rhGH and rhIGF-1 Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency

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

Intervention: somatropin (Drug); mecasermin and somatropin (Drug); mecasermin and somatropin (Drug); mecasermin and somatropin (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Tercica

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

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

IGF-1 (insulin-like growth factor-1) is a hormone that is normally produced in the body in response to another hormone called growth hormone. Growth Hormone is produced by a small gland at the base of the brain (the pituitary). Together IGF-1 and GH are large contributors to growth during infancy, childhood, and adolescence.

Children with IGF Deficiency are short and have an imbalance in the levels of growth hormone and IGF-1 that their body produces. Their growth hormone levels are normal or even high, but IGF-1 levels do not increase normally in response to growth hormone. As a result, they have a type of growth hormone insensitivity and an inability to grow normally.

This study is a test to see whether daily dosing with a combination of rhIGF-1 and rhGH will help children with IGFD grow taller more quickly than children treated with rhGH alone. The study medications, rhIGF-1 and rhGH, are approved by the US Food and Drug Administration (FDA) for use in some growth disorders in children, but the combination of rhIGF-1 and rhGH in children with IGF-1 deficiency (IGFD) is investigational.

Official title: Recombinant Human Growth Hormone (rhGH) and Recombinant Human Insulin-Like Growth Factor-1 rhIGF-1) Combination Therapy in Children With Short Stature Associated With IGF-1 Deficiency: A Three-Year, Randomized, Multi-Center, Open-Label, Parallel-Group, Active Treatment Controlled, Dose Selection Trial

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

Primary outcome: Height velocity

Secondary outcome:

Change in height SDS

Safety assessments including: physical exam, funduscopic exams, adverse events, safety and metabolic labs and anti-GH and anti-IGF-1 antibodies

Changes in serum concentrations of GH, IGF-1, IGFBP-1, IGFPB-3, ALS and GHBP

Change in bone age

Changes in adipokines

Minimum age: 5 Years. Maximum age: N/A. Gender(s): Both.

Criteria:

Inclusion Criteria:

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

any trial-related activities

- IGF-1 SDS of ≤ -1 for age and gender

- Short stature, as defined by a height SDS of ≤ -2 for age and gender

- Chronological age ≥ 5 years

- Bone age ≤ 11 years in boys and ≤ 9 years in girls

- GH sufficiency, defined as a maximal stimulated GH response of greater than or equal

to 10 ng/mL at Visit 2 (note: upon approval of the Medical Monitor, the result of a prior GH stimulation test may satisfy this requirement).

- Prepubertal status

- Adequate nutrition as evidenced by a body mass index (BMI) greater than or equal to

the 5th percentile for age and gender

Exclusion Criteria:

- Severe Primary IGFD (defined as height and IGF-1 SDS ≤ 3, and stimulated GH response

greater than or equal to 10 ng/mL)

- Prior or current use of medications with the potential to alter growth patterns

including GH, IGF-1, IGFBP-3, gonadotrophin agonists (e. g., Lupron), aromatase inhibitors, androgens and estrogens

- Known or suspected allergy to rhGH, rhIGF-1 or a constituent of their formulations

- Current use of medications for attention deficit disorder

- A chronic health condition that requires anti-inflammatory steroids or daily

medication unless approved by the Medical Monitor

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

Tercica Inc., Brisbane, California 94005, United States; Recruiting
George M Bright, MD, Principal Investigator

Company Trials Summary

Starting date: December 2007
Ending date: December 2011
Last updated: August 27, 2008