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Enzyme Replacement Therapy

Single Assignment AVR-RD-01 for Fabry Disease

Phase 1 & 2
Waitlist Available
Research Sponsored by AvroBio
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at week 24 and week 48 post gene therapy
Awards & highlights

Summary

This trial will test a new treatment for Fabry disease, a rare inherited disorder that causes a build-up of a fatty substance in the body's cells. The trial will enroll 8-12 subjects who have not previously received treatment for the disease.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at week 24 and week 48 post gene therapy
This trial's timeline: 3 weeks for screening, Varies for treatment, and at week 24 and week 48 post gene therapy for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change From Baseline in Immunogenicity of AVR-RD-01
Change From Baseline in the Average Number of Gb3 Inclusions (ie, Myelinosomes) Per Kidney Peritubular Capillary (PTC) Per Subject
Evaluation of Aberrant Clonal Expansion
+2 more
Secondary outcome measures
Average Vector Copy Number (VCN) in Bone Marrow / Progenitor Cells as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)
Average Vector Copy Number (VCN) in Peripheral Blood Leukocytes as Assessed by Quantitative Polymerase Chain Reaction (qPCR) and/or Droplet Digital Polymerase Chain Reaction (ddPCR)
Change From Baseline (CFB) in AGA Enzyme Activity Level in Plasma and Peripheral Blood Leukocytes (PBLs)
+11 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: Single Assignment AVR-RD-01Experimental Treatment1 Intervention
AVR-RD-01 is an autologous CD34+-enriched cell fraction transduced with LV/AGA containing an RNA transcript that, after reverse transcription, results in codon-optimized cDNA that, upon its integration into the human genome, encodes for functional human AGA.

Find a Location

Who is running the clinical trial?

AvroBioLead Sponsor
3 Previous Clinical Trials
17 Total Patients Enrolled
1 Trials studying Fabry Disease
5 Patients Enrolled for Fabry Disease
AVROBIOLead Sponsor
6 Previous Clinical Trials
72 Total Patients Enrolled
1 Trials studying Fabry Disease
5 Patients Enrolled for Fabry Disease
Inderpal Panesar, MRPharmSStudy DirectorAVROBIO, Inc
3 Previous Clinical Trials
17 Total Patients Enrolled
1 Trials studying Fabry Disease
5 Patients Enrolled for Fabry Disease
~2 spots leftby Sep 2025
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