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

• ClinicalTrials.gov Identifier: NCT03616509
• Recruitment Status: Completed
• First Posted: August 6, 2018
• Last Update Posted: January 27, 2021
• Sponsor: Corporacion Parc Tauli
• Collaborator: Parc de Salut Mar
• Information provided by (Responsible Party): Olga Gimenez-Palop, Corporacion Parc Tauli

Tracking Information

• First Submitted Date: July 27, 2018
• First Posted Date: August 6, 2018
• Last Update Posted Date: January 27, 2021
• Actual Study Start Date: June 19, 2017
• Actual Primary Completion Date: July 26, 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 3, 2018)

Examine brain connectivity areas related with muscle tone maintenance [ Time Frame: 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.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: August 3, 2018)

• Evaluation of strength [ Time Frame: 24 months ]
  Evaluation of strength before and after 12 months on GH treatment
• Evaluation of body composition [ Time Frame: 24 months ]
  Evaluation of body composition by Dual energy x-ray absorptiometry (DEXA) before and after 12 months on GH treatment

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition
• Official Title: Treatment With Growth Hormone in Adults With PWS, Effect on Muscular Tone Evaluated by Functional MRI, Relationship With Strength and Body Composition

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 Provided
• Study Type: Interventional
• Study Phase: Phase 4

Study Design

Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description:

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

Masking: None (Open Label)
Primary Purpose: Treatment

• Condition: 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
  Other Name: Genotonorm Miniquick
• Drug: Placebo
  2 months on placebo, sodium chloride 0,9% injections
  Other Name: Physiological serum (sodium chloride 0,9%)

Study Arms

Experimental: Placebo and Growth Hormone

2 months on placebo followed by 12 months on GH

Interventions:

• Drug: Growth hormone
• Drug: Placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 3, 2018): 30
• Original Actual Enrollment: Same as current
• Actual Study Completion Date: July 26, 2019
• Actual Primary Completion Date: July 26, 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: Yes
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Spain

Administrative Information

• NCT Number: NCT03616509
• Other Study ID Numbers: END-GH-2017; 2017-002164-41 ( EudraCT Number )
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• 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

IPD Sharing Statement

• Plan to Share IPD: Undecided

• Current Responsible Party: Olga Gimenez-Palop, Corporacion Parc Tauli
• Original Responsible Party: Same as current
• Current Study Sponsor: Corporacion Parc Tauli
• Original Study Sponsor: Same as current
• Collaborators: Parc de Salut Mar

Investigators

• Principal Investigator: Olga Gimenez-Palop, MD, PhD; Corporacio Sanitària Parc Taulí

• PRS Account: Corporacion Parc Tauli
• Verification Date: January 2021