Global Growth Hormone Study in Adults With Prader-Willi Syndrome

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NCT04484051

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Study Location
Royal Prince Alfred Hospital
Camperdown, , NSW 2050, Australia
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Contact
0031618843010
[email protected]
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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Prader-Willi Syndrome
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
30 + years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- 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

Exclusion Criteria
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


- 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

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Advanced Information
Descriptive Information
Brief Title  ICMJE Global Growth Hormone Study in Adults With Prader-Willi Syndrome
Official Title  ICMJE Global Growth Hormone Study in Adults With Prader-Willi Syndroom
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.
Detailed Description

OBJECTIVES:

The overall objective is to measure the effect of growth hormone treatment (GHt) on physical and psychosocial health in adults of 30 years or older with Prader-Willi syndrome (PWS).

The primary objective is to measure the effect of GHt on lean body mass as measured by Dual Energy X-ray Absorptiometry scan in adults of 30 years or older with PWS.

The secondary objective is to measure the effect of GHt on total fat mass, bone density, physical health cardiovascular fitness, laboratory measurements, muscle strength, endurance, and psychosocial functioning in adults of 30 years or older with PWS. Also the occurrence of side-effects will be assessed.

STUDY DESIGN:

Randomized, double-blinded, placebo controlled crossover trial for two years with a washout period of 3 months.

STUDY POPULATION:

50 adults with PWS of 30 years or older who have not been treated with GH during the past three years.

INTERVENTION:

subcutaneous injections of growth hormone (Genotropin, 5.0 mg/mL) in a dosage of 0.6 - 0.8 mg/day. The comparator is placebo.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Prader-Willi Syndrome
Intervention  ICMJE
  • Drug: 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)
    Other Name: Genotropin (5,0 mg/mL)
  • Drug: 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.
Study Arms  ICMJE
  • 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.
    Intervention: Drug: Somatropin
  • Placebo Comparator: Placebo comparator: Placebo
    Placebo for 12 months.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Not yet recruiting
Estimated Enrollment  ICMJE
 (submitted: July 21, 2020)		50
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE January 1, 2024
Estimated Primary Completion Date January 1, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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

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
Sex/Gender  ICMJE
Sexes Eligible for Study:All
Ages  ICMJE 30 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Laura de Graaff, MD, PhD0031618843010[email protected]
Listed Location Countries  ICMJE Australia,   Netherlands
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04484051
Other Study ID Numbers  ICMJE GGAP
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
IPD Sharing Statement  ICMJE
Plan to Share IPD:Yes
Plan Description:The individual participant data has not been finalized yet. The investigators intent to share the full anonymized dataset, study protocol and statistical analysis plan upon request after publication of the results. Informed consent forms will not be shared.
Supporting Materials:Study Protocol
Supporting Materials:Statistical Analysis Plan (SAP)
Time Frame:The data will become available around july 2024 (after publication of the results). The data will be available for 15 years.
Access Criteria:Upon reasonable request.
Responsible Party dr. Laura C. G. de Graaff-Herder, Erasmus Medical Center
Study Sponsor  ICMJE Erasmus Medical Center
Collaborators  ICMJE
  • Pfizer
  • Foundation for Prader-Willi Research
  • Prader-Willi Fonds
Investigators  ICMJE
Principal Investigator:Laura de Graaff, MD, PhDErasmus MC, University Medical Center Rotterdam
PRS Account Erasmus Medical Center
Verification Date July 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP