Growth Hormone in Children Under 2 Years With Prader-Willi in Hospital of Sabadell

Prader-Willi Syndrome Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02205450
• Other study ID #: CPT_ENDOPED201401
• Status: Completed
• Start date: September 2014
• Est. completion date: July 29, 2019

Study information

• Verified date: March 2022
• Source: Corporacion Parc Tauli
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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. 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 16
• Est. completion date: July 29, 2019
• Est. primary completion date: July 29, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Months to 2 Years

Eligibility

Inclusion Criteria:

• Children under 2 years

Exclusion Criteria:

-

Related Conditions & MeSH terms

• Prader-Willi Syndrome
• Syndrome

Intervention

Drug:
• Recombinant Somatropin

Locations

Country	Name	City	State
Spain	Corporació Sanitària Parc Taulí	Sabadell	Barcelona

Sponsors (1)

Lead Sponsor	Collaborator
Corporacion Parc Tauli

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Two years
Secondary	Evaluate the impact of treatment with GH in kids under 2 years old on body composition		Every 3 months during 2 years
Secondary	Evaluate the impact of treatment with GH in kids under 2 years old on start walking		Every 3 months during 2 years
Secondary	Evaluate the impact of treatment with GH in kids under 2 years old on the speech beginning		Every 3 months during 2 years

External Links

•   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 Gabau