Long Term Follow-Up for Safety of AVR-RD-02

NCT06488261 | Active Not Recruiting FDA Drug

• 1 other identifier: 202309221
• Study Type: observational
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this observational study is to assess the safety and tolerability of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (3)

• Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder

  Baseline to Year 14

• Change from baseline over time in hemoglobin concentration

  Baseline to Year 14

• Change from baseline over time in platelet count

  Baseline to Year 14

Secondary Outcomes (5)

• Change from baseline over time in glucocerebrosidase (GCase) enzyme activity in whole blood

  Baseline to Year 14

• Average vector copy number (VCN) from whole blood using quantitative polymerase chain reaction (qPCR) analysis

  Baseline to Year 14

• Change from baseline over time in anti-GCase total antibodies and subsequent titers

  Baseline to Year 14

• Identification of potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA) from whole blood

  Baseline to Year 14

• Presence of replication competent lentivirus (RCL)

  Baseline to Year 14

Study Arms (1)

Subjects who received AVR-RD-02

Subjects who received AVR-RD-02 in a separate parent trial

Biological: AVR-RD-02

Interventions

AVR-RD-02 BIOLOGICAL

No study drug is administered in this study. Subjects who received AVR-RD-02 in a separate parent trial will be evaluated in this trial for long-term safety.

Subjects who received AVR-RD-02

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Subjects with Gaucher disease who previously received the investigational gene therapy product AVR-RD-02

You may qualify if:

• Subject must be willing and able to provide written informed consent for the JAB-GD-001 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures.
• Subject must have been enrolled in the preceding AVROBIO AVRO-RD-02-201 treatment study and have received AVR-RD-02 treatment

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.

Sponsors & Collaborators

• John Bernat: lead

Study Sites (1)

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

MeSH Terms

Conditions

Gaucher Disease

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; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Officials

• John A. Bernat, MD, PhD

  University of Iowa

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Clinical Associate Professor

Study Record Dates

• First Submitted: November 20, 2023
• First Posted: July 5, 2024
• Study Start: February 21, 2024
• Primary Completion (Estimated): June 1, 2037
• Study Completion (Estimated): October 1, 2037
• Last Updated: July 22, 2025

Record last verified: 2025-07

Locations

US (1)