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

Prader-Willi Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03616509
• Other study ID #: END-GH-2017
• Secondary ID: 2017-002164-41
• Status: Completed
• Phase: Phase 4
• Start date: June 19, 2017
• Est. completion date: July 26, 2019

Study information

• Verified date: January 2021
• Source: Corporacion Parc Tauli
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 30
• Est. completion date: July 26, 2019
• Est. primary completion date: July 26, 2019
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Prader-Willi Syndrome

Intervention

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

Locations

Country	Name	City	State
Spain	Parc Taulí Hospital Universitari	Sabadell	Barcelona

Sponsors (2)

Lead Sponsor	Collaborator
Corporacion Parc Tauli	Parc de Salut Mar

Country where clinical trial is conducted

Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Evaluation of strength	Evaluation of strength before and after 12 months on GH treatment	24 months
Secondary	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