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

NCT ID: NCT02205450

Last Updated: 2022-03-31

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 16 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2014-09-30
• Study Completion Date: 2019-07-29

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.

Conditions

Prader-Willi Syndrome

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Children under 2 years with Prader-Willi Syndrome

Recombinant Somatropin

Intervention Type: DRUG

Interventions

Recombinant Somatropin

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• Children under 2 years

Exclusion Criteria

-

• Minimum Eligible Age: 3 Months
• Maximum Eligible Age: 2 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Corporacion Parc Tauli

OTHER

Sponsor Role: lead

Responsible Party

Raquel Corripio-Collado

MD PhD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Raquel Corripio, PI

Role: PRINCIPAL_INVESTIGATOR

Corporacio PT

Locations

Corporació Sanitària Parc Taulí

Sabadell, Barcelona, Spain

Countries

Spain

Related Links

https://doi.org/10.1515/jpem-2018-0539

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

Other Identifiers

CPT_ENDOPED201401

Identifier Type: -

Identifier Source: org_study_id