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NCT Number: NCT04484051

Growth Hormone Study in Adults With Prader-Willi Syndrome

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.

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Key information

Conditions

Prader-Willi Syndrome Abnormalities, Multiple Cardiovascular Diseases Chromosome Disorders Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Imprinting Disorders Intellectual Disability Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutrition Disorders Nutritional and Metabolic Diseases Obesity Overnutrition Overweight

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Erasmus MC, University Medical Center Rotterdam

Rotterdam, South Holland, 3015GD, Netherlands

Location contact

Laura de Graaff, MD, PhD

CONTACT

[email protected]

0031618843010

About this study

OBJECTIVES:

To measure the effect of GHt on physical and psychosocial health in adults with PWS.

The primary endpoint is change in lean body mass (LBM (kg)) as assessed by Dual Energy X-ray Absorptiometry (DEXA) scan. Secondary endpoints are total fat mass, bone density, physical health and psychosocial health. Also the occurrence of side-effects will be assessed. Only data that are collected as part of regular patient care will be used.

STUDY DESIGN:

Open-label prospective cohort study.

STUDY POPULATION:

Adults with PWS who have not been treated with GH during the past three years and who will start with GHt as part of regular patient care.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)

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

Treatment and study plan

Primary outcomes

  1. Change in lean body mass

    Time frame: 36 months

    Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan

Secondary outcomes

  1. Change in fat mass

    Time frame: 36 months

    Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan

  2. Change in bone density

    Time frame: 36 months

    Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan

  3. Change in physical strength

    Time frame: 36 months

    Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests

  4. Change in laboratory measurements

    Time frame: 36 months

    Changes in the following laboratory measurements:

    • Glycosylated hemoglobin (mmol/mol)
    • Total cholesterol (mmol/L)
    • Low-density lipoprotein cholesterol (mmol/L)
    • High-density lipoprotein cholesterol (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)

  5. Change in psychosocial functioning

    Time frame: 36 months

    Change in psychosocial functioning as estimated with the Adult Behaviour Checklist

  6. Change in caregiver burden

    Time frame: 36 months

    Change in caregiver burden as estimated with the Zarit Burden Interview

Other outcomes

  1. Change in weight and waist-hip ratio

    Time frame: 36 months

    Change in weight (in kg) and waist-hip ratio

  2. Change in blood pressure

    Time frame: 36 months

    Change in blood pressure (in mmHg)

  3. Occurence of side-effects

    Time frame: 36 months

    Occurrence of side-effects

Study contacts

Contact information is provided by the study sponsor or research team.

Laura de Graaff, MD, PhD

CONTACT

[email protected]

0031618843010

Trui van Essen, MD

CONTACT

[email protected]

0031658869674

Sponsors and collaborators

Lead sponsor

Erasmus Medical Center

Other

Collaborators

  • Foundation for Prader-Willi Research
  • Prader-Willi Fonds

Registry information

Official study title

Growth Hormone Study in Adults With Prader-Willi Syndroom

Acronym: GAP

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Jul 23, 2020
Registry last updated
Feb 16, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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