Prader-Willi SyndromeGAPNCT04484051
Growth Hormone Study in Adults With Prader-Willi Syndrome
An observational study of Prader-Willi Syndrome, run by Erasmus Medical Center at 1 site in Netherlands.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Prader-Willi Syndrome
- Type of treatment
- Injection or infusion
- How many people
- 25 participants
- Where
- 1 siteNetherlands
- Study dates
- 24 Mar 2023to 1 Oct 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older 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.
Who may be able to join
- The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Netherlands1 site
Erasmus MC, University Medical Center Rotterdam
Rotterdam, South Holland, 3015GD
Laura de Graaff, MD, PhD, 0031618843010, l.degraaff@erasmusmc.nl
About this study
Official title: Growth Hormone Study in Adults With Prader-Willi Syndroom
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.
Read the 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
- Keywords
- Prader-Willi syndrome, Growth hormone, Body composition, Cardiovascular disease
- Medical subject headings
- Prader-Willi Syndrome, Cardiovascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in lean body mass
Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
Measured: 36 months
Secondary outcomes (6)
Change in fat mass
Change in fat mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
Measured: 36 months
Change in bone density
Change in bone density (in T-score) as measured by Dual Energy X-ray Absorptiometry scan
Measured: 36 months
Change in physical strength
Change in physical strenght as measured by handgrip dynamometer and sit-to-stand tests
Measured: 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)
Measured: 36 months
Change in psychosocial functioning
Change in psychosocial functioning as estimated with the Adult Behaviour Checklist
Measured: 36 months
Change in caregiver burden
Change in caregiver burden as estimated with the Zarit Burden Interview
Measured: 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2020
- Study start24 Mar 2023 (estimated)
- Primary completion1 Sep 2026 (estimated)
- Study completion1 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- More trials run by Erasmus Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Erasmus Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Foundation for Prader-Willi Research, Prader-Willi Fonds
- Responsible party
- Principal investigator, dr. Laura C. G. de Graaff-Herder, Erasmus Medical Center
- Investigators
- Laura de Graaff, MD, PhD, Principal investigator, Erasmus MC, University Medical Center Rotterdam
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04484051
- Sponsor's protocol ID
- GAP
- Acronym
- GAP
- Status last verified
- Feb 2023
- First submitted
- 15 Jul 2020
- Last updated
- 16 Feb 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 16 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Growth Hormone Study in Adults With Prader-Willi Syndroom
- ClinicalTrials.gov ID
- NCT04484051
- Acronym / sponsor ID
- GAP / GAP
- Conditions
- Prader-Willi Syndrome
- Keywords
- Prader-Willi syndrome, Growth hormone, Body composition, Cardiovascular disease
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Erasmus Medical CenterUniversity, hospital or other
- Collaborators
- Foundation for Prader-Willi Research, Prader-Willi Fonds
- Study start
- 24 Mar 2023 (estimated)
- Primary completion
- 1 Sep 2026 (estimated)
- Study completion
- 1 Oct 2026 (estimated)
- First posted
- 23 Jul 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older 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.
Key inclusion criteria (1 listed)
- The patient is diagnosed with Prader-Willi syndrome (genetically confirmed)
Key exclusion criteria (7 listed)
- Non cooperative behaviour
- Pregnancy
- Known malignancies
- Poorly controlled diabetes (HbA1c > 64 mmol/mol (8%))
- Untreated obstructive sleep apnea (apnea-hypopnea index > 5)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in lean body mass
Time frame: 36 months
Change in lean body mass (in kg) as measured by Dual Energy X-ray Absorptiometry scan
6 secondary outcomes
Change in fat mass
Time frame: 36 months
Change in bone density
Time frame: 36 months
Change in physical strength
Time frame: 36 months
Change in laboratory measurements
Time frame: 36 months
Change in psychosocial functioning
Time frame: 36 months
Change in caregiver burden
Time frame: 36 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Feb 2023.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Laura de Graaff, MD, PhD principal investigator
Erasmus MC, University Medical Center Rotterdam
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Prader-Willi Syndrome, run by Erasmus Medical Center at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Laura de Graaff, MD, PhD (0031618843010, l.degraaff@erasmusmc.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in lean body mass (time frame: 36 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Feb 2023.