Growth Hormone Use in Adults With Prader-Willi Syndrome

NCT00444964 | Completed Phase 3 Results DMC

• 1 other identifier: 303-C02R
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

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.

Conditions

Prader-Willi Syndrome

Outcome Measures

Primary Outcomes (3)

• Body Composition

  Anthropometric Measures and Body Composition

  12 and 24 months

• Blood Chemistry

  Insulin-like growth factor I- IGF-1

  12 and 24 months

• Physical Activity

  Duration of daily physical activity

  12 and 24 months

Study Arms (1)

Primary Cohort

EXPERIMENTAL

Nutropin AQ

Drug: Nutropin AQ

Interventions

Nutropin AQ DRUG

0.0125 mg/kg/day

Also known as: Growth Hormone

Primary Cohort

Eligibility Criteria

• Age: 16 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Mercy Hospital Kansas City: lead

Study Sites (1)

The Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Results Point of Contact

• Title: Jaylene Weigel
• Organization: Children's Mercy Hospital

jweigel@cmh.edu

816-701-1359

Study Officials

• Merlin G Butler, MD

  Children's Mercy Hospital Kansas City

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 6, 2007
• First Posted: March 8, 2007
• Study Start: April 1, 2005
• Primary Completion: January 1, 2012
• Study Completion: January 1, 2012
• Last Updated: January 8, 2021
• Results First Posted: January 8, 2021

Record last verified: 2020-12

Locations

US (1)