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

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

TERMINATED

Total Enrollment

4 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-07-06

Study Completion Date

2023-08-21

Brief Summary

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This is a multicenter, multinational long-term follow-up study to assess the long-term safety and durability of effect of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).

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Detailed Description

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Subjects who received AVR-RD-02 in the preceding treatment study and who meet all eligibility criteria may participate in the AVRO-RD-02-LTF01 study. For individual participating subjects, the Baseline visit for the AVRO-RD-02-LTF01 study will coincide with the subject's last visit in the preceding treatment study, whenever feasible. Subjects will be asked to return for study visits at approximately 6-month intervals for the first 4 years of the study and then annually thereafter for 10 years (for a total follow-up of 14 years in this study), during which time periodic safety and efficacy assessments will be performed to assess measures of safety, engraftment, and clinical response after AVR-RD-02 treatment.

Conditions

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Type 1 Gaucher Disease

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Subjects with Gaucher 1 Disease

This is a long-term follow-up study of subjects who previously received AVR-RD-02 (single dose administration) in a preceding treatment study. No investigational product will be administered in this study.

Safety and efficacy assessments

Intervention Type OTHER

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

Interventions

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Safety and efficacy assessments

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

Intervention Type OTHER

Other Intervention Names

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Gene Therapy Intervention

Eligibility Criteria

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Inclusion Criteria

* Subject must have been enrolled and received AVR-RD-02 as single dose administration
* Subject must be willing and able to provide written informed consent for the AVRO-RD-02-LTF01 study in accordance with applicable regulations and guidelines and to comply with all study visits and procedures.