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: March 27, 2015
Last verified: March 2015
Results First Received: February 17, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Growth Hormone Deficiency
Septo-Optic Dysplasia
Hypopituitarism
Intervention: Drug: Nutropin AQ

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

Reporting Groups
  Description
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.

Participant Flow:   Overall Study
    Treatment Group 1: Receiving Growth Hormone Treatment     Treatment Group 2: Control  
STARTED     13     7  
Received at Least One Dose of GH     13     3 [1]
COMPLETED     10     7 [2]
NOT COMPLETED     3     0  
Lost to Follow-up                 1                 0  
Withdrawal by Subject                 2                 0  
[1] Controls were crossed over to GH if they met criteria for growth deceleration during the study.
[2] Crossed over subjects were treated as controls for purpose of intent to treat analysis.



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Please note, 3 children were unenrolled from the study and were therefore excluded from our analysis of results. Therefore, while the participant flow section lists 20 participants, the baseline analysis and results are based on 17 participants.

Reporting Groups
  Description
Treatment Group 1A: Assigned to GH Subjects with growth deceleration were assigned to the GH replacement treatment group in accordance with standard of care.
Treatment Group 1B: Randomized to GH Subjects with normal growth were randomized to GH treatment or to control (no intervention).
Treatment Group 2: Control Subjects with normal growth were randomized to treatment or to control (no intervention).
Total Total of all reporting groups

Baseline Measures
    Treatment Group 1A: Assigned to GH     Treatment Group 1B: Randomized to GH     Treatment Group 2: Control     Total  
Number of Participants  
[units: participants]
  5     5     7     17  
Age  
[units: participants]
       
<=18 years     5     5     7     17  
Between 18 and 65 years     0     0     0     0  
>=65 years     0     0     0     0  
Age  
[units: Months]
Median ( Inter-Quartile Range )
  28  
  ( 24 to 30 )  
  41  
  ( 33 to 61 )  
  26  
  ( 23 to 50 )  
  30  
  ( 24 to 50 )  
Gender  
[units: participants]
       
Female     3     2     4     9  
Male     2     3     3     8  
Weight z-score [1]
[units: Z-score]
Median ( Inter-Quartile Range )
  -1.76  
  ( -2.48 to -1.6 )  
  0.6  
  ( -0.47 to 1.49 )  
  -1.36  
  ( -1.73 to 0.41 )  
  -1.36  
  ( -1.73 to 0.6 )  
Stature z-score [1]
[units: Z-score]
Median ( Inter-Quartile Range )
  -2.16  
  ( -2.35 to -1.94 )  
  -0.95  
  ( -1.73 to -0.22 )  
  -1.3  
  ( -1.79 to -0.91 )  
  -1.73  
  ( -2.16 to -0.95 )  
Weight-for-stature z-score [2]
[units: Z-score]
Median ( Inter-Quartile Range )
  -0.38  
  ( -0.99 to -0.23 )  
  1.94  
  ( 0.88 to 2.49 )  
  1.34  
  ( 0.22 to 1.73 )  
  0.82  
  ( -0.38 to 1.91 )  
Body Mass Index (BMI) z-score [3]
[units: Z-score]
Median ( Inter-Quartile Range )
  -0.2  
  ( -0.5 to 0.11 )  
  2.06  
  ( 1.14 to 2.62 )  
  1.35  
  ( 0.38 to 1.7 )  
  1.14  
  ( 0.05 to 2.06 )  
[1] 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).
[2]

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).

[3]

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).




  Outcome Measures

1.  Primary:   Change in Anthropometric Measures Over Time   [ Time Frame: Baseline and 36 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Dr. Mark Borchert
Organization: Children's Hospital Los Angeles
phone: 323-361-6219
e-mail: mborchert@chla.usc.edu


No publications provided


Responsible Party: Mark Borchert, M.D., Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT00140413     History of Changes
Other Study ID Numbers: 03.261
Study First Received: August 31, 2005
Results First Received: February 17, 2015
Last Updated: March 27, 2015
Health Authority: United States: Institutional Review Board