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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

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ClinicalTrials.gov Identifier: NCT02205450
Recruitment Status : Unknown
Verified July 2014 by Raquel Corripio-Collado, Corporacion Parc Tauli.
Recruitment status was:  Not yet recruiting
First Posted : July 31, 2014
Last Update Posted : July 31, 2014
Sponsor:
Information provided by (Responsible Party):
Raquel Corripio-Collado, 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.


Condition or disease Intervention/treatment
Prader-Willi Syndrome Drug: Recombinant Somatropin

Study Type : Observational
Estimated Enrollment : 15 participants
Observational Model: Cohort
Time Perspective: Prospective
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 Start Date : September 2014
Estimated Primary Completion Date : September 2016
Estimated Study Completion Date : September 2016


Group/Cohort Intervention/treatment
Children under 2 years with Prader-Willi Syndrome Drug: Recombinant Somatropin



Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Evaluate the impact of treatment with GH in kids under 2 years old on body composition [ Time Frame: Every 3 months during 2 years ]
  2. Evaluate the impact of treatment with GH in kids under 2 years old on start walking [ Time Frame: Every 3 months during 2 years ]
  3. 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 ]


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Ages Eligible for Study:   up to 2 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Hospitalary population
Criteria

Inclusion Criteria:

  • Children under 2 years

Exclusion Criteria:


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02205450


Contacts
Contact: Raquel Corripio, MD PhD +34937231010 ext 21005 rcorripio@tauli.cat

Locations
Spain
Corporació Sanitària Parc Taulí Not yet recruiting
Sabadell, Barcelona, Spain, 08208
Principal Investigator: Raquel Corripio, MD PhD         
Sponsors and Collaborators
Corporacion Parc Tauli

Responsible Party: Raquel Corripio-Collado, MD PhD, Corporacion Parc Tauli
ClinicalTrials.gov Identifier: NCT02205450     History of Changes
Other Study ID Numbers: CPT_ENDOPED201401
First Posted: July 31, 2014    Key Record Dates
Last Update Posted: July 31, 2014
Last Verified: July 2014

Keywords provided by Raquel Corripio-Collado, Corporacion Parc Tauli:
Prader-Willi Syndrome
Growth Hormone
Children

Additional relevant MeSH terms:
Prader-Willi Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs