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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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ClinicalTrials.gov Identifier: NCT03616509
Recruitment Status : Active, not recruiting
First Posted : August 6, 2018
Last Update Posted : August 6, 2018
Sponsor:
Collaborator:
Parc de Salut Mar
Information provided by (Responsible Party):
Olga Gimenez-Palop, 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.


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)
Primary Purpose: Treatment
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


Arm Intervention/treatment
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

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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Evaluation of strength [ Time Frame: 24 months ]
    Evaluation of strength before and after 12 months on GH treatment

  2. 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



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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

Information from the National Library of Medicine

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


Locations
Spain
Parc Taulí Hospital Universitari
Sabadell, Barcelona, Spain, 08208
Sponsors and Collaborators
Corporacion Parc Tauli
Parc de Salut Mar
Investigators
Principal Investigator: Olga Gimenez-Palop, MD, PhD Corporacio Sanitària Parc Taulí

Responsible Party: Olga Gimenez-Palop, Principal Investigator, Corporacion Parc Tauli
ClinicalTrials.gov Identifier: NCT03616509     History of Changes
Other Study ID Numbers: END-GH-2017
2017-002164-41 ( EudraCT Number )
First Posted: August 6, 2018    Key Record Dates
Last Update Posted: August 6, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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

Keywords provided by Olga Gimenez-Palop, Corporacion Parc Tauli:
Growth Hormone
Functional Magnetic Resonance
Strength
Body composition

Additional relevant MeSH terms:
Prader-Willi Syndrome
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Obesity
Overnutrition
Nutrition Disorders
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs