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

May 25, 2018 updated by: University Hospital, Rouen

Follow-up of Myocardial T1 Relaxation Time in Patients With Anderson Fabry Disease (AFD): Impact of Treatment by Agalsidase Alpha (Replagal®)

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

25

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • France
      • Rouen, France
        • Recruiting
        • ROUEN university hospital
        • Contact:
          • Jean-Nicolas DACHER, Pr
        • Principal Investigator:
          • Jean-Nicolas DACHER, Pr

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Diagnostic
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
24 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
6 months
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
12 months
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
18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University Hospital, Rouen

Investigators

  • Principal Investigator: Jean-Nicolas DACHER, Pr, ROUEN university hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 25, 2017

Primary Completion (Anticipated)

March 1, 2020

Study Completion (Anticipated)

March 1, 2020

Study Registration Dates

First Submitted

November 3, 2016

First Submitted That Met QC Criteria

November 4, 2016

First Posted (Estimate)

November 6, 2016

Study Record Updates

Last Update Posted (Actual)

May 30, 2018

Last Update Submitted That Met QC Criteria

May 25, 2018

Last Verified

May 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2016/092/HP

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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