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

NCT02205450 | Completed

• 1 other identifier: CPT_ENDOPED201401
• Study Type: observational
• Target: 16
• Locations: 1 country
• Sites: 1

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

Keywords

Prader-Willi Syndrome; Growth Hormone; Children

Outcome Measures

Primary Outcomes (1)

• To assess the safe use of GH in children under 2 year old with Prader Willi Syndrome

  Collect any Serious Adverse Event during the length of study

  Two years

Secondary Outcomes (3)

• Evaluate the impact of treatment with GH in kids under 2 years old on body composition

  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 the speech beginning

  Every 3 months during 2 years

Study Arms (1)

Children under 2 years with Prader-Willi Syndrome

Drug: Recombinant Somatropin

Interventions

Recombinant Somatropin DRUG

Children under 2 years with Prader-Willi Syndrome

Eligibility Criteria

• Age: 3 Months - 2 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Hospitalary population

You may qualify if:

• Children under 2 years

Sponsors & Collaborators

• Corporacion Parc Tauli: lead

Study Sites (1)

Corporació Sanitària Parc Taulí

Sabadell, Barcelona, 08208, Spain

Location

Related Links

• 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

MeSH Terms

Conditions

Prader-Willi Syndrome

Interventions

Growth Hormone

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; Obesity; Overweight; Overnutrition; Nutrition Disorders; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Pituitary Hormones, Anterior; Pituitary Hormones; Peptide Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Raquel Corripio, PI

  Corporacio PT

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD PhD

Study Record Dates

• First Submitted: July 30, 2014
• First Posted: July 31, 2014
• Study Start: September 1, 2014
• Primary Completion: July 29, 2019
• Study Completion: July 29, 2019
• Last Updated: March 31, 2022

Record last verified: 2022-03

Locations

ES (1)