Growth Hormone Use in Adults With Prader-Willi Syndrome

Phase 3

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Nutropin AQ

• Registration Number: NCT00444964
• Lead Sponsor: Children's Mercy Hospital Kansas City

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.

1. 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.

2. Improvement in quality of life measures as indicated by ratings on established behavior checklists in participants.

3. Changes in body composition (decreased fat, increased lean body mass and bone density) as determined by DEXA in participants.

4. 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.

Study Timeline

• Study Start: 2005-04
• Study First Submitted: 2007-03-06
• Study First Submitted QC: 2007-03-07
• Study First Posted: 2007-03-08
• Primary Completion: 2012-01-01
• Study Completion: 2012-01-01
• Results First Submitted: 2020-10-01
• Status Verified: 2020-12
• Results First Submitted QC: 2020-12-15
• Last Update Submitted: 2020-12-15
• Results First Posted: 2021-01-08
• Last Update Posted: 2021-01-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Primary Cohort	Nutropin AQ	Nutropin AQ

Primary Outcome Measures

Name	Time	Method
Body Composition	12 and 24 months	Anthropometric Measures and Body Composition
Blood Chemistry	12 and 24 months	Insulin-like growth factor I- IGF-1
Physical Activity	12 and 24 months	Duration of daily physical activity

Trial Locations

Locations (1)

The Children's Mercy Hospital; 🇺🇸 Kansas City, Missouri, United States