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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04999059




Registration number
NCT04999059
Ethics application status
Date submitted
4/08/2021
Date registered
10/08/2021
Date last updated
31/08/2023

Titles & IDs
Public title
Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201
Scientific title
A Long-Term Follow-Up Study of Subjects With Fabry Disease Who Previously Received Ex-Vivo, Lentiviral Vector-Mediated Gene-Modified Autologous Cell Therapy AVR-RD-01 in Study AVRO-RD-01-201
Secondary ID [1] 0 0
AVRO-RD-01-LTF01
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Fabry Disease 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Metabolic and Endocrine 0 0 0 0
Metabolic disorders
Metabolic and Endocrine 0 0 0 0
Other metabolic disorders

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - Safety and Efficacy Assessments

Participants with Fabry Disease - This is a long-term follow-up study of participants who previously received AVR-RD-01 (single dose administration) in the AVRO-RD-01-201 treatment study. No investigational product will be administered in this study.


Other interventions: Safety and Efficacy Assessments
Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of clinically significant AEs and SAEs
Timepoint [1] 0 0
Baseline to Year 15 post gene therapy infusion
Primary outcome [2] 0 0
Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests
Timepoint [2] 0 0
Baseline to Year 15 post gene therapy infusion
Primary outcome [3] 0 0
Number of participants with clinically relevant abnormalities, as assessed by vital signs
Timepoint [3] 0 0
Baseline to Year 15 post gene therapy infusion
Primary outcome [4] 0 0
Presence of anti-Alpha-galactosidase A (AGA) antibodies
Timepoint [4] 0 0
Baseline to Year 15 post gene therapy infusion
Primary outcome [5] 0 0
Presence of replication competent lentivirus (RCL)
Timepoint [5] 0 0
Baseline to Year 15 post gene therapy infusion
Primary outcome [6] 0 0
Evaluate for the presence of aberrant clonal expansion as assessed by integration site analysis (ISA)
Timepoint [6] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [1] 0 0
Change from baseline in AGA enzyme activity level and peripheral blood leukocytes (PBLs)
Timepoint [1] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [2] 0 0
Average Vector Copy Number (VCN) in peripheral blood leukocytes as assessed by quantitative polymerase chain reaction (qPCR) and/or droplet digital polymerase chain reaction (ddPCR)
Timepoint [2] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [3] 0 0
Change from baseline in Globotriaosylceramide (Gb3) biomarkers for Fabry disease in plasma and urine
Timepoint [3] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [4] 0 0
Average Vector Copy Number (VCN) in bone marrow / progenitor cells as assessed by quantitative polymerase chain reaction (qPCR) and/or droplet digital polymerase chain reaction (ddPCR)
Timepoint [4] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [5] 0 0
Change from baseline in eGFR
Timepoint [5] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [6] 0 0
Change from baseline in left ventricular mass index (LVMI) as assessed by cardiac magnetic resonance imaging (MRI)
Timepoint [6] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [7] 0 0
Change from baseline in abdominal pain and stool consistency as assessed by the Diary for Irritable Bowel Syndrome Symptoms-Diarrhea (DIBSS-D)
Timepoint [7] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [8] 0 0
Change from baseline in Brief Pain Inventory-Short Form (BPI-SF) questionnaire scores
Timepoint [8] 0 0
Baseline to Year 15 post gene therapy infusion
Secondary outcome [9] 0 0
Change from baseline in physical and mental functioning as assessed by the Short Form 36 (SF-36) Physical Component Summary (PCS) and Mental Component Summary (MCS) scores
Timepoint [9] 0 0
Baseline to Year 15 post gene therapy infusion

Eligibility
Key inclusion criteria
1. Subject must have been enrolled and received AVR-RD-01 in the AVRO-RD-01-201 study.
Minimum age
16 Years
Maximum age
50 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
1. 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.
2. Subject is currently enrolled in an AVROBIO-sponsored AVR-RD-01 treatment study.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/05/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
16/08/2023
Sample size
Target
Accrual to date
Final
5
Recruitment in Australia
Recruitment state(s)
Parkville VI
Recruitment hospital [1] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment hospital [2] 0 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 0 0
- Melbourne
Recruitment postcode(s) [2] 0 0
- Perth
Recruitment outside Australia
Country [1] 0 0
Brazil
State/province [1] 0 0
Rio Grande Do Sul

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
AVROBIO
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a multinational, long-term follow-up study to assess the long-term safety and durability of AVR-RD-01 treatment in participants who received a single dose administration of lentiviral gene therapy in Study AVRO-RD-01-201 (treatment study). No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from AVR-RD-01 gene therapy infusion.
Trial website
https://clinicaltrials.gov/study/NCT04999059
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Inderpal Panesar, MRPharmS
Address 0 0
AVROBIO, Inc
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04999059