Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia

• Status: Completed
• Trial end date: 2014-02-01
• Target enrollment: 0
• Participant gender: All

Summary

Hypotheses:

1. 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.

2. Early treatment of children with ONH and GH-deficiency can prevent adverse outcomes.

Aims:

1. Determine the prevalence and types of endocrinopathies in children diagnosed with ONH.

2. Correlate endocrine outcome with radiographic, ocular, and developmental findings in children with ONH.

3. 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.

4. Compare growth outcomes between children with isolated GH-deficiency and those with multiple hormone deficiencies.

• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Children's Hospital Los Angeles
• Collaborator: Genentech, Inc.
• Treatments: Hormones

Criteria

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.