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

Phase 4

Completed

• Conditions: Prader-Willi Syndrome
• Interventions: Drug: Growth hormone; Drug: Placebo

• Registration Number: NCT03616509
• Lead Sponsor: Corporacion Parc Tauli

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-06-19
• Study First Submitted: 2018-07-27
• Study First Submitted QC: 2018-08-03
• Study First Posted: 2018-08-06
• Primary Completion: 2019-07-26
• Study Completion: 2019-07-26
• Status Verified: 2021-01
• Last Update Submitted: 2021-01-26
• Last Update Posted: 2021-01-27

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

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

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Exclusion Criteria

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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Placebo and Growth Hormone	Placebo	2 months on placebo followed by 12 months on GH
Placebo and Growth Hormone	Growth hormone	2 months on placebo followed by 12 months on GH

Primary Outcome Measures

Name	Time	Method
Examine brain connectivity areas related with muscle tone maintenance	24 months	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.

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Parc Taulí Hospital Universitari; 🇪🇸 Sabadell, Barcelona, Spain