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Phase 3 Clinical Trial Initiated for RhIGF-1, Treatment for Primary IGF-1 Deficiency
"For more than 40 years, endocrinologists have been limited to growth hormone treatment for children with short stature," said Daniel E. Hale, M.D., Department of Pediatrics, University of Texas Health Science Center at San Antonio and investigator in the Phase IIIb study. "Now, based on new estimates of the number of children who are resistant to growth hormone and the availability of rhIGF-1 for clinical trials, we plan to evaluate the potential benefit of rhIGF-1 therapy in children with growth failure caused by Primary IGFD."
The randomized, multi-center Phase IIIb study of rhIGF-1 will enroll approximately 160 pre-pubertal children with Primary IGFD whose height and serum IGF-1 levels are more than two standard deviations below normal. The study is designed to evaluate the safety and efficacy of rhIGF-1 in promoting statural growth. The primary endpoint of the study is change in height standard deviation (SD) score over one year. The study is expected to take approximately two years to complete.
"This study, conducted in partnership with pediatric endocrinology investigators from over 30 pediatric endocrine centers, will build upon the positive results from Tercica's Phase III clinical trial of rhIGF-1 in children with more severe IGF-1 deficiency," said John A. Scarlett, MD, President and Chief Executive Officer of Tercica. "If successful, the study results will be the basis of a future regulatory filing."
Earlier this year, Tercica reported positive Phase III study results of the use of rhIGF-1 in Severe Primary IGFD. Results of the clinical study demonstrated a statistically significant increase (p
About Short Stature, Primary IGFD and Severe Primary IGFD
Short stature is medically defined as occurring in approximately 2.5 percent of all children. Children with short stature have a height significantly below the normal range for their age and gender.
Primary IGFD is diagnosed in children who have normal or elevated secretion of endogenous growth hormone and whose height and serum IGF-1 levels are more than two standard deviations below normal. A sub-set of these children, whose height and serum IGF-1 levels are more than three standard deviations below normal, are diagnosed with Severe Primary IGFD. An estimated 6,000 of the 30,000 children with Primary IGFD in the United States have Severe Primary IGFD.
Primary IGFD is typically caused by abnormalities of either the growth hormone receptor or growth hormone signaling pathway. Primary IGFD can lead, in children and adults, to a range of other metabolic disorders affecting long-term health. These metabolic disorders can include lipid abnormalities, decreased bone density, obesity, insulin resistance and cardiovascular disorders.
Source: Tercica, Inc.