Clinical trial NCT06488261: Long Term Follow-Up for Safety of AVR-RD-02
Official title: A Long-Term Follow-Up Safety Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Gaucher Disease
Lead sponsor: University of Iowa
Sponsor class: OTHER
First posted: 2024-07-05
Enrollment: 2
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Subjects with Gaucher disease who previously received the investigational gene therapy product AVR-RD-02

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).

Detailed description: 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.

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.

Primary outcomes:
measure: Incidence of newly diagnosed malignancy, hematologic disorder, and/or autoimmune or rheumatologic disorder
timeFrame: Baseline to Year 14
measure: Change from baseline over time in hemoglobin concentration
timeFrame: Baseline to Year 14
measure: Chane from baseline over time in platelet count
timeFrame: Baseline to Year 14
Secondary outcomes:
measure: Change from baseline over time in glucocerebrosidase (GCase) enzyme activity in whole blood
timeFrame: Baseline to Year 14
measure: Average vector copy number (VCN) from whole blood using quantitative polymerase chain reaction (qPCR) analysis
timeFrame: Baseline to Year 14
measure: Change from baseline over time in anti-GCase total antibodies and subsequent titers
timeFrame: Baseline to Year 14
measure: Identification of potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA) from whole blood
timeFrame: Baseline to Year 14
measure: Presence of replication competent lentivirus (RCL)
timeFrame: Baseline to Year 14
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
Study officials:
affiliation: University of Iowa
name: John A. Bernat, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: