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. Subjects will be randomized to either an observation arm or to active treatment.

Condition	Intervention	Phase
Insulin-Like Growth Factor-1 Deficiency Primary IGF-1 Deficiency	Drug: rhIGF-1 (mescasermin, Tercica, Inc.) for a period of 1 year	Phase III

Further Study Details: 

Primary Outcomes: Change in height standard deviation (SD) score over 1 year; Change in height velocity from the pretreatment height velocity over 1 year
Secondary Outcomes: 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)
Expected Total Enrollment:  160

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.

Ages Eligible for Study:  3 Years   -   11 Years,  Genders Eligible for Study:  Both

Criteria

Inclusion Criteria:

• Age 3–10 years inclusive for girls; ages 3–11 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.

Please refer to this study by ClinicalTrials.gov identifier  NCT00125164

Thorsten von Stein, M.D., Ph.D.      650 624 4919    thorsten.vonstein@tercica.com
Elizabeth Lawson      650 238 1545    elizabeth.lawson@tercica.com

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

Study chairs or principal investigators

George Bright, M.D.,  Study Director,  Tercica, Inc.   

Study ID Numbers:  MS301
Last Updated:  August 1, 2005
Record first received:  July 29, 2005
ClinicalTrials.gov Identifier:  NCT00125164
Health Authority: United States: Food and Drug Administration
ClinicalTrials.gov processed this record on 2005-08-02