Trial Review

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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
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
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04484051
Trial related presentations / publications
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

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
l.degraaff@erasmusmc.nl
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT04484051