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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT04484051
Registration number
NCT04484051
Ethics application status
Date submitted
15/07/2020
Date registered
23/07/2020
Date last updated
16/02/2023
Titles & IDs
Public title
Growth Hormone Study in Adults With Prader-Willi Syndrome
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Scientific title
Growth Hormone Study in Adults With Prader-Willi Syndroom
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Secondary ID [1]
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GAP
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Universal Trial Number (UTN)
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Trial acronym
GAP
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Prader-Willi Syndrome
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Mental Health
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Learning disabilities
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Observational
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Patient registry
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Target follow-up duration
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Target follow-up type
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Description of intervention(s) / exposure
Routine clinical care with Genotropin treatment - Data collection throughout routine clinical care with subcutaneous injections Genotropin, 0.6-0.8 mg/day. Participants start with 0.2 mg/day and the dose increases with 0.2 mg/day per month to a maximum dose of 0.6-0.8 mg/day.
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Change in lean body mass
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Assessment method [1]
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Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
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Timepoint [1]
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36 months
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Secondary outcome [1]
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Change in fat mass
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Assessment method [1]
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Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
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Timepoint [1]
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36 months
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Secondary outcome [2]
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Change in bone density
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Assessment method [2]
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Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan
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Timepoint [2]
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36 months
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Secondary outcome [3]
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Change in physical strength
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Assessment method [3]
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Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests
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Timepoint [3]
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36 months
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Secondary outcome [4]
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Change in laboratory measurements
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Assessment method [4]
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Changes in the following laboratory measurements:
Glycosylated hemoglobin (mmol/mol)
Total cholesterol (mmol/L)
Low-density lipoprotein cholesterol (mmol/L)
High-density lipoprotein cholesterol (mmol/L)
Insulin-like growth factor 1 (nmol/L)
Free thyroxine 4 (pmol/L)
Luteinizing hormone (U/I)
Follicle stimulating hormone (U/I)
Estradiol or testosterone (nmol/L)
Sex hormone binding globulin (nmol/L)
Aspartate transaminase (U/L)
Alanine transaminase (U/L)
Alkaline phosphatase (U/L)
Gamma glutamyl transpeptidase (U/L)
Total bilirubin (micromol/L)
Lactate dehydrogenase (U/L)
Urea (mmol/L)
Creatinine (micromol/L)
Hemoglobin (mmol/L)
Hematocrit (L/L)
Mean corpuscular volume (fL)
Leukocytes (10^9/L)
Thrombocytes (10^9/L)
25-OH vitamin D (nmol/L)
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Timepoint [4]
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36 months
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Secondary outcome [5]
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Change in psychosocial functioning
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Assessment method [5]
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Change in psychosocial functioning as estimated with the Adult Behaviour Checklist
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Timepoint [5]
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36 months
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Secondary outcome [6]
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Change in caregiver burden
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Assessment method [6]
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Change in caregiver burden as estimated with the Zarit Burden Interview
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Timepoint [6]
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36 months
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Eligibility
Key inclusion criteria
- The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)
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Minimum age
18
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Non cooperative behaviour
- Pregnancy
- Known malignancies
- Poorly controlled diabetes (HbA1c > 64 mmol/mol (8%))
- Untreated obstructive sleep apnea (apnea-hypopnea index > 5)
- Body mass index above 40 kg/m2
- Upper-airway obstruction of any cause
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Study design
Purpose
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Duration
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Selection
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Timing
Prospective
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Statistical methods / analysis
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Recruitment
Recruitment status
Not yet recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
24/03/2023
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Actual
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/10/2026
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Actual
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Sample size
Target
25
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment outside Australia
Country [1]
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Netherlands
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State/province [1]
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Zuid-Holland
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Funding & Sponsors
Primary sponsor type
Other
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Name
Erasmus Medical Center
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Address
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Country
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Other collaborator category [1]
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Other
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Name [1]
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Foundation for Prader-Willi Research
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Address [1]
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Country [1]
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Other collaborator category [2]
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Other
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Name [2]
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Prader-Willi Fonds
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Address [2]
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Country [2]
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Ethics approval
Ethics application status
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Summary
Brief summary
The overall objective of this study is to measure the effect of growth hormone (GH) treatment
on physical and psychosocial health in adults with Prader-Willi syndrome. Adults with PWS who
have not been treated with GH during the past three years and who will start with GH
treatment as part of regular patient care will be asked for informed consent to participate
in this open-label prospective cohort study. We hypothesize that growth hormone treatment
will improve the physical and psychosocial health.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04484051
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Trial related presentations / publications
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Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed
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Contacts
Principal investigator
Name
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Laura de Graaff, MD, PhD
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Address
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Erasmus MC, University Medical Center Rotterdam
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Laura de Graaff, MD, PhD
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Address
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Country
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Phone
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0031618843010
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04484051
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