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

NCT00140413 | Completed Phase 4 Results

• 1 other identifier: 03.261
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Hypotheses:

1. 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. 2.Early treatment of children with ONH and GH-deficiency can prevent adverse outcomes.
3. 3.Determine the prevalence and types of endocrinopathies in children diagnosed with ONH.
4. 4.Correlate endocrine outcome with radiographic, ocular, and developmental findings in children with ONH.
5. 5.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.
6. 6.Compare growth outcomes between children with isolated GH-deficiency and those with multiple hormone deficiencies.

Conditions

Growth Hormone Deficiency; Septo-Optic Dysplasia; Hypopituitarism

Keywords

Optic Nerve Hypoplasia

Outcome Measures

Primary Outcomes (1)

• 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) 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).

  Baseline and 36 months

Study Arms (2)

Treatment Group 1: Receiving GH Treatment

EXPERIMENTAL

Treatment group assignment was based on subject's stature SDS relative to the mid-parental target height (MPTH) at baseline and subsequent classification as growth deceleration or normal growth. Subjects with growth deceleration were assigned to the GH treatment group in accordance with standard of care. Subjects with normal growth were randomized to treatment or to control (no intervention). The intervention was Nutropin AQ. The starting dose was calculated as 0.3 mg/kg/wk and subsequently modified based on observed length/height velocity and serum IGF-I levels.

Drug: Nutropin AQ

Treatment Group 2: Control

NO INTERVENTION

Treatment group assignment was based on subject's stature SDS relative to the mid-parental target height (MPTH) at baseline and subsequent classification as growth deceleration or normal growth. Subjects with normal growth were randomized to treatment or to control. The control group received no intervention; however, control subjects were switched (crossed over) to the GH replacement group if, during the course of the study, they met criteria for growth deceleration.

Interventions

Nutropin AQ DRUG

Daily injection. Dosage dependent on weight.

Treatment Group 1: Receiving GH Treatment

Eligibility Criteria

• Age: Up to 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital Los Angeles: lead
• Genentech, Inc.: collaborator

Study Sites (1)

Childrens Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

MeSH Terms

Conditions

Dwarfism, Pituitary; Septo-Optic Dysplasia; Hypopituitarism; Optic Nerve Hypoplasia

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Dwarfism; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Bone Diseases, Endocrine; Pituitary Diseases; Hypothalamic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Endocrine System Diseases; Congenital Cranial Dysinnervation Disorders; Oculomotor Nerve Diseases; Ocular Motility Disorders; Cranial Nerve Diseases; Agenesis of Corpus Callosum; Nervous System Malformations; Eye Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Optic Nerve Diseases; Eye Diseases, Hereditary; Genetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Dr. Mark Borchert
• Organization: Children's Hospital Los Angeles

mborchert@chla.usc.edu

323-361-6219

Study Officials

• Mark Borchert, MD

  Childrens Hospital Los Angeles; University of Southern California

  PRINCIPAL INVESTIGATOR

• Mitchell Geffner, MD

  Children's Hospital Los Angeles

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Pediatric Neuro-Ophthalmologist

Study Record Dates

• First Submitted: August 31, 2005
• First Posted: September 1, 2005
• Study Start: December 1, 2004
• Primary Completion: February 1, 2014
• Study Completion: February 1, 2014
• Last Updated: April 1, 2015
• Results First Posted: April 1, 2015

Record last verified: 2015-03

Locations

US (1)