Clinical trial NCT04484051: Growth Hormone Study in Adults With Prader-Willi Syndrome
Official title: Growth Hormone Study in Adults With Prader-Willi Syndroom
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Prader-Willi Syndrome
Keywords: Prader-Willi syndrome
Growth hormone
Body composition
Cardiovascular disease
Lead sponsor: Erasmus Medical Center
Sponsor class: OTHER
Collaborators: name: Foundation for Prader-Willi Research
name: Prader-Willi Fonds
First posted: 2020-07-23
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_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.

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.

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

Primary outcomes:
measure: Change in lean body mass
timeFrame: 36 months
Secondary outcomes:
measure: Change in fat mass
timeFrame: 36 months
measure: Change in bone density
timeFrame: 36 months
measure: Change in physical strength
timeFrame: 36 months
measure: Change in laboratory measurements
timeFrame: 36 months
measure: Change in psychosocial functioning
timeFrame: 36 months
measure: Change in caregiver burden
timeFrame: 36 months
Other outcomes:
measure: Change in weight and waist-hip ratio
timeFrame: 36 months
measure: Change in blood pressure
timeFrame: 36 months
measure: Occurence of side-effects
timeFrame: 36 months
Locations:
city: Rotterdam
country: Netherlands
facility: Erasmus MC, University Medical Center Rotterdam
state: Zuid-Holland
Study officials:
affiliation: Erasmus MC, University Medical Center Rotterdam
name: Laura de Graaff, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: