Growth Hormone Use in Adults With Prader-Willi Syndrome

• Sponsor: Children's Mercy Hospital Kansas City (Other)
• Overall Status: Completed
• CT.gov ID: NCT00444964
• Collaborator: (none)
• Enrollment: 11
• Location: 1
• Arm: 1
• Actual Duration (Months): 81
• Patients Per Site Per Month: 0.1

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Prader-Willi Syndrome	Drug: Nutropin AQ	Phase 3

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 Design

• Study Type: Interventional
• Actual Enrollment: 11 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Diagnostic
• Official Title: Growth Hormone Use in Adults With Prader-Willi Syndrome
• Actual Study Start Date: Apr 1, 2005
• Actual Primary Completion Date: Jan 1, 2012
• Actual Study Completion Date: Jan 1, 2012

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Primary Cohort Nutropin AQ	Drug: Nutropin AQ; 0.0125 mg/kg/day; Other Names: Growth Hormone

Outcome Measures

Primary Outcome Measures

1. Body Composition [12 and 24 months]

   Anthropometric Measures and Body Composition

2. Blood Chemistry [12 and 24 months]

   Insulin-like growth factor I- IGF-1

3. Physical Activity [12 and 24 months]

   Duration of daily physical activity

Eligibility Criteria

Criteria

• Ages Eligible for Study: 16 Years to 60 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	The Children's Mercy Hospital	Kansas City	Missouri	United States	64108

Sponsors and Collaborators

• Children's Mercy Hospital Kansas City

Investigators

• Principal Investigator: Merlin G Butler, MD, Children's Mercy Hospital Kansas City

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Children's Mercy Hospital Kansas City
• ClinicalTrials.gov Identifier: NCT00444964
• Other Study ID Numbers: 303-C02R
• First Posted: Mar 8, 2007
• Last Update Posted: Jan 8, 2021
• Last Verified: Dec 1, 2020

• Additional relevant MeSH terms: Prader-Willi Syndrome; Syndrome; Hormones

Study Results

Participant Flow

Arm/Group Title	Primary Cohort
Arm/Group Description	Nutropin AQ: 0.0125 mg/kg/day
Period Title: Overall Study
STARTED	11
COMPLETED	11
NOT COMPLETED	0

Baseline Characteristics

Arm/Group Title	Primary Cohort
Arm/Group Description	Nutropin AQ: 0.0125 mg/kg/day
Overall Participants	11
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	32.3 (11.1)
Sex: Female, Male (Count of Participants)
Female	6 54.5%
Male	5 45.5%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%
Asian	0 0%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	0 0%
White	11 100%
More than one race	0 0%
Unknown or Not Reported	0 0%

Outcome Measures

1. Primary Outcome

Title	Body Composition
Description	Anthropometric Measures and Body Composition
Time Frame	12 and 24 months

Outcome Measure Data

Analysis Population Description

All patients enrolled

Arm/Group Title	Primary Cohort
Arm/Group Description	Nutropin AQ: 0.0125 mg/kg/day
Measure Participants	11
Baseline	51.2 (2.1)
12 Months	48.5 (2.2)
24 Months	52.1 (2.3)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Primary Cohort
	Comments
	Type of Statistical Test	Other
	Comments
Statistical Test of Hypothesis	p-Value	<0.05
	Comments
	Method	Mixed Models Analysis
	Comments

2. Primary Outcome

Title	Blood Chemistry
Description	Insulin-like growth factor I- IGF-1
Time Frame	12 and 24 months

Outcome Measure Data

Analysis Population Description

All patients enrolled

Arm/Group Title	Primary Cohort
Arm/Group Description	Nutropin AQ: 0.0125 mg/kg/day
Measure Participants	11
Baseline	93 (37.6)
12 months	385 (48.6)
24 Months	140 (53.1)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Primary Cohort
	Comments
	Type of Statistical Test	Other
	Comments
Statistical Test of Hypothesis	p-Value	<0.05
	Comments
	Method	Mixed Models Analysis
	Comments

3. Primary Outcome

Title	Physical Activity
Description	Duration of daily physical activity
Time Frame	12 and 24 months

Outcome Measure Data

Analysis Population Description

All patients enrolled

Arm/Group Title	Primary Cohort
Arm/Group Description	Nutropin AQ: 0.0125 mg/kg/day
Measure Participants	11
Baseline	177 (27)
12 Months	256 (33)
24 Months	215 (49)

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	Primary Cohort
	Comments
	Type of Statistical Test	Other
	Comments
Statistical Test of Hypothesis	p-Value	<0.05
	Comments
	Method	Mixed Models Analysis
	Comments

Adverse Events

Time Frame	24 months
Arm/Group Title	Primary Cohort
Arm/Group Description	Nutropin AQ: 0.0125 mg/kg/day
All Cause Mortality
	Primary Cohort
	Affected / at Risk (%)	# Events
Total	/ (NaN)
Serious Adverse Events
	Primary Cohort
	Affected / at Risk (%)	# Events
Total	1/11 (9.1%)
Musculoskeletal and connective tissue disorders
Fall	1/11 (9.1%)	1
Other (Not Including Serious) Adverse Events
	Primary Cohort
	Affected / at Risk (%)	# Events
Total	0/11 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

There is NOT 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.