RECRUITING

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

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Conditions

Fabry Disease

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

A multi-centre, multi-country, observational, non-interventional, retrospective and prospective (hybrid) study among Fabry disease participants treated with pegunigalsidase alfa (Elfabrio®) in routine clinical care.

Official Title

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

Quick Facts

Study Start:2024-11-06
Study Completion:2029-11
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06663358

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.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Male or female aged \> 18 years of age at the time of consent.
  2. * Genetically confirmed diagnosis of Fabry disease.
  3. * Either taking or planning to take pegunigalsidase alfa as treatment for Fabry disease.
  4. * No contraindications for cardiac magnetic resonance imaging (cMRI)
  5. * Informed consent form (ICF) signed and dated indicating the individual has been informed of and agreed to all pertinent aspects of the study and is willing to comply with all study requirements, including completion of electronic patient reported outcomes (ePROs).
  6. * Cardiac Cohort:
  7. * Evidence of Fabry disease (FD)-related heart disease including one or more of the following:
  8. * Left ventricular hypertrophy (LVH) measured by left ventricular mass index (LVMI) (g/m2) elevation above age/sex specific reference ranges.
  9. * Posterior septum wall thickness (e.g., \>=13mm) not explained by other factors (e.g., hypertension)
  10. * Low native T1 mapping on cMRI.
  11. * Typical Fabry-like scar on cMRI
  12. * Participants can receive cardiac magnetic resonance imaging (cMRI) with gadolinium enhancement as part of their SoC.
  13. * Estimated glomerular filtration rate (eGFR) \>45 mL/min/1.73 m2, assessed within the prior 6 months.
  14. * Naïve Cohort:
  15. * Most recent eGFR\>45 mL/min/1.73 m2, assessed within prior 6 months.
  16. * Male participants should have abnormal elevation in plasma lysoGb3 as assessed within 6 months prior to enrolment.
  17. * Long-Term Cohort:
  18. * Participants previously enrolled in the open label study CLI-06657AA1-04 (Previously PB-102-F60) (using pegunigalsidase alfa at a dose of 1mg/kg every 2 weeks) who have initiated or plan to initiate commercial pegunigalsidase alfa (Elfabrio®).
  1. * Contraindication to magnetic resonance imaging (MRI) including known history of hypersensitivity to gadolinium contrast agent that is not managed by the use of premedication.
  2. * Pregnant at the time of enrolment.
  3. * Presence of any medical, emotional, behavioural, or psychological condition that, in the judgment of the physician, could interfere with the ability to participate in the study.
  4. * Active participation in any interventional study for Fabry disease
  5. * Treatment regimen at the time of enrolment in the study is different from the approved 1mg/kg every two weeks (note if regimen subsequently changes during the prospective part of the study, the participants can remain in the study)
  6. * Prior participation in a pegunigalsidase alfa trial using a dose of 2 mg/kg administered every 4 weeks.
  7. * Cardiac Cohort:
  8. * History of acute myocardial infarction or congestive heart failure with reduced left ventricular (LV) ejection fraction of less than 35%.
  9. * Cerebral vascular accident (CVA) in the prior 6 months.
  10. * Chronic liver cirrhosis.
  11. * FD-unrelated heart disease (e.g., scarring due to myocardial infarction, symptomatic occlusive coronary artery disease, moderate valvular heart disease not thought to be Fabry related).
  12. * The participant is or has been treated with any investigational drug for Fabry disease within 6 months of study start or investigational gene therapy for Fabry disease at any time point in the past.
  13. * Severe cardiac fibrosis defined as more than 3 segments that each have \>50% fibrosis upon late gadolinium enhancement cMRI at any prior cMRI.
  14. * Naïve Cohort:
  15. * Prior exposure to a FD therapy (Replagal®, Fabrazyme®, and Galafold®) at any time point.
  16. * Severe cardiac fibrosis defined as more than 3 segments that each have \>50% fibrosis upon late gadolinium enhancement cMRI on any prior cMRI

Contacts and Locations

Study Contact

Medical Information Chiesi
CONTACT
(888) 661-9260
us.medical@chiesi.com

Study Locations (Sites)

Infusion Associates
Grand Rapids, Michigan, 49525
United States

Collaborators and Investigators

Sponsor: Chiesi Farmaceutici S.p.A.

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 Registration Dates

Study Start Date2024-11-06
Study Completion Date2029-11

Study Record Updates

Study Start Date2024-11-06
Study Completion Date2029-11

Terms related to this study

Additional Relevant MeSH Terms

  • Fabry Disease