Growth Hormone Study in Adults With Prader-Willi Syndrome

ConditionPrader-Willi Syndrome

Trial statusNot yet recruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age18+

SponsorErasmus Medical Center

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

Non cooperative behaviour

Pregnancy

Known malignancies

Poorly controlled diabetes (HbA1c > 64 mmol/mol (8%))

Trial 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.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

25 Participants

are grouped into 1 trial group

Group A: Routine clinical care with Genotropin treatment

Trial outcomes

Primary outcomes

Change in lean body mass

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

Time frame

36 months

Secondary outcomes

Change in fat mass

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

Time frame

36 months

Change in bone density

Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan

Time frame

36 months

Change in physical strength

Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests

Time frame

36 months

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)

Time frame

36 months

Other outcomes

Change in weight and waist-hip ratio

Change in weight (in kg) and waist-hip ratio

Time frame

36 months

Change in blood pressure

Change in blood pressure (in mmHg)

Time frame

36 months

Occurence of side-effects

Occurrence of side-effects

Time frame

36 months

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Erasmus Medical Center

Lead sponsor

Foundation for Prader-Willi Research

Collaborator

Prader-Willi Fonds

Collaborator

Source ClinicalTrials.gov

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