GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition

NCT03616509 | Completed Phase 4

• 2 other identifiers: END-GH-20172017-002164-41
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

Prader-Willi syndrome (PWS) is a genetic disorder associated with growth hormone (GH) deficiency, central hypotonia and hyperphagia that leads to life-threatening obesity. Treatment with GH in adult patients is not well stablished in guidelines of Health National System (HNS). The investigators has experience in the study of brain connectivity in these patients in relation to satiety. To date, there is no evidence about the effect of GH on central hypotonia (brain areas related with muscle tone maintenance). So, the main objective is to examine these anatomical areas before and one year after GH treatment. Methodology: Structural and functional magnetic resonance imaging to 30 PWS patients before and after GH treatment and we will compare them to a control group. Expected results: PWS group will show abnormal functional and structural connectivity in circuitry of muscle tone maintenance that will improve after GH treatment. These favorable changes and the absence of secondary effects will help to justify the use of this treatment and its inclusion in practical clinical guidelines of HNS for the management of this syndrome in the adulthood.

Conditions

Prader-Willi Syndrome

Keywords

Growth Hormone; Functional Magnetic Resonance; Strength; Body composition

Outcome Measures

Primary Outcomes (1)

• Examine brain connectivity areas related with muscle tone maintenance

  Examine brain connectivity areas related with muscle tone maintenance making a functional MRI while the subject is making motor maneuvers, before anf after 12 months on GH treatment.

  24 months

Secondary Outcomes (2)

• Evaluation of strength

  24 months

• Evaluation of body composition

  24 months

Study Arms (1)

Placebo and Growth Hormone

EXPERIMENTAL

2 months on placebo followed by 12 months on GH

Drug: Growth hormone; Drug: Placebo

Interventions

Growth hormone DRUG

12 months on Growth hormone, initial dose 0,2 mg per day, then adjusted by insulin like growth factor (IGF-1) level

Also known as: Genotonorm Miniquick

Placebo and Growth Hormone

Placebo DRUG

2 months on placebo, sodium chloride 0,9% injections

Also known as: Physiological serum (sodium chloride 0,9%)

Placebo and Growth Hormone

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• PWS \>=18 years with growth hormone deficit
• Signed informed consent by the patients or their legal guardian

You may not qualify if:

• Severe obesity
• No controled diabetes mellitus
• No treated obstructive sleep apnea or severe obstructive sleep apnea
• Active cancer
• Active psychosis

Sponsors & Collaborators

• Corporacion Parc Tauli: lead
• Parc de Salut Mar: collaborator

Study Sites (1)

Parc Taulí Hospital Universitari

Sabadell, Barcelona, 08208, Spain

Location

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

• Olga Gimenez-Palop, MD, PhD

  Corporacio Sanitària Parc Taulí

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: First phase: 2m with placebo Second phase: 12 m with growth hormone

Study Record Dates

• First Submitted: July 27, 2018
• First Posted: August 6, 2018
• Study Start: June 19, 2017
• Primary Completion: July 26, 2019
• Study Completion: July 26, 2019
• Last Updated: January 27, 2021

Record last verified: 2021-01

Locations

ES (1)