Clincosm LogoSign in Get a demo

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

Sponsor
AVROBIO (Industry)
Overall Status
Enrolling by invitation
CT.gov ID
NCT04999059
Collaborator
(none)
12
Enrollment
2
Locations
210.8
Anticipated Duration (Months)
6
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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 for approximately 14 years.

Condition or Disease Intervention/Treatment Phase
  • Other: Safety and Efficacy Assessments

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 final Week 48 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 14 years) during which time continued safety, engraftment, and efficacy of AVR-RD-01 treatment will be assessed.

Study Design

Study Type:
Observational
Anticipated Enrollment :
12 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
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
Actual Study Start Date :
May 8, 2019
Anticipated Primary Completion Date :
Dec 1, 2036
Anticipated Study Completion Date :
Dec 1, 2036

Arms and Interventions

Arm Intervention/Treatment
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.

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

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Incidence of clinically significant AEs and SAEs [Baseline to Year 15 post gene therapy]

    2. Presence of replication competent lentivirus (RCL) and anti AGA antibodies [Baseline to Year 15 post gene therapy]

    Secondary Outcome Measures

    1. Change from baseline in AGA enzyme activity level [Baseline to Year 15 post gene therapy]

    2. Change from baseline in Gb3 biomarkers for Fabry disease [Baseline to Year 15 post gene therapy]

    3. Change from baseline in eGFR [Baseline to Year 15 post gene therapy]

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    16 Years to 50 Years
    Sexes Eligible for Study:
    Male
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:
    1. Subject must have completed 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.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Royal Melbourne Hospital Melbourne Parkville VIC Australia
    2 Royal Perth Hospital Perth Australia

    Sponsors and Collaborators

    • AVROBIO

    Investigators

    • Study Director: Medical Director, AVROBIO, Inc

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    AVROBIO
    ClinicalTrials.gov Identifier:
    NCT04999059
    Other Study ID Numbers:
    • AVRO-RD-01-LTF01
    First Posted:
    Aug 10, 2021
    Last Update Posted:
    Aug 10, 2021
    Last Verified:
    Aug 1, 2021
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Keywords provided by AVROBIO
    Fabry Disease
    Cell therapy
    Gene therapy
    Long-term follow-up
    Additional relevant MeSH terms:
    Fabry Disease

    Study Results

    No Results Posted as of Aug 10, 2021