Individuals with abnormal rhythmicity will be treated with melatonin to assess if sleep patterns are improved.

Biological Clock Dysfunction

Optic Nerve Hypoplasia

Oral administration before bed. We will test two doses (0.5 or 3.0 mg/m2. 6 week duration.)

Melatonin

Oral administration before bed. 6 week duration.

Placebo Comparator

Children's Hospital of Los Angeles

Biological Clock Dysfunction in Optic Nerve Hypoplasia

Identification and Treatment of Biological Clock Dysfunction in Optic Nerve Hypoplasia

Esther Nakamura-Reyes

Yale University

Children's Hospital Los Angeles

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.

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.

Subjects with growth deceleration were assigned to the GH replacement treatment group in accordance with standard of care.

Treatment Group 1A: Assigned to GH

Subjects with normal growth were randomized to GH treatment or to control (no intervention).

Treatment Group 1B: Randomized to GH

Subjects with normal growth were randomized to treatment or to control (no intervention).

Treatment Group 2: Control

Total

Sex: Female, Male

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).

Weight z-score

Stature z-score

Weight (wt.) for stature expresses wt. relative to stature (length or height); data are not age specific. The wt. for stature reference data from CDC growth charts compares a child's wt. with the wt. distribution of children of the same stature but not necessarily of the same age.
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. All z-scores were obtained using Epi Info ™ 3.5.4. (Centers for Disease Control, Atlanta, GA).

Weight-for-stature z-score

BMI adjusts weight for stature and is calculated as weight (kg)/height (m) squared. In children, BMI is age specific. BMI reference data from the CDC growth charts compares a child's BMI with the BMI distribution of children of the same age but not necessarily of the same stature.
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. All z-scores were obtained using Epi Info ™ 3.5.4. (Centers for Disease Control, Atlanta, GA).

Body Mass Index (BMI) z-score

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).

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 replacement treatment group in accordance with standard of care. Subjects with normal growth were randomized to treatment or to control (no intervention). The starting daily dose for GH replacement was calculated as 0.3 mg/kg/wk and subsequently modified based on observed length/height velocity and serum IGF-I levels.

Treatment Group 1: Receiving Growth Hormone 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 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.
A total of 7 subjects were randomized to the control group, 3 of whom were crossed over to treatment due to growth deceleration. Outcome measures for these 3 who crossed over were analyzed separately, under Treatment Group 3.

3 control subjects were switched (crossed over) to the GH replacement group during the course of the study due to growth deceleration.

Treatment Group 3: Control Crossed Over to Treatment

Change in Anthropometric Measures Over Time

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 replacement treatment group in accordance with standard of care. Subjects with normal growth were randomized to treatment or to control (no intervention). The starting dose for GH replacement was calculated as 0.3 mg/kg/wk and subsequently modified based on observed length/height velocity and serum IGF-I levels.

Overall Study

Dr. Mark Borchert

The denominator below (# at risk) is based on the number of subjects in Group 1 who received at least one dose of growth hormone.

The 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.

Growth Hormone Deficiency

Septo-Optic Dysplasia

Hypopituitarism

Daily injection. Dosage dependent on weight.

optic nerve hypoplasia/obesity

Biological Clock Dysfunction in Optic Nerve Hypoplasia

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

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