Phase 3
N=11
Growth Hormone Use in Adults With Prader-Willi Syndrome
Prader-Willi Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT00444964 ↗Enrolled (actual)
11
Serious AEs
9.1%
Results posted
Jan 2021
Primary outcome: Primary: Body Composition — 51.2; 48.5; 52.1 percentage of body fat — p=<0.05
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Nutropin AQ (Drug)
- Age
- Pediatric, Adult · 16+ yrs
- Sex
- All
- Sponsor
- Children's Mercy Hospital Kansas City
- Primary completion
- Jan 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Body Composition |
51.2; 48.5; 52.1 | <0.05 sig |
| PRIMARY Blood Chemistry |
93; 385; 140 | <0.05 sig |
| PRIMARY Physical Activity |
177; 256; 215 | <0.05 sig |
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.
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
Data sourced from ClinicalTrials.gov (NCT00444964). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.