the mean for age and gender), abnormal serum IGF-1 (<-2 SDS 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 of GH resistance.

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

Minimum age: 3 Years. Maximum age: 11 Years. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Ages 3-10 years inclusive for girls; ages 3-11 years inclusive for boys

- Prepubertal

- Height SD score of < -2

- IGF-1 SD score of < -2

Exclusion Criteria:

- Prior treatment with GH, IGF-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.

Tercica, Inc., Brisbane, California 94005, United States

Overview of clinical trial information

Starting date: October 2004
Ending date: September 2008
Last updated: June 11, 2008