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 Identifier: NCT02205450
• Recruitment Status: Unknown
• First Posted: July 31, 2014
• Last Update Posted: July 31, 2014
• Sponsor: Corporacion Parc Tauli
• Information provided by (Responsible Party): Raquel Corripio-Collado, Corporacion Parc Tauli

Tracking Information

• First Submitted Date: July 30, 2014
• First Posted Date: July 31, 2014
• Last Update Posted Date: July 31, 2014
• Study Start Date: September 2014
• Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: July 30, 2014)

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

• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted

Current Secondary Outcome Measures (submitted: July 30, 2014)

• 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 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief 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
• 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

Brief 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. 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.

• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• Sampling Method: Non-Probability Sample
• Study Population: Hospitalary population
• Condition: Prader-Willi Syndrome
• Intervention: Drug: Recombinant Somatropin

Study Groups/Cohorts

Children under 2 years with Prader-Willi Syndrome

Intervention: Drug: Recombinant Somatropin

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: July 30, 2014): 15
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: September 2016
• Estimated Primary Completion Date: September 2016 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Children under 2 years

Exclusion Criteria:

-

Sex/Gender

• Sexes Eligible for Study: All

• Ages: up to 2 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Spain

Administrative Information

• NCT Number: NCT02205450
• Other Study ID Numbers: CPT_ENDOPED201401
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Raquel Corripio-Collado, Corporacion Parc Tauli
• Study Sponsor: Corporacion Parc Tauli
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Corporacion Parc Tauli
• Verification Date: July 2014