We are experiencing 4 week turn-around time in review of submissions and resubmissions. We recommend commencing this process concurrently with your ethics submission and allowing at least 8 weeks for registration to be completed from date of first submission. We currently do not have the capacity to expedite reviews.

Note also there are delays to review of updates. We appreciate your patience.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

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




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

Titles & IDs
Public title
Global Growth Hormone Study in Adults With Prader-Willi Syndrome
Scientific title
Global Growth Hormone Study in Adults With Prader-Willi Syndroom
Secondary ID [1] 0 0
GGAP
Universal Trial Number (UTN)
Trial acronym
GGAP
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
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Somatropin
Treatment: Drugs - Placebo

Active Comparator: Active comparator: Genotropin - 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.

Placebo Comparator: Placebo comparator: Placebo - Placebo for 12 months.


Treatment: Drugs: Somatropin
The intervention is growth hormone treatment (Genotropin), 0.6 - 0.8 mg/day subcutaneous for one year. It is an intramural medicament with an add-on. Participants start with 0.2 mg/day. The growth hormone dose increases with 0.2 mg/day per month to a maximum dose of 0.6 - 0.8 mg/day based on clinical signs (occurrence of side-effects)

Treatment: Drugs: Placebo
The comparator is placebo, 0.6 - 0.8 mg/day subcutaneous for one year. Participants start with 0.2 mg/day. The dose increases with 0.2 mg/day per month to a maximum dose of 0.6 - 0.8 mg/day.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in lean body mass - Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
Timepoint [1] 0 0
27 months
Secondary outcome [1] 0 0
Change in fat mass - Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
Timepoint [1] 0 0
27 months
Secondary outcome [2] 0 0
Change in bone density - Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan
Timepoint [2] 0 0
27 months
Secondary outcome [3] 0 0
Change in cardiovascular fitness - Change in cardiovascular fitness as estimated with an ECG during the treadmill stress test. We look for axis devations in LEAD I and aVF. We also look for signs of ischemia (ST depression, T wave inversion and pathologic Q waves).
Timepoint [3] 0 0
27 months
Secondary outcome [4] 0 0
Change in laboratory measurements - Changes in the following laboratory measurements:
Fasting blood glucose (mmol/L)
Glycosylated hemoglobin (mmol/mol)
Total cholesterol (mmol/L)
Low-density lipoprotein cholesterol (mmol/L)
High-density lipoprotein cholesterol (mmol/L)
Triglycerides (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)
Timepoint [4] 0 0
27 months
Secondary outcome [5] 0 0
Change in muscle strength - Change in muscle strength as determined with a handgrip dynamometer
Timepoint [5] 0 0
27 months
Secondary outcome [6] 0 0
Change in endurance - Change in endurance as estimated with the treadmill stress test
Timepoint [6] 0 0
27 months
Secondary outcome [7] 0 0
Change in psychosocial functioning - Change in psychosocial functioning as estimated with the Adult Behaviour Checklist
Timepoint [7] 0 0
27 months

Eligibility
Key inclusion criteria
- The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)

- The patient is 30 years or older

- In case of previous GH treatment (for example in trial setting), GH should be stopped
at least three years before starting the study

- The patient is treated by a dietitian (caloric restriction) for at least three months
Minimum age
30 Years
Maximum age
No limit
Gender
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

- Change in testosterone or estrogen replacement therapy in the last three months prior
to study

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment postcode(s) [1] 0 0
NSW 2050 - Camperdown
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
Commercial sector/Industry
Name [1] 0 0
Pfizer
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Foundation for Prader-Willi Research
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Prader-Willi Fonds
Address [3] 0 0
Country [3] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
The overall objective of this study is to measure the effect of growth hormone treatment on
physical and psychosocial health in adults of 30 years or older with Prader-Willi syndrome.
Patients are randomized to placebo or growth hormone treatment during the first year. They
will switch treatment during the second year, so that each participant receives one year of
growth hormone treatment and one year of placebo (cross-over study). We hypothesize that
growth hormone treatment will improve the physical and psychosocial health.
Trial website
https://clinicaltrials.gov/show/NCT04484051
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
l.degraaff@erasmusmc.nl
Contact person for scientific queries

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