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Active, Not Recruiting

NCT Number: NCT06488261

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

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).

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Gaucher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases Sphingolipidoses

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

About this study

Subjects who received AVR-RD-02 in the preceding treatment study (NCT04145037) and who meet all eligibility criteria may participate. Subjects will be asked to return for study visits annually for 14 years to assess measures of safety and persistence of AVR-RD-02 treatment.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Subjects must meet all of the following inclusion criteria for participation in this study:

  • 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

Exclusion criteria

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.

Treatment and study plan

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.

Primary outcomes

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

    Time frame: Baseline to Year 14

  2. Change from baseline over time in hemoglobin concentration

    Time frame: Baseline to Year 14

  3. Change from baseline over time in platelet count

    Time frame: Baseline to Year 14

Secondary outcomes

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

    Time frame: Baseline to Year 14

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

    Time frame: Baseline to Year 14

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

    Time frame: Baseline to Year 14

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

    Time frame: Baseline to Year 14

  5. Presence of replication competent lentivirus (RCL)

    Time frame: Baseline to Year 14

Sponsors and collaborators

Lead sponsor

John Bernat

Other

Registry information

Official study title

A Long-Term Follow-Up Safety Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

Important dates

Study start
2024
Primary completion
2037
Study completion
2037
First posted
Jul 5, 2024
Registry last updated
Jul 22, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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