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

NCTNCT00140413Other id03.261

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Completed
Phase: Phase 4
Enrollment: 20 participants (actual)
Sponsor: Children's Hospital Los Angeles · Other
Design: Randomized · Open label (no masking)
Countries: 1 country
United States
Start: Dec 2004
Primary completion: Feb 2014
Completion: Feb 2014
First posted: 1 Sept 2005 (estimated)
Last update posted: 1 Apr 2015 (estimated)
Conditions: Growth Hormone Deficiency · Septo-Optic Dysplasia · Hypopituitarism

Timeline

1 trial · 1 completed · 1 with posted results

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

200420062008201020122014

Ph 4
Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia
NCT00140413
Dec 2004 to Feb 2014

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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.

Interventions

Nutropin AQDrug

Arms2

Experimental
Treatment Group 1: Receiving GH Treatment
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.
No intervention
Treatment Group 2: Control
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.

Primary outcomes1

Outcomes the sponsor listed as primary.

Change in Anthropometric Measures Over Time
Time frame · Baseline and 36 months
Registry description
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).

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeMedian· Baseline and 36 months

Change in Anthropometric Measures Over Time

Group	Value (Z-score)
Treatment Group 1: Receiving Growth Hormone Treatment	1.97
Treatment Group 2: Control	-0.61
Treatment Group 3: Control Crossed Over to Treatment	1.39

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
Group	Deaths	Serious events	Other events
Treatment Group 1: Receiving Growth Hormone TreatmentThe denominator below (# at risk) is based on the number of subjects in Group 1 who received at least one dose of growth hormone.	–	0 / 13	1 / 13
Treatment Group 2: ControlThe denominator below (# at risk) is based on the number of subjects in Group 2 who received at least one dose of growth hormone. Per protocol, control subjects received no intervention; however, 3 control subjects were switched (crossed over) to the GH replacement group during the course of the study due to growth deceleration.	–	0 / 3	0 / 3

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: All
Age: up to 5 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

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.

PepBank is a research reference. Talk to a doctor before you use any peptide.