Prader-Willi SyndromePhase 3NCT00444964
Growth Hormone Use in Adults With Prader-Willi Syndrome
A Phase 3 study testing Nutropin AQ in people with Prader-Willi Syndrome, run by Children's Mercy Hospital Kansas City at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 16 to 60 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Nutropin AQ
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2005to 1 Jan 2012
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 16 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
The Children's Mercy Hospital
Kansas City, Missouri, 64108
About this study
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.
Read the 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.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Nutropin AQ
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nutropin AQDrug
Also called Growth Hormone
0.0125 mg/kg/day
| Study group | Role | What they receive |
|---|---|---|
| Primary Cohort | Experimental | Nutropin AQ Receives: Drug: Nutropin AQ |
What is being measured
The primary outcome is the main question the study is designed to answer.
Body Composition
Anthropometric Measures and Body Composition
Measured: 12 and 24 months
Blood Chemistry
Insulin-like growth factor I- IGF-1
Measured: 12 and 24 months
Physical Activity
Duration of daily physical activity
Measured: 12 and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Mar 2007 (estimated)
- Study startApr 2005
- Primary completion1 Jan 2012
- Study completion1 Jan 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Merlin G Butler, MD, Principal investigator, Children's Mercy Hospital Kansas City
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00444964
- Sponsor's protocol ID
- 303-C02R
- Status last verified
- Dec 2020
- First submitted
- 6 Mar 2007
- Last updated
- 8 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Growth Hormone Use in Adults With Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT00444964
- Acronym / sponsor ID
- 303-C02R
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Children's Mercy Hospital Kansas CityUniversity, hospital or other
- Study start
- Apr 2005 (actual)
- Primary completion
- 1 Jan 2012 (actual)
- Study completion
- 1 Jan 2012 (actual)
- First posted
- 8 Mar 2007 (estimated)
- Results
- Posted 8 Jan 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Primary Cohort | Experimental | Drug: Nutropin AQNutropin AQ |
Who may qualify
- Ages
- 16 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (8 listed)
- Pregnancy
- Previous treatment with growth hormone
- Uncontrolled endocrine disease, (i.e. diabetes or thyroid)
- History of severe scoliosis
- Heart disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Nutropin AQAlso: Growth Hormone0.0125 mg/kg/day | Drug | Primary Cohort |
Primary outcome
Body Composition
Time frame: 12 and 24 months
Anthropometric Measures and Body Composition
Blood Chemistry
Time frame: 12 and 24 months
Insulin-like growth factor I- IGF-1
Physical Activity
Time frame: 12 and 24 months
Duration of daily physical activity
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Merlin G Butler, MD principal investigator
Children's Mercy Hospital Kansas City
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Nutropin AQ in people with Prader-Willi Syndrome, run by Children's Mercy Hospital Kansas City at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Body Composition (time frame: 12 and 24 months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2021.