GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition
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|ClinicalTrials.gov Identifier: NCT03616509|
Recruitment Status : Active, not recruiting
First Posted : August 6, 2018
Last Update Posted : August 6, 2018
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.
|Condition or disease||Intervention/treatment||Phase|
|Prader-Willi Syndrome||Drug: Growth hormone Drug: Placebo||Phase 4|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||First phase: 2m with placebo Second phase: 12 m with growth hormone|
|Masking:||None (Open Label)|
|Official Title:||Treatment With Growth Hormone in Adults With PWS, Effect on Muscular Tone Evaluated by Functional MRI, Relationship With Strength and Body Composition|
|Actual Study Start Date :||June 19, 2017|
|Estimated Primary Completion Date :||May 2, 2019|
|Estimated Study Completion Date :||May 2, 2019|
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 Name: Genotonorm Miniquick
2 months on placebo, sodium chloride 0,9% injections
Other Name: Physiological serum (sodium chloride 0,9%)
- 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.
- 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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03616509
|Parc Taulí Hospital Universitari|
|Sabadell, Barcelona, Spain, 08208|
|Principal Investigator:||Olga Gimenez-Palop, MD, PhD||Corporacio Sanitària Parc Taulí|