Trial intelligence / Profile preview

AVR-RD-01 in Treatment-Naive Classic Fabry Disease

NCT03454893Phase 2

Key facts

Status: Terminated
Start: Feb 2018
Primary completion: Mar 2022
Countries: 3
Sponsor: AVROBIO, Inc.
Modality: Gene Editing, Gene Silencing, Stem Cell Therapies

Drugs: AVR-RD-01
Conditions: Fabry disease (FD)

Overview

Phase 2, open-label, single-arm trial of AVR-RD-01 in Fabry disease (FD), sponsored by AVROBIO, Inc. Primary endpoints: Incidence of and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs) at 48 weeks, Change From Baseline in Immunogenicity of AVR-RD-01 at 48 weeks and 3 more. Started Feb 2018; status: terminated.

Design

1 arm

Study design: Non-randomizedOpen-label

Single Assignment AVR-RD-01
Single IV infusion of AVR-RD-01

Who it enrols

Male subjects 16+ with classic Fabry disease (deficient AGA activity) who are treatment-naive (no ERT/chaperone in last 3 years). Excludes those with anti-AGA antibodies, severe kidney/heart disease, or prior gene therapy.

Endpoints

Primary

Incidence of and Severity of Adverse Events (AEs) and Serious Adverse Events (SAEs)48 weeks
Change From Baseline in Immunogenicity of AVR-RD-0148 weeks
Presence of Replication Competent Lentivirus (RCL)48 weeks
Evaluation of Aberrant Clonal Expansion48 weeks
Change From Baseline in the Average Number of Gb3 Inclusions (ie, Myelinosomes) Per Kidney Peritubular Capillary (PTC) Per Subject48 weeks

Secondary

Average Vector Copy Number (VCN) in Peripheral Blood Leukocytes48 weeks
Change From Baseline (CFB) in AGA Enzyme Activity Level in Plasma and Peripheral Blood Leukocytes (PBLs)48 weeks
Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Plasma48 weeks
Change From Baseline in Globotriaosylceramide (Gb3) Biomarkers for Fabry Disease in Urine48 weeks
Change From Baseline in Substrate (i.e. Gb3) in Skin Biopsy48 weeks
Change From Baseline in Renal Function as Assessed by Measured Glomerular Filtration Rate (mGFR)48 weeks
Change From Baseline in Renal Function as Assessed by Estimated Glomerular Filtration Rate (eGFR)48 weeks
Change From Baseline in Renal Function as Assessed by Urine Total Protein Levels48 weeks

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