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

Tracking Information
First Submitted Date  ICMJE July 27, 2018
First Posted Date  ICMJE August 6, 2018
Last Update Posted Date January 27, 2021
Actual Study Start Date  ICMJE June 19, 2017
Actual Primary Completion Date July 26, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (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  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE GH in Adults With PWS, Effect on Hypotonia Evaluated by Functional MRI, Relationship With Strength and Body Composition
Official Title  ICMJE 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  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE 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  ICMJE Prader-Willi Syndrome
Intervention  ICMJE
  • 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  ICMJE Experimental: Placebo and Growth Hormone
2 months on placebo followed by 12 months on GH
Interventions:
  • Drug: Growth hormone
  • Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 3, 2018)
30
Original Actual Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE July 26, 2019
Actual Primary Completion Date July 26, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE Yes
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Spain
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03616509
Other Study ID Numbers  ICMJE 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  ICMJE
Plan to Share IPD: Undecided
Current Responsible Party Olga Gimenez-Palop, Corporacion Parc Tauli
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Corporacion Parc Tauli
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Parc de Salut Mar
Investigators  ICMJE
Principal Investigator: Olga Gimenez-Palop, MD, PhD Corporacio Sanitària Parc Taulí
PRS Account Corporacion Parc Tauli
Verification Date January 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP