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

This study has been completed.
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Mark Borchert, M.D., Children's Hospital Los Angeles
ClinicalTrials.gov Identifier:
NCT00140413
First received: August 31, 2005
Last updated: May 27, 2014
Last verified: May 2014
  Purpose

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.

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

Resource links provided by NLM:


Further study details as provided by Children's Hospital Los Angeles:

Primary Outcome Measures:
  • Prevalence and type of endocrinopathies at baseline; growth and obesity at two years [ Time Frame: Study completion ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Endocrine outcomes as correlated with radiographic, ocular, and developmental findings [ Time Frame: Study completion ] [ Designated as safety issue: No ]

Enrollment: 20
Study Start Date: December 2004
Study Completion Date: February 2014
Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Nutropin AQ
    Daily injection. Dosage dependent on weight.
Detailed Description:

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
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00140413

Locations
United States, California
Childrens Hospital Los Angeles
Los Angeles, California, United States, 90027
Sponsors and Collaborators
Children's Hospital Los Angeles
Genentech, Inc.
Investigators
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: May 27, 2014
Health Authority: United States: Institutional Review Board

Keywords provided by Children's Hospital Los Angeles:
Optic Nerve Hypoplasia

Additional relevant MeSH terms:
Endocrine System Diseases
Dwarfism, Pituitary
Hypopituitarism
Septo-Optic Dysplasia
Dwarfism
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Bone Diseases, Endocrine
Pituitary Diseases
Hypothalamic Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Nervous System Malformations
Congenital Abnormalities

ClinicalTrials.gov processed this record on October 19, 2014