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

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Recombinant Somatropin

• Registration Number: NCT02205450
• Lead Sponsor: Corporacion Parc Tauli

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 available

Study Timeline

• Study First Submitted: 2014-07-30
• Study First Submitted QC: 2014-07-30
• Study First Posted: 2014-07-31
• Study Start: 2014-09
• Primary Completion: 2019-07-29
• Study Completion: 2019-07-29
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-15
• Last Update Posted: 2022-03-31

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 16

Inclusion Criteria

• Children under 2 years

Exclusion Criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Children under 2 years with Prader-Willi Syndrome	Recombinant Somatropin	-

Primary Outcome Measures

Name	Time	Method
To assess the safe use of GH in children under 2 year old with Prader Willi Syndrome	Two years	Collect any Serious Adverse Event during the length of study

Secondary Outcome Measures

Name	Time	Method
Evaluate the impact of treatment with GH in kids under 2 years old on the speech beginning	Every 3 months during 2 years
Evaluate the impact of treatment with GH in kids under 2 years old on start walking	Every 3 months during 2 years
Evaluate the impact of treatment with GH in kids under 2 years old on body composition	Every 3 months during 2 years

Trial Locations

Locations (1)

Corporació Sanitària Parc Taulí; 🇪🇸 Sabadell, Barcelona, Spain