Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia
- The prevalence of endocrinopathies, and growth hormone (GH) deficiency in particular, among young children diagnosed with optic nerve hypoplasia (ONH) is higher than is commonly thought.
- Early treatment of children with ONH and GH-deficiency can prevent adverse outcomes.
- Determine the prevalence and types of endocrinopathies in children diagnosed with ONH.
- Correlate endocrine outcome with radiographic, ocular, and developmental findings in children with ONH.
- Examine the effect of GH treatment on growth and obesity in children with ONH, GH-deficiency, and either subnormal or normal growth compared to children with ONH that are not GH-deficient.
- Compare growth outcomes between children with isolated GH-deficiency and those with multiple hormone deficiencies.
Growth Hormone Deficiency
Drug: Nutropin AQ
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia|
- Prevalence and type of endocrinopathies at baseline; growth and obesity at two years [ Time Frame: Study completion ] [ Designated as safety issue: No ]
- Endocrine outcomes as correlated with radiographic, ocular, and developmental findings [ Time Frame: Study completion ] [ Designated as safety issue: No ]
|Study Start Date:||December 2004|
|Study Completion Date:||February 2014|
|Primary Completion Date:||February 2014 (Final data collection date for primary outcome measure)|
Drug: Nutropin AQ
Subjects for this study will be recruited from active and newly enrolled subjects in our larger ONH study. The study duration is two years and we anticipate 38 subjects will enroll. Subjects will be recruited for this study if they present with either growth deceleration or at least one subnormal result for IGF-1 or IGFBP-3.
Baseline information collected includes: height, weight, head circumference, examinations by an endocrinologist and ophthalmologist, endocrine laboratory testing, fundus photography, electrophysiology testing, head MRI, and a developmental assessment. A glucagon stimulation test will be performed and subjects who are deemed GH-deficient and who have delayed growth will be assigned to GH treatment, in line with standard clinical practice. Those with normal growth but determined to be GH-deficient by a glucagon stimulation test will be randomized to treatment with GH vs observation only.
Subjects randomized to treatment with GH will be provided with GH for the duration of their participation in the study. Enrolled subjects will return every four months to monitor progress. Subjects will undergo a physical examination at each visit, including height, weight, head circumference, and body fat. In addition, subjects randomized to growth hormone will have laboratory testing of thyroid, IGF-1 and IGFBP-3 hormones, and fasting lipid levels.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00140413
|United States, California|
|Childrens Hospital Los Angeles|
|Los Angeles, California, United States, 90027|
|Principal Investigator:||Mark Borchert, MD||Childrens Hospital Los Angeles; University of Southern California|
|Principal Investigator:||Mitchell Geffner, MD||Children's Hospital Los Angeles|