Trial Outcomes & Findings for Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia (NCT NCT00140413)
NCT ID: NCT00140413
Last Updated: 2015-04-01
Results Overview
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).
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
20 participants
Primary outcome timeframe
Baseline and 36 months
Results posted on
2015-04-01
Participant Flow
Participants were recruited from an IRB-approved prospective optic nerve hypoplasia (ONH) registry study at Children's Hospital Los Angeles, between January 2005 through March 2011.
39 participants were screened for eligibility: 20 enrolled in the study; 6 failed to meet inclusion criteria; 9 declined participation; and 4 consented to participate but withdrew from the study prior to beginning any study procedures.
Participant milestones
| Measure |
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 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.
|
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.
|
|---|---|---|
|
Overall Study
STARTED
|
13
|
7
|
|
Overall Study
Received at Least One Dose of GH
|
13
|
3
|
|
Overall Study
COMPLETED
|
10
|
7
|
|
Overall Study
NOT COMPLETED
|
3
|
0
|
Reasons for withdrawal
| Measure |
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 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.
|
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.
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
1
|
0
|
|
Overall Study
Withdrawal by Subject
|
2
|
0
|
Baseline Characteristics
Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia
Baseline characteristics by cohort
| Measure |
Treatment Group 1A: Assigned to GH
n=5 Participants
Subjects with growth deceleration were assigned to the GH replacement treatment group in accordance with standard of care.
|
Treatment Group 1B: Randomized to GH
n=5 Participants
Subjects with normal growth were randomized to GH treatment or to control (no intervention).
|
Treatment Group 2: Control
n=7 Participants
Subjects with normal growth were randomized to treatment or to control (no intervention).
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
17 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Continuous
|
28 Months
n=5 Participants
|
41 Months
n=7 Participants
|
26 Months
n=5 Participants
|
30 Months
n=4 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
9 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
|
Weight z-score
|
-1.76 Z-score
n=5 Participants
|
0.6 Z-score
n=7 Participants
|
-1.36 Z-score
n=5 Participants
|
-1.36 Z-score
n=4 Participants
|
|
Stature z-score
|
-2.16 Z-score
n=5 Participants
|
-0.95 Z-score
n=7 Participants
|
-1.3 Z-score
n=5 Participants
|
-1.73 Z-score
n=4 Participants
|
|
Weight-for-stature z-score
|
-0.38 Z-score
n=5 Participants
|
1.94 Z-score
n=7 Participants
|
1.34 Z-score
n=5 Participants
|
0.82 Z-score
n=4 Participants
|
|
Body Mass Index (BMI) z-score
|
-0.2 Z-score
n=5 Participants
|
2.06 Z-score
n=7 Participants
|
1.35 Z-score
n=5 Participants
|
1.14 Z-score
n=4 Participants
|
PRIMARY outcome
Timeframe: Baseline and 36 monthsPrimary 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).
Outcome measures
| Measure |
Treatment Group 1: Receiving Growth Hormone Treatment
n=10 Participants
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 2: Control
n=4 Participants
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.
|
Treatment Group 3: Control Crossed Over to Treatment
n=3 Participants
3 control subjects were switched (crossed over) to the GH replacement group during the course of the study due to growth deceleration.
|
|---|---|---|---|
|
Change in Anthropometric Measures Over Time
Change in stature z-score
|
1.97 Z-score
Interval 0.56 to 2.29
|
-0.61 Z-score
Interval -0.9625 to -0.305
|
1.39 Z-score
Interval 0.62 to 1.43
|
|
Change in Anthropometric Measures Over Time
Change in body mass index (BMI) z-score
|
-0.06 Z-score
Interval -0.24 to 0.39
|
-0.505 Z-score
Interval -0.7025 to -0.3075
|
-1.035 Z-score
Interval -1.1525 to -0.9175
|
|
Change in Anthropometric Measures Over Time
Change in weight z-score
|
1.22 Z-score
Interval 0.49 to 2.16
|
-0.38 Z-score
Interval -0.715 to -0.195
|
0.58 Z-score
Interval -0.06 to 1.92
|
|
Change in Anthropometric Measures Over Time
Change in weight-for-stature z-score
|
0.52 Z-score
Interval -0.21 to 0.705
|
-0.37 Z-score
Interval -0.825 to -0.1075
|
0.95 Z-score
Interval 0.1 to 1.8
|
Adverse Events
Treatment Group 1: Receiving Growth Hormone Treatment
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Treatment Group 2: Control
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Treatment Group 1: Receiving Growth Hormone Treatment
n=13 participants at risk
The denominator below (# at risk) is based on the number of subjects in Group 1 who received at least one dose of growth hormone.
|
Treatment Group 2: Control
n=3 participants at risk
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.
|
|---|---|---|
|
Nervous system disorders
Headache
|
7.7%
1/13 • Number of events 1 • 36 months (study duration)
|
0.00%
0/3 • 36 months (study duration)
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee With respect to any proposed publication, the PI must submit to the sponsor a copy of the proposed publication at least 2 months prior to submission for the purposes of: providing the sponsor the oppotunity to review and comment on the contents of proposed publication; and to identify any trade secret, proprietary or confidential information to be removed from the proposed publication. The sponsor will provide comments within 30 days of receipt of proposed publication.
- Publication restrictions are in place
Restriction type: OTHER