Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201

Overview

This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201 (treatment study). No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from AVR-RD-01 gene therapy infusion.

Full Title of Study: “A Long-Term Follow-Up Study of Subjects With Fabry Disease Who Previously Received Ex-Vivo, Lentiviral Vector-Mediated Gene-Modified Autologous Cell Therapy AVR-RD-01 in Study AVRO-RD-01-201”

Study Type

  • Study Type: Observational
  • Study Design
    • Time Perspective: Prospective
  • Study Primary Completion Date: August 16, 2023

Detailed Description

Participants enrolled in the AVRO-RD-01-201 study will be offered participation in the AVRO-RD-01-LTF01 study. The Baseline visit for the AVRO-RD-01-LTF01 study will coincide with the participant's last visit in the AVRO-RD-01-201 study. Participants confirmed eligible for the AVRO-RD-01-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for 10 years (for a total of 15 years from AVR-RD-01 infusion) during which time continued safety, engraftment, and efficacy of treatment will be assessed.

Interventions

  • Other: Safety and Efficacy Assessments
    • Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.

Arms, Groups and Cohorts

  • Participants with Fabry Disease
    • This is a long-term follow-up study of participants who previously received AVR-RD-01 (single dose administration) in the AVRO-RD-01-201 treatment study. No investigational product will be administered in this study.

Clinical Trial Outcome Measures

Primary Measures

  • Incidence of clinically significant AEs and SAEs
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Number of participants with clinically relevant abnormalities, as assessed by vital signs
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Presence of anti-Alpha-galactosidase A (AGA) antibodies
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Presence of replication competent lentivirus (RCL)
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Evaluate for the presence of aberrant clonal expansion as assessed by integration site analysis (ISA)
    • Time Frame: Baseline to Year 15 post gene therapy infusion

Secondary Measures

  • Change from baseline in AGA enzyme activity level and peripheral blood leukocytes (PBLs)
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • 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)
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Change from baseline in Globotriaosylceramide (Gb3) biomarkers for Fabry disease in plasma and urine
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • 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)
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Change from baseline in eGFR
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Change from baseline in left ventricular mass index (LVMI) as assessed by cardiac magnetic resonance imaging (MRI)
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Change from baseline in abdominal pain and stool consistency as assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D)
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Change from baseline in Brief Pain Inventory-Short Form (BPI-SF) questionnaire scores
    • Time Frame: Baseline to Year 15 post gene therapy infusion
  • Change from baseline in physical and mental functioning as assessed by the Short Form 36 (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS) scores
    • Time Frame: Baseline to Year 15 post gene therapy infusion

Participating in This Clinical Trial

Inclusion Criteria

1. Subject must have been enrolled and received AVR-RD-01 in the AVRO-RD-01-201 study. Exclusion Criteria:

1. Subject has any medical, psychological, or other condition that, in the opinion of the Investigator:

  • Might interfere with the subject's participation in the study (including consenting to procedures); and/or – Poses any additional risk to the subject; and/or – Might confound the results of any study-required assessments. 2. Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-01 treatment study.

Gender Eligibility: Male

Minimum Age: 16 Years

Maximum Age: 50 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • AVROBIO
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Inderpal Panesar, MRPharmS, Study Director, AVROBIO, Inc

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