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Global Growth Hormone Study in Adults With Prader-Willi Syndrome (GGAP)

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 evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04484051
Recruitment Status : Not yet recruiting
First Posted : July 23, 2020
Last Update Posted : July 23, 2020
Sponsor:
Collaborators:
Pfizer
Foundation for Prader-Willi Research
Prader-Willi Fonds
Information provided by (Responsible Party):
dr. Laura C. G. de Graaff-Herder, Erasmus Medical Center

Tracking Information
First Submitted Date  ICMJE July 15, 2020
First Posted Date  ICMJE July 23, 2020
Last Update Posted Date July 23, 2020
Estimated Study Start Date  ICMJE October 1, 2020
Estimated Primary Completion Date January 1, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 21, 2020)
Change in lean body mass [ Time Frame: 27 months ]
Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: July 21, 2020)
  • Change in fat mass [ Time Frame: 27 months ]
    Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
  • Change in bone density [ Time Frame: 27 months ]
    Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan
  • Change in cardiovascular fitness [ Time Frame: 27 months ]
    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).
  • Change in laboratory measurements [ Time Frame: 27 months ]
    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)
  • Change in muscle strength [ Time Frame: 27 months ]
    Change in muscle strength as determined with a handgrip dynamometer
  • Change in endurance [ Time Frame: 27 months ]
    Change in endurance as estimated with the treadmill stress test
  • Change in psychosocial functioning [ Time Frame: 27 months ]
    Change in psychosocial functioning as estimated with the Adult Behaviour Checklist
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: July 21, 2020)
  • Change in weight and waist-hip ratio [ Time Frame: 27 months ]
    Change in weight (in kg) and waist-hip ratio
  • Change in blood pressure [ Time Frame: 27 months ]
    Change in blood pressure (in mmHg)
  • Occurence of side-effects [ Time Frame: 27 months ]
    Occurrence of side-effects
Original Other Pre-specified Outcome Measures Same as current
 
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, PhD 0031618843010 l.degraaff@erasmusmc.nl
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.
Current Responsible Party dr. Laura C. G. de Graaff-Herder, Erasmus Medical Center
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Erasmus Medical Center
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE
  • Pfizer
  • Foundation for Prader-Willi Research
  • Prader-Willi Fonds
Investigators  ICMJE
Principal Investigator: Laura de Graaff, MD, PhD Erasmus 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