Prader-Willi SyndromePhase 4NCT03616509
GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition
A Phase 4 study testing Growth hormone in people with Prader-Willi Syndrome, run by Corporacion Parc Tauli at 1 site in Spain.
Trial summary
Prader-Willi syndrome (PWS) is a genetic disorder associated with growth hormone (GH) deficiency, central hypotonia and hyperphagia that leads to life-threatening obesity. Treatment with GH in adult patients is not well stablished in guidelines of Health National System (HNS). Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Growth hormone
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 1 siteSpain
- Study dates
- 19 Jun 2017to 26 Jul 2019
- Phase
- Phase 4
- 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
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- PWS >=18 years with growth hormone deficit
- Signed informed consent by the patients or their legal guardian
Who cannot join
- Severe obesity
- No controled diabetes mellitus
- No treated obstructive sleep apnea or severe obstructive sleep apnea
- Active cancer
- Active psychosis
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Treatment With Growth Hormone in Adults With PWS, Effect on Muscular Tone Evaluated by Functional MRI, Relationship With Strength and Body Composition
Prader-Willi syndrome (PWS) is a genetic disorder associated with growth hormone (GH) deficiency, central hypotonia and hyperphagia that leads to life-threatening obesity. Treatment with GH in adult patients is not well stablished in guidelines of Health National System (HNS). The investigators has experience in the study of brain connectivity in these patients in relation to satiety. To date, there is no evidence about the effect of GH on central hypotonia (brain areas related with muscle tone maintenance). So, the main objective is to examine these anatomical areas before and one year after GH treatment.
Methodology: Structural and functional magnetic resonance imaging to 30 PWS patients before and after GH treatment and we will compare them to a control group.
Expected results: PWS group will show abnormal functional and structural connectivity in circuitry of muscle tone maintenance that will improve after GH treatment. These favorable changes and the absence of secondary effects will help to justify the use of this treatment and its inclusion in practical clinical guidelines of HNS for the management of this syndrome in the adulthood.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Growth hormone, Placebo
- Keywords
- Growth Hormone, Functional Magnetic Resonance, Strength, Body composition
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Growth hormoneDrug
Also called Genotonorm Miniquick
12 months on Growth hormone, initial dose 0,2 mg per day, then adjusted by insulin like growth factor (IGF-1) level
PlaceboDrug
Also called Physiological serum (sodium chloride 0,9%)
2 months on placebo, sodium chloride 0,9% injections
| Study group | Role | What they receive |
|---|---|---|
| Placebo and Growth Hormone | Experimental | 2 months on placebo followed by 12 months on GH Receives: Drug: Growth hormone; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Examine brain connectivity areas related with muscle tone maintenance
Examine brain connectivity areas related with muscle tone maintenance making a functional MRI while the subject is making motor maneuvers, before anf after 12 months on GH treatment.
Measured: 24 months
Secondary outcomes (2)
Evaluation of strength
Evaluation of strength before and after 12 months on GH treatment
Measured: 24 months
Evaluation of body composition
Evaluation of body composition by Dual energy x-ray absorptiometry (DEXA) before and after 12 months on GH treatment
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Aug 2018
- Study start19 Jun 2017
- Primary completion26 Jul 2019
- Study completion26 Jul 2019
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.
- 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- First phase: 2m with placebo Second phase: 12 m with growth hormone
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- Corporacion Parc Tauli
- Sponsor type
- University, hospital or other
- Collaborators
- Parc de Salut Mar
- Responsible party
- Principal investigator, Olga Gimenez-Palop, Corporacion Parc Tauli
- Investigators
- Olga Gimenez-Palop, MD, PhD, Principal investigator, Corporacio Sanitària Parc Taulí
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03616509
- Sponsor's protocol ID
- END-GH-2017
- Other IDs
- 2017-002164-41, 2017
- Status last verified
- Jan 2021
- First submitted
- 27 Jul 2018
- Last updated
- 27 Jan 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 27 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment With Growth Hormone in Adults With PWS, Effect on Muscular Tone Evaluated by Functional MRI, Relationship With Strength and Body Composition
- ClinicalTrials.gov ID
- NCT03616509
- Acronym / sponsor ID
- END-GH-2017
- Conditions
- Prader-Willi Syndrome
- Keywords
- Growth Hormone, Functional Magnetic Resonance, Strength, Body composition
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- Corporacion Parc TauliUniversity, hospital or other
- Collaborators
- Parc de Salut Mar
- Study start
- 19 Jun 2017 (actual)
- Primary completion
- 26 Jul 2019 (actual)
- Study completion
- 26 Jul 2019 (actual)
- First posted
- 6 Aug 2018 (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.
Prader-Willi syndrome (PWS) is a genetic disorder associated with growth hormone (GH) deficiency, central hypotonia and hyperphagia that leads to life-threatening obesity. Treatment with GH in adult patients is not well stablished in guidelines of Health National System (HNS). The investigators has experience in the study of brain connectivity in these patients in relation to satiety. To date, there is no evidence about the effect of GH on central hypotonia (brain areas related with muscle tone maintenance). So, the main objective is to examine these anatomical areas before and one year after GH treatment.
Methodology: Structural and functional magnetic resonance imaging to 30 PWS patients before and after GH treatment and we will compare them to a control group.
Expected results: PWS group will show abnormal functional and structural connectivity in circuitry of muscle tone maintenance that will improve after GH treatment. These favorable changes and the absence of secondary effects will help to justify the use of this treatment and its inclusion in practical clinical guidelines of HNS for the management of this syndrome in the adulthood.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- First phase: 2m with placebo Second phase: 12 m with growth hormone
| Arm | Type | Receives |
|---|---|---|
| Placebo and Growth Hormone | Experimental | Drug: Growth hormone, Drug: Placebo2 months on placebo followed by 12 months on GH |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- PWS >=18 years with growth hormone deficit
- Signed informed consent by the patients or their legal guardian
Key exclusion criteria (5 listed)
- Severe obesity
- No controled diabetes mellitus
- No treated obstructive sleep apnea or severe obstructive sleep apnea
- Active cancer
- Active psychosis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Growth hormoneAlso: Genotonorm Miniquick12 months on Growth hormone, initial dose 0,2 mg per day, then adjusted by insulin like growth factor (IGF-1) level | Drug | Placebo and Growth Hormone |
| PlaceboAlso: Physiological serum (sodium chloride 0,9%)2 months on placebo, sodium chloride 0,9% injections | Drug | Placebo and Growth Hormone |
Primary outcome
Examine brain connectivity areas related with muscle tone maintenance
Time frame: 24 months
Examine brain connectivity areas related with muscle tone maintenance making a functional MRI while the subject is making motor maneuvers, before anf after 12 months on GH treatment.
2 secondary outcomes
Evaluation of strength
Time frame: 24 months
Evaluation of body composition
Time frame: 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jan 2021.
- Individual participant dataSharing planned: Undecided
Related trials for Prader-Willi Syndrome
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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.
Olga Gimenez-Palop, MD, PhD principal investigator
Corporacio Sanitària Parc Taulí
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Growth hormone in people with Prader-Willi Syndrome, run by Corporacion Parc Tauli at 1 site in Spain.
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 2 inclusion and 5 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: finished, as of the sponsor's last update on 27 Jan 2021. 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 Spain. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Examine brain connectivity areas related with muscle tone maintenance (time frame: 24 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 27 Jan 2021.