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Growth Hormone Use in Adults With Prader-Willi Syndrome

Primary Purpose

Prader-Willi Syndrome

Status
Completed
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
Nutropin AQ
Sponsored by
Children's Mercy Hospital Kansas City
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional diagnostic trial for Prader-Willi Syndrome

Eligibility Criteria

16 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • 16 to 60 years old
  • Male or female with diagnosed Prader-Willi syndrome confirmed by genetic testing (e.g., mPCR)
  • Low IGF-1 level (e.g.,≤25%) at baseline
  • Ability to provide informed consent or availability of a suitable legally authorized representative

Exclusion Criteria:

  • Pregnancy
  • Previous treatment with growth hormone
  • Uncontrolled endocrine disease, (i.e. diabetes or thyroid)
  • History of severe scoliosis
  • Heart disease
  • Uncontrolled high blood pressure or history of stroke
  • Morbid obesity (using PWS growth charts)
  • Severe sleep apnea or known breathing difficulties/obstruction (as per history or diagnostic testing results

Sites / Locations

  • The Children's Mercy Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Primary Cohort

Arm Description

Nutropin AQ

Outcomes

Primary Outcome Measures

Body Composition
Anthropometric Measures and Body Composition
Blood Chemistry
Insulin-like growth factor I- IGF-1
Physical Activity
Duration of daily physical activity

Secondary Outcome Measures

Full Information

First Posted
March 6, 2007
Last Updated
December 15, 2020
Sponsor
Children's Mercy Hospital Kansas City
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1. Study Identification

Unique Protocol Identification Number
NCT00444964
Brief Title
Growth Hormone Use in Adults With Prader-Willi Syndrome
Official Title
Growth Hormone Use in Adults With Prader-Willi Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
April 2005 (Actual)
Primary Completion Date
January 1, 2012 (Actual)
Study Completion Date
January 1, 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Mercy Hospital Kansas City

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation of treatment for at least 12 months.
Detailed Description
The main research question this protocol aims to answer is whether treatment with growth hormone will impact body composition, quality of life, and energy balance in PWS adults, and if there is a loss of effects after cessation of treatment for at least 12 months. Specific outcomes to be evaluated are as follows: • Increased IGF-1 as a function of human growth hormone dosage compared with baseline. Improvement of indicators or risk factors for co-morbid diseases [diabetes (by measuring insulin and glucose levels), cardiovascular disease (by measuring lipids and fatty acids), and pulmonary function] in participants. Improvement in quality of life measures as indicated by ratings on established behavior checklists in participants. Changes in body composition (decreased fat, increased lean body mass and bone density) as determined by DEXA in participants. Increased energy expenditure as determined by whole-room calorimeter measures (8 hour energy expenditure, RMR, TEF, mechanical work); diet records, physical activity monitors and strength measures.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome

7. Study Design

Primary Purpose
Diagnostic
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
11 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Primary Cohort
Arm Type
Experimental
Arm Description
Nutropin AQ
Intervention Type
Drug
Intervention Name(s)
Nutropin AQ
Other Intervention Name(s)
Growth Hormone
Intervention Description
0.0125 mg/kg/day
Primary Outcome Measure Information:
Title
Body Composition
Description
Anthropometric Measures and Body Composition
Time Frame
12 and 24 months
Title
Blood Chemistry
Description
Insulin-like growth factor I- IGF-1
Time Frame
12 and 24 months
Title
Physical Activity
Description
Duration of daily physical activity
Time Frame
12 and 24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
16 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 16 to 60 years old Male or female with diagnosed Prader-Willi syndrome confirmed by genetic testing (e.g., mPCR) Low IGF-1 level (e.g.,≤25%) at baseline Ability to provide informed consent or availability of a suitable legally authorized representative Exclusion Criteria: Pregnancy Previous treatment with growth hormone Uncontrolled endocrine disease, (i.e. diabetes or thyroid) History of severe scoliosis Heart disease Uncontrolled high blood pressure or history of stroke Morbid obesity (using PWS growth charts) Severe sleep apnea or known breathing difficulties/obstruction (as per history or diagnostic testing results
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Merlin G Butler, MD
Organizational Affiliation
Children's Mercy Hospital Kansas City
Official's Role
Principal Investigator
Facility Information:
Facility Name
The Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States

12. IPD Sharing Statement

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Growth Hormone Use in Adults With Prader-Willi Syndrome

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