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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04484051




Registration number
NCT04484051
Ethics application status
Date submitted
15/07/2020
Date registered
23/07/2020

Titles & IDs
Public title
Growth Hormone Study in Adults With Prader-Willi Syndrome
Scientific title
Growth Hormone Study in Adults With Prader-Willi Syndroom
Secondary ID [1] 0 0
GAP
Universal Trial Number (UTN)
Trial acronym
GAP
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Prader-Willi Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Mental Health 0 0 0 0
Learning disabilities
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
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.
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in lean body mass
Timepoint [1] 0 0
36 months
Secondary outcome [1] 0 0
Change in fat mass
Timepoint [1] 0 0
36 months
Secondary outcome [2] 0 0
Change in bone density
Timepoint [2] 0 0
36 months
Secondary outcome [3] 0 0
Change in physical strength
Timepoint [3] 0 0
36 months
Secondary outcome [4] 0 0
Change in laboratory measurements
Timepoint [4] 0 0
36 months
Secondary outcome [5] 0 0
Change in psychosocial functioning
Timepoint [5] 0 0
36 months
Secondary outcome [6] 0 0
Change in caregiver burden
Timepoint [6] 0 0
36 months

Eligibility
Key inclusion criteria
* The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
24/03/2023
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2026
Actual
Sample size
Target
25
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
Netherlands
State/province [1] 0 0
Zuid-Holland

Funding & Sponsors
Primary sponsor type
Other
Name
Erasmus Medical Center
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Foundation for Prader-Willi Research
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Prader-Willi Fonds
Address [2] 0 0
Country [2] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Laura de Graaff, MD, PhD
Address 0 0
Erasmus MC, University Medical Center Rotterdam
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Laura de Graaff, MD, PhD
Address 0 0
Country 0 0
Phone 0 0
0031618843010
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04484051