Prader-Willi SyndromeNCT02205450
Growth Hormone in Children Under 2 Years With Prader-Willi in Hospital of Sabadell
An observational study of Prader-Willi Syndrome, run by Corporacion Parc Tauli at 1 site in Spain.
Trial summary
The PWS is a genetic disease with intellectual disabilities associated with multiple manifestations in other body systems. It is characterized by hypothalamic-pituitary abnormalities with severe hypotonia during the early years of life, conditioning feeding difficulties. Read more about this study
- Who is it for?
- Ages 3 months to 2 years
- Conditions the study is for
- Prader-Willi Syndrome
- What is being tested?
- Recombinant Somatropin
- How many people
- 16 participants
- Where
- 1 siteSpain
- Study dates
- Sep 2014to 29 Jul 2019
- 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 3 months to 2 years
- All sexes
- No healthy volunteers
- Child
Study population: Hospitalary population
Who may be able to join
- Children under 2 years
Who cannot join
- -
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Experience With Growth Hormone (GH) in Children Under 2 Years With Prader-Willi Syndrome (PWS) in the Pediatric Endocrine Department of the Hospital of Sabadell
The PWS is a genetic disease with intellectual disabilities associated with multiple manifestations in other body systems. It is characterized by hypothalamic-pituitary abnormalities with severe hypotonia during the early years of life, conditioning feeding difficulties. Hyperphagia appears later, causing severe obesity in pre - school ages. Other endocrine abnormalities associated produce short stature, GH deficiency and hypogonadotropic hypogonadism. These patients also have varying cognitive dysfunction associated as well as learning problems, compounded by the development of psychological-psychiatric and behavioral problems language. The aetiology of GH decreased secretion of the SPW is controversial, it is known that IGF -1 levels are reduced in children and adults with PWS. The rational use of GH is derived from knowledge of comorbidities observed in PWS, which seem to be related to GH deficiency: hypotonia, altered body composition, decreased growth, even obesity.
- The GH is accepted since 2000 for the treatment of PWS. Following fatal episodes in our country, it was decided to start treatment at 2 years of age in an arbitrary manner, but not in the U.S. or France. Subsequent studies have found that GH per se is not a risk factor for mortality. The currently published data supporting the benefits of GH treatment when started between 4 and 6 months of life, even some experts advocate starting at 3 months, but due to the lack of consensus on the age of onset treatment, despite the benefits of your home at an early age before the onset of obesity often starts around 2 years of life.
HYPOTHESIS The use of GH is safe and effective in patients with PWS children under 2 years old.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Recombinant Somatropin
- Keywords
- Prader-Willi Syndrome, Growth Hormone, Children
- 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.
Recombinant SomatropinDrug
| Study group | Role | What they receive |
|---|---|---|
| Children under 2 years with Prader-Willi Syndrome | Not described Receives: Drug: Recombinant Somatropin |
What is being measured
The primary outcome is the main question the study is designed to answer.
To assess the safe use of GH in children under 2 year old with Prader Willi Syndrome
Collect any Serious Adverse Event during the length of study
Measured: Two years
Secondary outcomes (3)
Evaluate the impact of treatment with GH in kids under 2 years old on body composition
Measured: Every 3 months during 2 years
Evaluate the impact of treatment with GH in kids under 2 years old on start walking
Measured: Every 3 months during 2 years
Evaluate the impact of treatment with GH in kids under 2 years old on the speech beginning
Measured: Every 3 months during 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Jul 2014 (estimated)
- Study startSep 2014
- Primary completion29 Jul 2019
- Study completion29 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Corporacion Parc Tauli
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Raquel Corripio-Collado, Corporacion Parc Tauli
- Investigators
- Raquel Corripio, PI, Principal investigator, Corporacio PT
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02205450
- Sponsor's protocol ID
- CPT_ENDOPED201401
- Status last verified
- Mar 2022
- First submitted
- 30 Jul 2014
- Last updated
- 31 Mar 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Experience With Growth Hormone (GH) in Children Under 2 Years With Prader-Willi Syndrome (PWS) in the Pediatric Endocrine Department of the Hospital of Sabadell
- ClinicalTrials.gov ID
- NCT02205450
- Acronym / sponsor ID
- CPT_ENDOPED201401
- Conditions
- Prader-Willi Syndrome
- Keywords
- Prader-Willi Syndrome, Growth Hormone, Children
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Corporacion Parc TauliUniversity, hospital or other
- Study start
- Sep 2014
- Primary completion
- 29 Jul 2019 (actual)
- Study completion
- 29 Jul 2019 (actual)
- First posted
- 31 Jul 2014 (estimated)
- 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 PWS is a genetic disease with intellectual disabilities associated with multiple manifestations in other body systems. It is characterized by hypothalamic-pituitary abnormalities with severe hypotonia during the early years of life, conditioning feeding difficulties. Hyperphagia appears later, causing severe obesity in pre - school ages. Other endocrine abnormalities associated produce short stature, GH deficiency and hypogonadotropic hypogonadism. These patients also have varying cognitive dysfunction associated as well as learning problems, compounded by the development of psychological-psychiatric and behavioral problems language. The aetiology of GH decreased secretion of the SPW is controversial, it is known that IGF -1 levels are reduced in children and adults with PWS. The rational use of GH is derived from knowledge of comorbidities observed in PWS, which seem to be related to GH deficiency: hypotonia, altered body composition, decreased growth, even obesity.
- The GH is accepted since 2000 for the treatment of PWS. Following fatal episodes in our country, it was decided to start treatment at 2 years of age in an arbitrary manner, but not in the U.S. or France. Subsequent studies have found that GH per se is not a risk factor for mortality. The currently published data supporting the benefits of GH treatment when started between 4 and 6 months of life, even some experts advocate starting at 3 months, but due to the lack of consensus on the age of onset treatment, despite the benefits of your home at an early age before the onset of obesity often starts around 2 years of life.
HYPOTHESIS The use of GH is safe and effective in patients with PWS children under 2 years old.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Children under 2 years with Prader-Willi Syndrome | – | Drug: Recombinant Somatropin |
Who may qualify
- Ages
- 3 months to 2 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Hospitalary population
Key inclusion criteria (1 listed)
- Children under 2 years
Key exclusion criteria (1 listed)
- -
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 |
|---|---|---|
| Recombinant Somatropin | Drug | Children under 2 years with Prader-Willi Syndrome |
Primary outcome
To assess the safe use of GH in children under 2 year old with Prader Willi Syndrome
Time frame: Two years
Collect any Serious Adverse Event during the length of study
3 secondary outcomes
Evaluate the impact of treatment with GH in kids under 2 years old on body composition
Time frame: Every 3 months during 2 years
Evaluate the impact of treatment with GH in kids under 2 years old on start walking
Time frame: Every 3 months during 2 years
Evaluate the impact of treatment with GH in kids under 2 years old on the speech beginning
Time frame: Every 3 months during 2 years
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 31 Mar 2022.
- Safety and effectiveness of growth hormone therapy in infants with Prader-Willi syndrome younger than 2 years: a prospective study Raquel Corripio ORCID logo, Carla Tubau, Laura Calvo, Carme Brun, Núria Capdevila, Helena Larramona and Elisabeth GabauLink provided by the sponsor.
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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.
Raquel Corripio, PI principal investigator
Corporacio PT
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 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 1 inclusion and 1 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 31 Mar 2022. 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.
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?
To assess the safe use of GH in children under 2 year old with Prader Willi Syndrome (time frame: Two years). 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 31 Mar 2022.