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

NCT04999059 | Terminated FDA Drug

• 1 other identifier: AVRO-RD-01-LTF01
• Study Type: observational
• Target: 5
• Locations: 2 countries
• Sites: 3

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 up to 15 years from AVR-RD-01 gene therapy infusion.

Conditions

Fabry Disease

Keywords

Fabry Disease; Cell therapy; Gene therapy; Long-term follow-up; Lysosomal storage disorder; Lenti-viral

Outcome Measures

Primary Outcomes (6)

• Incidence of clinically significant AEs and SAEs

  Baseline to Year 15 post gene therapy infusion

• Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests

  Baseline to Year 15 post gene therapy infusion

• Number of participants with clinically relevant abnormalities, as assessed by vital signs

  Baseline to Year 15 post gene therapy infusion

• Presence of anti-Alpha-galactosidase A (AGA) antibodies

  Baseline to Year 15 post gene therapy infusion

• Presence of replication competent lentivirus (RCL)

  Baseline to Year 15 post gene therapy infusion

• Evaluate for the presence of aberrant clonal expansion as assessed by integration site analysis (ISA)

  Baseline to Year 15 post gene therapy infusion

Secondary Outcomes (9)

• Change from baseline in AGA enzyme activity level and peripheral blood leukocytes (PBLs)

  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)

  Baseline to Year 15 post gene therapy infusion

• Change from baseline in Globotriaosylceramide (Gb3) biomarkers for Fabry disease in plasma and urine

  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)

  Baseline to Year 15 post gene therapy infusion

• Change from baseline in eGFR

  Baseline to Year 15 post gene therapy infusion

Study Arms (1)

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

Interventions

Safety and Efficacy Assessments OTHER

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

Also known as: Gene Therapy Intervention

Participants with Fabry Disease

Eligibility Criteria

• Age: 16 Years - 50 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

Subjects who received AVR-RD-01 in the preceding treatment study, meet all eligibility criteria, and agree to comply with the study visit schedule and procedures.

You may qualify if:

• \. Subject must have been enrolled and received AVR-RD-01 in the AVRO-RD-01-201 study.

You may not qualify if:

• 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.
• Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-01 treatment study.

Sponsors & Collaborators

• AVROBIO: lead

Study Sites (3)

Royal Melbourne Hospital

Melbourne, Parkville VIC, Australia

Location

Royal Perth Hospital

Perth, Australia

Location

Hospital de Clinicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, 90420-020, Brazil

Location

Biospecimen

Retention: SAMPLES WITH DNA

Blood sample collections including serum chemistry (and electrolytes), hematology, and anti-AGA antibodies, and urinalysis. Bone marrow aspirate for efficacy, and reproductive testing.

MeSH Terms

Conditions

Fabry Disease; Lysosomal Storage Diseases

Interventions

Safety

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Accident Prevention; Accidents; Public Health; Environment and Public Health

Study Officials

• Inderpal Panesar, MRPharmS

  AVROBIO, Inc

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 4, 2021
• First Posted: August 10, 2021
• Study Start: May 8, 2019
• Primary Completion: August 16, 2023
• Study Completion: August 16, 2023
• Last Updated: August 31, 2023

Record last verified: 2022-10

Locations

BR (1); AU (2)