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

NCT ID: NCT06488261

Last Updated: 2025-07-22

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Total Enrollment

2 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-02-21

Study Completion Date

2037-10-31

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Gaucher Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Subjects who received AVR-RD-02

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

AVR-RD-02

Intervention Type 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.

Interventions

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AVR-RD-02

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.

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

1. 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.
2. 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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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John Bernat

OTHER

Sponsor Role lead

Responsible Party

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John Bernat

Clinical Associate Professor

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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John A. Bernat, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

University of Iowa

Locations

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University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States

Site Status

Countries

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United States

Other Identifiers

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202309221

Identifier Type: -

Identifier Source: org_study_id

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