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Clinical trial

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

ID
Name
202309221
Description
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).
Trial arms
Trial start
2024-02-21
Estimated PCD
2037-06-01
Trial end
2037-10-01
Status
Active (not recruiting)
Treatment
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.
Arms:
Subjects who received AVR-RD-02
Size
2
Primary endpoint
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
Chane from baseline over time in platelet count
Baseline to Year 14
Eligibility criteria
Inclusion Criteria Subjects must meet all of the following inclusion criteria for participation in this study: 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.
Protocol
{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 2, 'type': 'ACTUAL'}}
Updated at
2024-07-04

1 organization

1 product

1 indication

Organization
John Bernat
Product
AVR-RD-02
Indication
Gaucher's Disease