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Effects of Growth Hormone in Chronically Ill Children

Primary Purpose

Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis

Status
Withdrawn
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Growth Hormone
Whole body Protein turnover
DEXA scan
Sponsored by
University of Texas Southwestern Medical Center
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hurler Syndrome

Eligibility Criteria

3 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Ages 3-17 years Tanner stages 1-3 Each child must have received adequate nutritional therapy supplied by aggressive oral supplementation, gastrostomy tube, or TPN for at least 1 year prior to enrollment. All children will have been referred for continued poor growth and will be less than the 10th percentile for height compared to age and gender normal values. low IGF-1 level at the time of enrollment (measured in the Endocrine clinic). Chronic illness to be included are Hurler Syndrome (MPS-1) with short stature and muscle wasting, cerebral palsy with muscle wasting, juvenile rheumatoid arthritis with muscle wasting and short stature, Crohn's disease and HIV infection. Exclusion Criteria: previous diagnosis with diabetes, chronic fevers (temp > 101.5) or chronic bacterial infection. substantial change in steroid dosing, or having a formerly steroid negative patient start long-term-steroids (anticipated use greater that 7 days

Sites / Locations

  • Children's Medical Center of Dallas

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

First Posted
February 1, 2006
Last Updated
December 12, 2018
Sponsor
University of Texas Southwestern Medical Center
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1. Study Identification

Unique Protocol Identification Number
NCT00286689
Brief Title
Effects of Growth Hormone in Chronically Ill Children
Study Type
Interventional

2. Study Status

Record Verification Date
December 2018
Overall Recruitment Status
Withdrawn
Why Stopped
Record Owner no longer with the institution
Study Start Date
February 3, 2006 (Actual)
Primary Completion Date
February 3, 2006 (Actual)
Study Completion Date
February 3, 2006 (Actual)

3. Sponsor/Collaborators

Name of the Sponsor
University of Texas Southwestern Medical Center

4. Oversight

5. Study Description

Brief Summary
The specific aims for this study are - To determine the effect of GH on height, height velocity, body weight and lean body mass. This specific aim tests the hypothesis that GH significantly improves height, height velocity, weight, weight velocity and lean body mass in chronically ill children who have grown poorly despite adequate nutritional rehabilitation. To determine the effect of GH on whole body protein turnover (WBPT), IGF-1 levels and on cytokines. This specific aim tests the hypothesis that chronically ill children have increased catabolism, caused by high levels of circulating cytokines and low levels of IGF-1, and that these abnormalities improve with GH treatment. Evaluation of bone mineral density and bone turnover. This specific aim tests the hypothesis that bone density is low in chronically ill children secondary to increased osteoclast activity correlating with elevated cytokine levels. We hypothesize that the anabolic effects of growth hormone (GH) will improve the height and weight of chronically ill children who have failed to grow despite receiving adequate nutrition via gastrostomy tube or oral supplementation.
Detailed Description
We will test our hypotheses by using a pilot study, in which we will recruit 18 chronically ill children from our clinical practice. We will obtain medical records for each patient 12 months prior to starting the study. Those patients without pre-study medical records will be studied for 12 months prior to starting GH. If we can obtain 6 months of prior medical records, then the patients will be studied for 6 months before starting GH. Anything less than 6 months will be studied for the full 12 months prior to starting GH. Patients will receive treatment with GH (0.3 mg/kg/wk) for 12 months and their growth will be compared to the year before treatment. All subjects will be followed every three months for the entire study. We will measure height and weight using a standardized stadiometer and scale, respectively, every three months during the study. From these measurements we will calculate height and weight velocity and height and weight Z score. Lean body mass (LBM) will be measured by DEXA every six months. Utilizing the stable isotope 1-[13C] leucine, we will measure whole body protein turnover (WBPT). Measurements of WBPT will be correlated with LBM and changes in height and weight velocity. This data will be compared to that from age matched normal children (archival data maintained by the PI). We will measure IGF-1 and the cytokines TNF-α, IL-6 and IL 10 at baseline and very six months. We will evaluate GH effects on these levels.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hurler Syndrome, Cerebral Palsy, Juvenile Rheumatoid Arthritis, Crohn Disease, HIV Infections

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Drug
Intervention Name(s)
Growth Hormone
Intervention Type
Procedure
Intervention Name(s)
Whole body Protein turnover
Intervention Type
Procedure
Intervention Name(s)
DEXA scan

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Ages 3-17 years Tanner stages 1-3 Each child must have received adequate nutritional therapy supplied by aggressive oral supplementation, gastrostomy tube, or TPN for at least 1 year prior to enrollment. All children will have been referred for continued poor growth and will be less than the 10th percentile for height compared to age and gender normal values. low IGF-1 level at the time of enrollment (measured in the Endocrine clinic). Chronic illness to be included are Hurler Syndrome (MPS-1) with short stature and muscle wasting, cerebral palsy with muscle wasting, juvenile rheumatoid arthritis with muscle wasting and short stature, Crohn's disease and HIV infection. Exclusion Criteria: previous diagnosis with diabetes, chronic fevers (temp > 101.5) or chronic bacterial infection. substantial change in steroid dosing, or having a formerly steroid negative patient start long-term-steroids (anticipated use greater that 7 days
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dana S Hardin, MD
Organizational Affiliation
University of Texas Southwestern Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Medical Center of Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States

12. IPD Sharing Statement

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Effects of Growth Hormone in Chronically Ill Children

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