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

Overview

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 Type

  • Study Type: Observational
  • Study Design
    • Time Perspective: Prospective
  • Study Primary Completion Date: September 2016

Interventions

  • Drug: Recombinant Somatropin

Arms, Groups and Cohorts

  • Children under 2 years with Prader-Willi Syndrome

Clinical Trial Outcome Measures

Primary Measures

  • 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

Secondary Measures

  • 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

Participating in This Clinical Trial

Inclusion Criteria

  • Children under 2 years

Exclusion Criteria

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: 2 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Corporacion Parc Tauli
  • Provider of Information About this Clinical Study
    • Principal Investigator: Raquel Corripio-Collado, MD PhD – Corporacion Parc Tauli
  • Overall Contact(s)
    • Raquel Corripio, MD PhD, +34937231010, rcorripio@tauli.cat

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