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

Primary Purpose

Prader-Willi Syndrome

Status
Completed
Phase
Phase 4
Locations
Spain
Study Type
Interventional
Intervention
Growth hormone
Placebo
Sponsored by
Corporacion Parc Tauli
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Prader-Willi Syndrome focused on measuring Growth Hormone, Functional Magnetic Resonance, Strength, Body composition

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesAccepts Healthy Volunteers

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

Sites / Locations

  • Parc Taulí Hospital Universitari

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Placebo and Growth Hormone

Arm Description

2 months on placebo followed by 12 months on GH

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

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

Full Information

First Posted
July 27, 2018
Last Updated
January 26, 2021
Sponsor
Corporacion Parc Tauli
Collaborators
Parc de Salut Mar
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1. Study Identification

Unique Protocol Identification Number
NCT03616509
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
Study Type
Interventional

2. Study Status

Record Verification Date
January 2021
Overall Recruitment Status
Completed
Study Start Date
June 19, 2017 (Actual)
Primary Completion Date
July 26, 2019 (Actual)
Study Completion Date
July 26, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Corporacion Parc Tauli
Collaborators
Parc de Salut Mar

4. Oversight

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
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Prader-Willi Syndrome
Keywords
Growth Hormone, Functional Magnetic Resonance, Strength, Body composition

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
Model Description
First phase: 2m with placebo Second phase: 12 m with growth hormone
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo and Growth Hormone
Arm Type
Experimental
Arm Description
2 months on placebo followed by 12 months on GH
Intervention Type
Drug
Intervention Name(s)
Growth hormone
Other Intervention Name(s)
Genotonorm Miniquick
Intervention Description
12 months on Growth hormone, initial dose 0,2 mg per day, then adjusted by insulin like growth factor (IGF-1) level
Intervention Type
Drug
Intervention Name(s)
Placebo
Other Intervention Name(s)
Physiological serum (sodium chloride 0,9%)
Intervention Description
2 months on placebo, sodium chloride 0,9% injections
Primary Outcome Measure Information:
Title
Examine brain connectivity areas related with muscle tone maintenance
Description
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.
Time Frame
24 months
Secondary Outcome Measure Information:
Title
Evaluation of strength
Description
Evaluation of strength before and after 12 months on GH treatment
Time Frame
24 months
Title
Evaluation of body composition
Description
Evaluation of body composition by Dual energy x-ray absorptiometry (DEXA) before and after 12 months on GH treatment
Time Frame
24 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Olga Gimenez-Palop, MD, PhD
Organizational Affiliation
Corporacio Sanitària Parc Taulí
Official's Role
Principal Investigator
Facility Information:
Facility Name
Parc Taulí Hospital Universitari
City
Sabadell
State/Province
Barcelona
ZIP/Postal Code
08208
Country
Spain

12. IPD Sharing Statement

Plan to Share IPD
Undecided

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

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