Contact SCGE




Gene Therapy Trial Report

Summary

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease


NCTID NCT03454893 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Fabry Disease
Disease Ontology Term DOID:14499
Compound Name AVR-RD-01
Sponsor AVROBIO
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 15 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant GLA
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Dose 1 Target range: 3-20E6 CD34+ cells/kg
Dose 2 Dose range: 3.1 - 13.8E6 CD34+ cells/kg (reported in IIT trial)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2017-12-20
Completion Date 2022-03-14
Last Update 2024-01-05

Participation Criteria


Eligible Age 16 Years - 50 Years
Standard Ages Child, Adult
Sexes Eligible for Study MALE
Eligibility Criteria
Inclusion Criteria: 1. 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) 2. Subject had a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity (defined as \< 1% of normal). Exclusion Criteria: 1. Subject had a galactosidase alpha (GLA) gene mutation associated with late-onset cardiac variant Fabry disease. 2. Subject had previously received ERT and/or chaperone therapy within 3 years for treatment of Fabry disease. 3. Subject had tested positive for anti-AGA antibodies at the time of screening. 4. Subject had eGFR \< 60 mL/min/1.73 m² (ie, chronic kidney disease \[CKD\] stage ≥ 3) at Screening. 5. Subject had a prior history of myocardial infarction (MI). 6. 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). 7. Subject had a history of moderate to severe valvular heart disease requiring valve replacement. 8. 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. 9. 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 \[history of intermittent atrial fibrillation not requiring treatment was allowed\]. 10. Subject had a prior history of stroke and/or transient ischemic attack (TIA). 11. Subject had aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) ≥ 3 times the upper limit of normal (ULN) at Screening. 12. Subject had a prior history of (or current) malignancy; the one exception is a prior history of resected basal cell carcinoma. 13. Subject had previously received treatment with AVR-RD-01 or any other gene therapy. Other inclusion/exclusion criteria apply.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 5
Locations United States,Brazil,Australia

Regulatory Information


Has US IND True
FDA Designations Orphan Drug Designation
Recent Updates Fabry program was discontinued in Jan 2022

Resources/Links