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

Growth Hormone Deficiency Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00140413
• Other study ID #: 03.261
• Status: Completed
• Phase: Phase 4
• First received: August 31, 2005
• Last updated: March 27, 2015
• Start date: December 2004
• Est. completion date: February 2014

Study information

• Verified date: March 2015
• Source: Children's Hospital Los Angeles
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Description:

Subjects for this study will be recruited from active and newly enrolled subjects in our larger ONH study. The study duration is three 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 control (no intervention; observation only).

Subjects assigned or 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 assigned or randomized to growth hormone will have laboratory testing of thyroid, IGF-1 and IGFBP-3 hormones, and fasting lipid levels.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: February 2014
• Est. primary completion date: February 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 5 Years

Eligibility

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.

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency
• Hypopituitarism
• Septo-Optic Dysplasia

Intervention

Drug:
• Nutropin AQ
Daily injection. Dosage dependent on weight.

Locations

Country	Name	City	State
United States	Childrens Hospital Los Angeles	Los Angeles	California

Sponsors (2)

Lead Sponsor	Collaborator
Children's Hospital Los Angeles	Genentech, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Anthropometric Measures Over Time	Primary outcome measures included change in stature, weight, BMI, and weight-for-stature z-scores over the course of the study. Z-score indicates how many standard deviations an element is from the mean. It is calculated as z = (x - µ) s, where µ is the mean of the population, and s is the standard deviation of the population. A positive z-score indicates a datum above the mean, while a negative z-score indicates a datum below the mean. All z-scores were obtained using Epi Info ™ 3.5.4. (Centers for Disease Control, Atlanta, GA).	Baseline and 36 months	No

External Links

•   Optic Nerve Hypoplasia Research