Treatment of Children and Adolescents With Growth Failure Associated With Primary Insulin-Like Growth Factor-1 (IGF-1) Deficiency

Condition(s) targeted: Primary IGF-1 Deficiency; Growth Disorders

Intervention: rh IGF-1 (mecasermin) (Drug); rh IGF-1 mecasermin (Drug)

Phase: Phase 3

Status: Recruiting

Sponsored by: Tercica

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

Overall contact:
Rod Van Syoc, Phone: 650 238 1598, Email: rodney.vansyoc@tercica.com

This is an extension study to Tercica study MS301 and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and adolescents treated for primary IGF-1 deficiency (IGFD). The secondary objective is to use the data collected to learn more about the relationship of IGF-1 exposure to the promotion of normal growth and pubertal development.

Official title: Recombinant Human Insulin-Like Growth Factor-1 (IGF-1) Treatment of Children With Growth Failure Associated With Primary IGF-1 Deficiency: An Open-Label, Multi-Center, Extension Study

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

Primary outcome: Height Velocity

Secondary outcome:

Height velocities during subsequent years of rh IGF-1 treatment

Height velocity standard deviation (SD) score

Height SD score

Detailed description: Primary IGFD is a term that has been used to describe patients with intrinsic cellular defects in GH action. In this protocol, subjects that have completed one year of mecasermin treatment on Tercica protocol MS301 will be allow to enroll in this extension study. All subjects will receive treatment.

This is a Phase IIIb open-label, multi-center, parallel dose, extension study conducted in approximately 40 centers across the United States.

Minimum age: 4 Years. Maximum age: 15 Years. Gender(s): Both.

Criteria:

Inclusion Criteria:

- Parents or legally authorized representatives must give signed informed consent before

any trial related activities are conducted

- Where required, assent of the subject will be appropriately documented prior to any

study related activities

- Completion of assessments at Visit 9 (Month 120 of Study MS301)

Exclusion Criteria:

- Incomplete participation in MS301

- Known or suspected allergy to the trial product (mecasermin, recombinant human IGF-1

injection) or its formulation

- Development or presence of a chronic condition except as approved by the Medical

Monitor

- Pregnancy

- Any social or medical condition that, in the opinion of the investigator, would be

detrimental to either the subject or the study

Rod Van Syoc, Phone: 650 238 1598, Email: rodney.vansyoc@tercica.com

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

Starting date: November 2005
Ending date: December 2009
Last updated: August 4, 2008