Status:
TERMINATED
Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease
Lead Sponsor:
AVROBIO
Conditions:
Fabry Disease
Eligibility:
MALE
16-50 years
Phase:
PHASE1
PHASE2
Brief Summary
This was a multinational, open-label study to assess the efficacy and safety of AVR-RD-01 in approximately 15 male subjects, who were 16 years of age or older and postpubertal with a confirmed diagnos...
Detailed Description
The duration of each subject's participation in this study was approximately 64 weeks (or 1 year, 12 weeks), comprised of five study periods (Screening, Baseline, Pre-transplant, Transplant, and Post-...
Eligibility Criteria
Inclusion
- Subject was male, 16 years of age or older (18 years of age or older in the US), and post pubertal,(minimum age by region)
- Subject had a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity (defined as \< 1% of normal).
Exclusion
- Subject had a galactosidase alpha (GLA) gene mutation associated with late-onset cardiac variant Fabry disease.
- Subject had previously received ERT and/or chaperone therapy within 3 years for treatment of Fabry disease.
- Subject had tested positive for anti-AGA antibodies at the time of screening.
- Subject had eGFR \< 60 mL/min/1.73 m² (ie, chronic kidney disease \[CKD\] stage ≥ 3) at Screening.
- Subject had a prior history of myocardial infarction (MI).
- Subject had a history of coronary artery disease (CAD) with angina requiring percutaneous transluminal coronary angioplasty (with or without stent placement) and/or coronary artery bypass graft (CABG).
- Subject had a history of moderate to severe valvular heart disease requiring valve replacement.
- Subject had a history of heart failure, moderate to severe diastolic dysfunction, and/or left ventricular ejection fraction (LVEF) ≤ 45% on echocardiogram (ECHO) performed at rest at Screening.
- Subject had a history of clinically significant cardiac arrhythmia (eg, heart block \[second or third degree\], atrial fibrillation requiring therapy, ventricular fibrillation, ventricular tachycardia, supraventricular tachycardia, or cardiac arrest).
- Note \[history of intermittent atrial fibrillation not requiring treatment was allowed\].
- Subject had a prior history of stroke and/or transient ischemic attack (TIA).
- Subject had aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≥ 3 times the upper limit of normal (ULN) at Screening.
- Subject had a prior history of (or current) malignancy; the one exception is a prior history of resected basal cell carcinoma.
- Subject had previously received treatment with AVR-RD-01 or any other gene therapy.
- Other inclusion/exclusion criteria apply.
Key Trial Info
Start Date :
February 21 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 14 2022
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT03454893
Start Date
February 21 2018
End Date
March 14 2022
Last Update
January 5 2024
Active Locations (5)
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1
Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
2
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States, 15213
3
Royal Melbourne Hospital
Melbourne, Parkville VIC, Australia
4
Royal Perth Hospital
Perth, Australia