Growth Hormone Study in Adults With Prader-Willi Syndrome | NCT04484051

Studies
Growth Hormone Study in Adults With Prader-Willi Syndrome

NCT04484051

Sponsor

Erasmus Medical Center

Status

Not yet recruiting

Last Update Posted

Feb 16, 2023Actual

Enrollment

25Estimated

Phase

Not provided

Conditions

Prader-Willi Syndrome

Interventions

Not provided

Countries

Netherlands

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Feb 2023

Overall Recruitment Status or Expanded Access Status

Not yet recruiting

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Mar 24, 2023Estimated

Primary Completion Date

Sep 1, 2026Estimated

Completion Date

Oct 1, 2026Estimated

First Submitted Date

Jul 15, 2020

First Submission Date that Met QC Criteria

Jul 21, 2020

First Posted Date

Jul 23, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Feb 15, 2023

Last Update Posted Date

Feb 16, 2023Actual

Sponsor/Collaborators Module

Responsible Party, by Official Title

dr. Laura C. G. de Graaff-Herder, MD, PhD, Erasmus Medical CenterPrincipal Investigator

Lead Sponsor

Erasmus Medical CenterOTHER

Collaborators

Name	Class
Foundation for Prader-Willi Research	OTHER
Prader-Willi Fonds	UNKNOWN

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

25Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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.

Interventions

Not provided

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Measure	Description	Time Frame
Change in fat mass	Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan	36 months
Change in bone density	Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan

Other Outcomes

Measure	Description	Time Frame
Change in weight and waist-hip ratio	Change in weight (in kg) and waist-hip ratio	36 months
Change in blood pressure	Change in blood pressure (in mmHg)

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

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.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Name	Role	Phone	Extension	Email
Laura de Graaff, MD, PhD	Contact	0031618843010		l.degraaff@erasmusmc.nl
Trui van Essen, MD	Contact	0031658869674		g.vanessen@erasmusmc.nl

Overall Officials

Name	Affiliation	Role
Laura de Graaff, MD, PhD	Erasmus MC, University Medical Center Rotterdam	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Erasmus MC, University Medical Center Rotterdam		Rotterdam	South Holland	3015GD	Netherlands

IPD Sharing Statement Module

Plan to Share IPD

Yes

Description

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.

Types

Study Protocol
Statistical Analysis Plan (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.

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D011218	Prader-Willi Syndrome
D002318	Cardiovascular Diseases

Ancestor Terms

ID	Term
D008607	Intellectual Disability
D019954	Neurobehavioral Manifestations
D009461	Neurologic Manifestations
D009422	Nervous System Diseases

Browse Leaves

Not provided

Browse Branches

Not provided