Growth Hormone Study in Adults With Prader-Willi Syndrome

Prader-Willi Syndrome Clinical Trial

— GAP  

Official title:

Growth Hormone Study in Adults With Prader-Willi Syndroom

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT04484051
• Other study ID #: GAP
• Status: Not yet recruiting
• Start date: March 24, 2023
• Est. completion date: October 1, 2026

Study information

• Verified date: February 2023
• Source: Erasmus Medical Center
• Contact: Laura de Graaff, MD, PhD
• Phone: 0031618843010
• Email: l.degraaff[@]erasmusmc.nl
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 25
• Est. completion date: October 1, 2026
• Est. primary completion date: September 1, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Locations

Country	Name	City	State
Netherlands	Erasmus MC, University Medical Center Rotterdam	Rotterdam	Zuid-Holland

Sponsors (3)

Lead Sponsor	Collaborator
Erasmus Medical Center	Foundation for Prader-Willi Research, Prader-Willi Fonds

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Change in weight and waist-hip ratio	Change in weight (in kg) and waist-hip ratio	36 months
Other	Change in blood pressure	Change in blood pressure (in mmHg)	36 months
Other	Occurence of side-effects	Occurrence of side-effects	36 months
Primary	Change in lean body mass	Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan	36 months
Secondary	Change in fat mass	Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan	36 months
Secondary	Change in bone density	Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan	36 months
Secondary	Change in physical strength	Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests	36 months
Secondary	Change in laboratory measurements	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)	36 months
Secondary	Change in psychosocial functioning	Change in psychosocial functioning as estimated with the Adult Behaviour Checklist	36 months
Secondary	Change in caregiver burden	Change in caregiver burden as estimated with the Zarit Burden Interview	36 months