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Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (MyFABT1)

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ClinicalTrials.gov Identifier: NCT02956954
Recruitment Status : Recruiting
First Posted : November 7, 2016
Last Update Posted : May 30, 2018
Sponsor:
Information provided by (Responsible Party):
University Hospital, Rouen

Brief Summary:

Anderson Fabry disease (AFD) is an X-linked lysosomal storage disorder caused by a deficiency of the enzyme alpha-galactosidase. AFD can involve various organs and lead to a series of clinical abnormalities. Left ventricular hypertrophy in middle-aged men is one of its life threatening complications. It was shown that pending the absence of myocardial replacement fibrosis, substitution therapy could improve myocardial morphology and function as well as exercise capacity. Today, there is no available marker of the efficacy of the treatment on the heart morphology and function.

The T1 time (or longitudinal relaxation time) is one of the major components of the image formation in Magnetic Resonance Imaging (along with T2 time and proton density). Several techniques have been described to assess the myocardial T1-time.

One of them called MOLLI (Modified Look Locker Inversion Recovery), was made available in research centres by the Siemens company. In a study published in 2013, Sado et al. showed in a series of various conditions (hypertension, AFD, hypertrophic cardiomyopathy, AL amyloidosis, aortic stenosis and healthy volunteers) that a septal T1 below a threshold of 940ms could discriminate AFD patients. No overlap was shown with other conditions in this study. Our experience with T1 mapping supports that finding (even though our threshold could be slightly different), and we could recently detect by MRI a number of AFD patients, some of them with hypertrophy, some others without hypertrophy. The effect of Replagal® on the T1 relaxation time remains unknown.

The purpose of that study was to follow-up the heart morphology, function and myocardial T1 relaxation time in a population of treated/untreated patients.


Condition or disease Intervention/treatment Phase
Anderson-Fabry Disease Drug: Enzyme replacement therapy (Agalsidase alpha (Replagal®)) Procedure: Magnetic Resonance Imaging Not Applicable

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®)
Actual Study Start Date : March 25, 2017
Estimated Primary Completion Date : March 2020
Estimated Study Completion Date : March 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: MRI Scans

Arm Intervention/treatment
Experimental: Patient treated with Enzyme replacement therapy
Magnetic Resonance Imaging will be done every 6 months for patient treated with Enzyme replacement therapy (Agalsidase alpha (Replagal®))
Drug: Enzyme replacement therapy (Agalsidase alpha (Replagal®))
Patient treated with Enzyme replacement therapy as usual (Agalsidase alpha (Replagal®)). The treatment is prescribed in routine and not specially for the protocol

Procedure: Magnetic Resonance Imaging
Magnetic Resonance Imaging will be assessed every 6 months during 2 years

Sham Comparator: Patient no treated with Enzyme replacement therapy
Magnetic Resonance Imaging will be done every 6 months for patient treated with Enzyme replacement therapy (Agalsidase alpha (Replagal®))
Procedure: Magnetic Resonance Imaging
Magnetic Resonance Imaging will be assessed every 6 months during 2 years




Primary Outcome Measures :
  1. Difference from baseline in Septal myocardial T1 relaxation time [ Time Frame: 24 months ]
    Septal myocardial T1 relaxation time will be evaluated using MRI for treated and untreated patient


Secondary Outcome Measures :
  1. Difference from baseline in Septal myocardial T1 relaxation time [ Time Frame: 6 months ]
    Septal myocardial T1 relaxation time will be evaluated using MRI for treated and untreated patient

  2. Difference from baseline in Septal myocardial T1 relaxation time [ Time Frame: 12 months ]
    Septal myocardial T1 relaxation time will be evaluated using MRI for treated and untreated patient

  3. Difference from baseline in Septal myocardial T1 relaxation time [ Time Frame: 18 months ]
    Septal myocardial T1 relaxation time will be evaluated using MRI for treated and untreated patient



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient with proven Anderson Fabry Disease
  • Patient with no Agalsidase alpha (Replagal®) treatment or
  • Patient with Agalsidase alpha (Replagal®) treatment ongoing

Exclusion Criteria:

  • Pace Maker / Implantable Cardiac Defibrillator
  • Claustrophobia
  • Ocular foreign body
  • Allergy to gadolinium chelates
  • Pregnancy ongoing
  • Age < 18 years l

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): NCT02956954


Contacts
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Contact: Jean-Nicolas DACHER, Pr jean-nicolas.dacher@chu-rouen.fr
Contact: Julien BLOT julien.blot@chu-rouen.fr

Locations
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France
Rouen University Hospital Recruiting
Rouen, France
Contact: Jean-Nicolas DACHER, Pr         
Principal Investigator: Jean-Nicolas DACHER, Pr         
Sponsors and Collaborators
University Hospital, Rouen
Investigators
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Principal Investigator: Jean-Nicolas DACHER, Pr Rouen University Hospital

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Responsible Party: University Hospital, Rouen
ClinicalTrials.gov Identifier: NCT02956954     History of Changes
Other Study ID Numbers: 2016/092/HP
First Posted: November 7, 2016    Key Record Dates
Last Update Posted: May 30, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by University Hospital, Rouen:
Enzyme replacement therapy

Additional relevant MeSH terms:
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Fabry Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders