Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia
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Purpose
Hypotheses:
- 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.
Aims:
- 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.
| Condition | Intervention | Phase |
|---|---|---|
|
Growth Hormone Deficiency Septo-Optic Dysplasia Hypopituitarism |
Drug: Nutropin AQ |
Phase 4 |
| Study Type: | Interventional |
| 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 ]
| Estimated Enrollment: | 38 |
| Study Start Date: | December 2004 |
| Estimated Study Completion Date: | December 2014 |
| Estimated Primary Completion Date: | May 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.
Eligibility| Ages Eligible for Study: | up to 5 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- New subjects diagnosed with ONH less than or equal to 2 years of age and subjects actively enrolled (in currently approved prospective ONH study) will be eligible for enrollment.
Contacts and Locations| Contact: Cassandra A Fink, MPH | 323.361.2267 | cfink@chla.usc.edu |
| Contact: Joyce Sutedja | 323.361.6219 | jsutedja@chla.usc.edu |
| United States, California | |
| Childrens Hospital Los Angeles | Recruiting |
| Los Angeles, California, United States, 90027 | |
| Contact: Cassandra A Fink, MPH 323-361-2267 cfink@chla.usc.edu | |
| Principal Investigator: Mark Borchert, MD | |
| Principal Investigator: | Mark Borchert, MD | Childrens Hospital Los Angeles; University of Southern California |
| Principal Investigator: | Mitchell Geffner, MD | Children's Hospital Los Angeles |
More Information
Additional Information:
No publications provided
| Responsible Party: | Mark Borchert, M.D., Pediatric Neuro-Ophthalmologist, Children's Hospital Los Angeles |
| ClinicalTrials.gov Identifier: | NCT00140413 History of Changes |
| Other Study ID Numbers: | 03.261 |
| Study First Received: | August 31, 2005 |
| Last Updated: | February 27, 2012 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by Children's Hospital Los Angeles:
|
Optic Nerve Hypoplasia |
Additional relevant MeSH terms:
|
Pituitary Diseases Dwarfism, Pituitary Hypopituitarism Septo-Optic Dysplasia Endocrine System Diseases Dwarfism Bone Diseases, Developmental Bone Diseases Musculoskeletal Diseases Bone Diseases, Endocrine |
Hypothalamic Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Nervous System Malformations Congenital Abnormalities Hormones Hormones, Hormone Substitutes, and Hormone Antagonists Physiological Effects of Drugs Pharmacologic Actions |
ClinicalTrials.gov processed this record on May 16, 2013