Clinical trial NCT02205450: Growth Hormone in Children Under 2 Years With Prader-Willi in 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Prader-Willi Syndrome
Keywords: Prader-Willi Syndrome
Growth Hormone
Children
Lead sponsor: Corporacion Parc Tauli
Sponsor class: OTHER
First posted: 2014-07-31
Enrollment: 16
Sex: ALL
Minimum age (years): 3
Maximum age (years): 2
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Hospitalary population

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.


Eligibility criteria: Inclusion Criteria:

* Children under 2 years

Exclusion Criteria:

-

Primary outcomes:
measure: To assess the safe use of GH in children under 2 year old with Prader Willi Syndrome
timeFrame: Two years
Secondary outcomes:
measure: Evaluate the impact of treatment with GH in kids under 2 years old on body composition
timeFrame: Every 3 months during 2 years
measure: Evaluate the impact of treatment with GH in kids under 2 years old on start walking
timeFrame: Every 3 months during 2 years
measure: Evaluate the impact of treatment with GH in kids under 2 years old on the speech beginning
timeFrame: Every 3 months during 2 years
Other outcomes:
Locations:
city: Sabadell
country: Spain
facility: Corporació Sanitària Parc Taulí
state: Barcelona
Study officials:
affiliation: Corporacio PT
name: Raquel Corripio, PI
role: PRINCIPAL_INVESTIGATOR
References: