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

Type 1 Gaucher Disease Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04836377
• Other study ID #: AVRO-RD-02-LTF01
• Status: Terminated
• Start date: July 6, 2021
• Est. completion date: August 21, 2023

Study information

• Verified date: September 2022
• Source: AVROBIO
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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).

Description:

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 4
• Est. completion date: August 21, 2023
• Est. primary completion date: August 21, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 99 Years

Eligibility

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.

Exclusion Criteria:

• Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-02 treatment study.

Related Conditions & MeSH terms

• Gaucher Disease
• Type 1 Gaucher Disease

Intervention

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

Locations

Country	Name	City	State
Canada	M.A.G.I.C. Clinic Ltd	Calgary	Calgary Alberta
United States	Hackensack University Medical Center	Hackensack	New Jersey
United States	University of Iowa	Iowa City	Iowa

Sponsors (1)

Lead Sponsor	Collaborator
AVROBIO

Countries where clinical trial is conducted

United States,  Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline over time in spleen volume as assessed by abdominal Magnetic Resonance Imaging (MRI)		Baseline to Year 15 post gene therapy infusion
Primary	Baseline over time in liver volume as assessed by abdominal MRI		Baseline to Year 15 post gene therapy infusion
Primary	Baseline over time in hemoglobin		Baseline to Year 15 post gene therapy infusion
Primary	Change from Baseline over time in platelet count		Baseline to Year 15 post gene therapy infusion
Primary	Change from Baseline in plasma lyso-Gb1 levels by liquid chromatography tandem mass spectrometry (LC/MS/MS)		Baseline to Year 15 post gene therapy infusion
Primary	Incidence of newly-diagnosed malignancy, hematologic disorder, and/or immune related events/immunogenicity		Baseline to Year 15 post gene therapy infusion
Primary	Number of participants with clinically relevant abnormalities as assessed by vital signs		Baseline to Year 15 post gene therapy infusion
Primary	Number of participants with clinically relevant abnormalities, as assessed by physical examinations findings		Baseline to Year 15 post gene therapy infusion
Primary	Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests		Baseline to Year 15 post gene therapy infusion
Primary	Number of participants with clinically relevant abnormalities, as assessed by electrocardiograms (ECGs)		Baseline to Year 15 post gene therapy infusion
Secondary	Change from baseline over time in glucocerebrosidase (GCase) GCase enzyme activity level in		Baseline to Year 15 post gene therapy infusion
Secondary	Change from baseline over time in Enzyme Replacement Therapy (ERT) frequency and dosing		Baseline to Year 15 post gene therapy infusion
Secondary	Changes in Gaucher biomarker indices of Gaucher disease in chitotriosidase enzyme activity levels in plasma		Baseline to Year 15 post gene therapy infusion
Secondary	Changes in Gaucher biomarker indices of Gaucher disease in bone marrow burden (BMB) score as assessed by bone MRI		Baseline to Year 15 post gene therapy infusion
Secondary	Changes in Gaucher biomarker indices of Gaucher disease in bone mineral density (BMD) as assessed by dual-energy X-ray absorptiometry (DXA)		Baseline to Year 15 post gene therapy infusion
Secondary	Average Vector Copy Number (VCN) in bone marrow aspirate as assessed by quantitative polymerase chain reaction (qPCR) and/or droplet digital polymerase chain reaction (ddPCR)		Baseline to Year 15 post gene therapy infusion
Secondary	Change from Baseline in anti-GCase total antibodies and subsequent titers by an electrochemiluminescence method		Baseline to Year 15 post gene therapy infusion
Secondary	Changes in Gaucher disease in potential aberrant clonal expansion(s) assessed by performing insertional site analysis (ISA)		Baseline to Year 15 post gene therapy infusion
Secondary	Presence of replication competent lentivirus (RCL)		Baseline to Year 15 post gene therapy infusion