Trial | NCT04484051  Report issue

Title

Growth Hormone Study in Adults With Prader-Willi Syndrome
Growth Hormone Study in Adults With Prader-Willi Syndroom

  • Phase

    Phase 3

  • Study Type

    Observational

  • Status

    Not yet recruiting

  • Study Participants

    25
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.
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.
Study Started
Mar 24
2023
Anticipated
Primary Completion
Sep 01
2026
Anticipated
Study Completion
Oct 01
2026
Anticipated
Last Update
Feb 16
2023

Routine clinical care with Genotropin treatment None

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.

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.

Criteria 

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
No Results Posted