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

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.

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Key information

Conditions

Growth Hormone Deficiency Agenesis of Corpus Callosum Bone Diseases Bone Diseases, Developmental Bone Diseases, Endocrine Brain Diseases Central Nervous System Diseases Congenital Abnormalities Congenital Cranial Dysinnervation Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Cranial Nerve Diseases Dwarfism Dwarfism, Pituitary Endocrine System Diseases Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Hypopituitarism Hypothalamic Diseases Musculoskeletal Diseases Nervous System Diseases Nervous System Malformations Ocular Motility Disorders Oculomotor Nerve Diseases Optic Nerve Diseases Optic Nerve Hypoplasia Pituitary Diseases Septo-Optic Dysplasia

Age range

Up to 5 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Nutropin AQ

Drug

Daily injection. Dosage dependent on weight.

Primary outcomes

Change in Anthropometric Measures Over Time

Time frame: Baseline and 36 months

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) σ, where µ is the mean of the population, and σ 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).

Sponsors and collaborators

Lead sponsor

Children's Hospital Los Angeles

Other

Collaborators

Genentech, Inc.

Registry information

Important dates

Study start: 2004
Primary completion: 2014
Study completion: 2014
First posted: Sep 1, 2005
Registry last updated: Apr 1, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed