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

NCT03454893 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2024-01-05

Study results available
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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 diagnosis of classic Fabry disease based on deficient alpha galactosidase A (AGA) enzyme activity who were considered treatment naïve, i.e., had not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy within 3 years of the time of Screening.

Conditions

Interventions

DRUG

AVR-RD-01

Single IV infusion of between 3 - 20 x 10\^6 CD34+ cells/kg.

Sponsors & Collaborators

  • AVROBIO

    lead INDUSTRY

Principal Investigators

  • Inderpal Panesar, MRPharmS · AVROBIO, Inc

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
16 Years
Max Age
50 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-02-21
Primary Completion
2022-03-14
Completion
2022-03-14
FDA Drug
Yes

Countries

  • United States
  • Australia
  • Brazil

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03454893 on ClinicalTrials.gov