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

Treatment With Growth Hormone in Adults With PWS, Effect on Muscular Tone Evaluated by Functional MRI, Relationship With Strength and Body Composition

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.

Study Overview

• Status: Completed
• Conditions: Prader-Willi Syndrome
• Intervention / Treatment: Drug: Growth hormone; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 4

Contacts and Locations

Study Locations

• Spain
  • Barcelona
    • Sabadell, Barcelona, Spain, 08208
      • Parc Tauli Hospital Universitari

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Placebo and Growth Hormone; 2 months on placebo followed by 12 months on GH	Drug: Growth hormone; 12 months on Growth hormone, initial dose 0,2 mg per day, then adjusted by insulin like growth factor (IGF-1) level; Other Names: Genotonorm Miniquick; Drug: Placebo; 2 months on placebo, sodium chloride 0,9% injections; Other Names: Physiological serum (sodium chloride 0,9%)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Evaluation of strength	Evaluation of strength before and after 12 months on GH treatment	24 months
Evaluation of body composition	Evaluation of body composition by Dual energy x-ray absorptiometry (DEXA) before and after 12 months on GH treatment	24 months

Collaborators and Investigators

• Sponsor: Corporacion Parc Tauli
• Collaborators: Parc de Salut Mar
• Investigators: Principal Investigator: Olga Gimenez-Palop, MD, PhD, Corporacio Sanitaria Parc Tauli

Study record dates

Study Major Dates

• Study Start (Actual): June 19, 2017
• Primary Completion (Actual): July 26, 2019
• Study Completion (Actual): July 26, 2019

Study Registration Dates

• First Submitted: July 27, 2018
• First Submitted That Met QC Criteria: August 3, 2018
• First Posted (Actual): August 6, 2018

Study Record Updates

• Last Update Posted (Actual): January 27, 2021
• Last Update Submitted That Met QC Criteria: January 26, 2021
• Last Verified: January 1, 2021

More Information

Terms related to this study

• Keywords: Body composition; Growth Hormone; Strength; Functional Magnetic Resonance
• Additional Relevant MeSH Terms: Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Congenital Abnormalities; Overnutrition; Nutrition Disorders; Genetic Diseases, Inborn; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Obesity; Prader-Willi Syndrome; Physiological Effects of Drugs; Hormones, Hormone Substitutes, and Hormone Antagonists; Hormones
• Other Study ID Numbers: END-GH-2017; 2017-002164-41 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No