Growth Hormone Study in Adults With Prader-Willi Syndrome

NCT ID: NCT04484051

Last Updated: 2023-02-16

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Total Enrollment: 25 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-03-24
• Study Completion Date: 2026-10-01

Brief 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.

Detailed 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.

Conditions

Prader-Willi Syndrome

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Routine clinical care with Genotropin treatment

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.

No interventions assigned to this group

Eligibility 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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Foundation for Prader-Willi Research

OTHER

Sponsor Role: collaborator

Prader-Willi Fonds

UNKNOWN

Sponsor Role: collaborator

Erasmus Medical Center

OTHER

Sponsor Role: lead

Responsible Party

dr. Laura C. G. de Graaff-Herder

MD, PhD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Laura de Graaff, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Erasmus MC, University Medical Center Rotterdam

Locations

Erasmus MC, University Medical Center Rotterdam

Rotterdam, South Holland, Netherlands

Countries

Netherlands

Central Contacts

Laura de Graaff, MD, PhD

Role: CONTACT

l.degraaff@erasmusmc.nl

0031618843010

Trui van Essen, MD

Role: CONTACT

g.vanessen@erasmusmc.nl

0031658869674

Facility Contacts

Laura de Graaff, MD, PhD

Role: primary

l.degraaff@erasmusmc.nl

0031618843010

Other Identifiers

GAP

Identifier Type: -

Identifier Source: org_study_id