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

Erasmus University

Status and phase: Not yet enrolling; Phase 3

Conditions: Prader-Willi Syndrome

Study type: Observational

Funder types: Other

Identifiers

NCT04484051

GAP

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 25 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

25 participants in 1 patient group

Routine clinical care with Genotropin treatment

Description:

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.