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

NCT04836377 | Terminated FDA Drug

• 1 other identifier: AVRO-RD-02-LTF01
• Study Type: observational
• Target: 4
• Locations: 2 countries
• Sites: 3

Brief 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).

Conditions

Type 1 Gaucher Disease

Keywords

Gaucher; Guard1

Outcome Measures

Primary Outcomes (10)

• Change from baseline over time in spleen volume as assessed by abdominal Magnetic Resonance Imaging (MRI)

  Baseline to Year 15 post gene therapy infusion

• Baseline over time in liver volume as assessed by abdominal MRI

  Baseline to Year 15 post gene therapy infusion

• Baseline over time in hemoglobin

  Baseline to Year 15 post gene therapy infusion

• Change from Baseline over time in platelet count

  Baseline to Year 15 post gene therapy infusion

• 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

• Incidence of newly-diagnosed malignancy, hematologic disorder, and/or immune related events/immunogenicity

  Baseline to Year 15 post gene therapy infusion

• Number of participants with clinically relevant abnormalities as assessed by vital signs

  Baseline to Year 15 post gene therapy infusion

• Number of participants with clinically relevant abnormalities, as assessed by physical examinations findings

  Baseline to Year 15 post gene therapy infusion

• Number of participants with clinically relevant abnormalities, as assessed by clinical laboratory tests

  Baseline to Year 15 post gene therapy infusion

• Number of participants with clinically relevant abnormalities, as assessed by electrocardiograms (ECGs)

  Baseline to Year 15 post gene therapy infusion

Secondary Outcomes (9)

• Change from baseline over time in glucocerebrosidase (GCase) GCase enzyme activity level in

  Baseline to Year 15 post gene therapy infusion

• Change from baseline over time in Enzyme Replacement Therapy (ERT) frequency and dosing

  Baseline to Year 15 post gene therapy infusion

• Changes in Gaucher biomarker indices of Gaucher disease in chitotriosidase enzyme activity levels in plasma

  Baseline to Year 15 post gene therapy infusion

• 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

• 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

Study Arms (1)

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.

Other: Safety and efficacy assessments

Interventions

Safety and efficacy assessments OTHER

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

Also known as: Gene Therapy Intervention

Subjects with Gaucher 1 Disease

Eligibility Criteria

• Age: 18 Years - 99 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

To permit longitudinal assessment of safety, efficacy and durability of effect of AVR-RD-02 gene therapy infusion in the AVRO-RD-02-LTF01 study, subjects must have received AVR-RD-02 in a preceding treatment study and agree to comply with the study visit schedule and procedures for AVRO-RD-02-LTF01 for inclusion in the Long-Term Follow-Up study.

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• AVROBIO: lead

Study Sites (3)

University of Iowa

Iowa City, Iowa, 52242, United States

Location

Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States

Location

M.A.G.I.C. Clinic Ltd

Calgary, Calgary Alberta, T2M 0L6, Canada

Location

Biospecimen

Retention: SAMPLES WITH DNA

* Blood sample collections consisting of serum chemistry (and electrolytes), hematology, and urinalysis * Blood sample collection to assess for the presence of anti-GCase antibodies

MeSH Terms

Conditions

Gaucher Disease

Interventions

Safety

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Intervention Hierarchy (Ancestors)

Accident Prevention; Accidents; Public Health; Environment and Public Health

Study Officials

• Milena Veselinovic, MD

  AVROBIO

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 23, 2021
• First Posted: April 8, 2021
• Study Start: July 6, 2021
• Primary Completion: August 21, 2023
• Study Completion: August 21, 2023
• Last Updated: August 29, 2023

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will share

Locations

US (2); CA (1)