NCT04484051

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.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for all trials

Timeline
5mo left

Started Mar 2023

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress89%
Mar 2023Oct 2026

First Submitted

Initial submission to the registry

July 15, 2020

Completed
8 days until next milestone

First Posted

Study publicly available on registry

July 23, 2020

Completed
2.7 years until next milestone

Study Start

First participant enrolled

March 24, 2023

Completed
3.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2026

Expected
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2026

Last Updated

February 16, 2023

Status Verified

February 1, 2023

Enrollment Period

3.4 years

First QC Date

July 15, 2020

Last Update Submit

February 15, 2023

Conditions

Prader-Willi Syndrome

Keywords

Prader-Willi syndromeGrowth hormoneBody compositionCardiovascular disease

Outcome Measures

Primary Outcomes (1)

  • Change in lean body mass

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

    36 months

Secondary Outcomes (6)

  • Change in fat mass

    36 months

  • Change in bone density

    36 months

  • Change in physical strength

    36 months

  • Change in laboratory measurements

    36 months

  • Change in psychosocial functioning

    36 months

  • +1 more secondary outcomes

Other Outcomes (3)

  • Change in weight and waist-hip ratio

    36 months

  • Change in blood pressure

    36 months

  • Occurence of side-effects

    36 months

Study Arms (1)

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.

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
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.

You may qualify if:

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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Erasmus MC, University Medical Center Rotterdam

Rotterdam, South Holland, 3015GD, Netherlands

Location

MeSH Terms

Conditions

Prader-Willi SyndromeCardiovascular Diseases

Condition Hierarchy (Ancestors)

Intellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesChromosome DisordersGenetic Diseases, InbornImprinting DisordersObesityOverweightOvernutritionNutrition DisordersNutritional and Metabolic Diseases

Study Officials

  • Laura de Graaff, MD, PhD

    Erasmus MC, University Medical Center Rotterdam

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Laura de Graaff, MD, PhD

CONTACT

0031618843010l.degraaff@erasmusmc.nl

Trui van Essen, MD

CONTACT

0031658869674g.vanessen@erasmusmc.nl

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, PhD

Study Record Dates

First Submitted

July 15, 2020

First Posted

July 23, 2020

Study Start

March 24, 2023

Primary Completion (Estimated)

September 1, 2026

Study Completion (Estimated)

October 1, 2026

Last Updated

February 16, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will share

The individual participant data has not been finalized yet. The investigators intent to share the full anonymized dataset, study protocol and statistical analysis plan upon request after publication of the results. Informed consent forms will not be shared.

Shared Documents
STUDY PROTOCOL, SAP
Time Frame
The data will become available around begin 2027 (after publication of the results). The data will be available for 15 years.
Access Criteria
Upon reasonable request.

Locations

NL(1)