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An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3

NCT05815004 · Status: WITHDRAWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL

Last updated 2023-08-09

No results posted yet for this study

Summary

The purpose of this study is to evaluate the efficacy and safety of autologous hemotopoietic stem cell (HSC) gene therapy, AVR-RD-02, compared to enzyme replacement therapy, for the treatment of Gaucher disease Type 3 in male and female participants aged 2 to 25 years.

The study will consist of 2 parts - Core (Part 1) followed by the ERT-crossover (Part 2)

Conditions

  • Gaucher Disease, Type 3

Interventions

DRUG

Gene therapy

AVR-RD-02 Drug product: active substance is autologous CD34+ enriched hematopoietic stem cells (HSCs) that have been genetically modified ex vivo with a lentiviral vector (LV) to contain a ribonucleic acid (RNA) transcript that, after reverse transcription, results in codon-optimized, complementary deoxyribonucleic acid (cDNA) that, upon its integration into human genome, encodes for functional human glucocerebrosidase (GCase)

DRUG

Enzyme Replacement Agent

A hydrolytic lysosomal glucocerebrosidase-specific enzyme

Sponsors & Collaborators

  • AVROBIO

    lead INDUSTRY

Principal Investigators

  • Milena Veselinovic, MD, PhD · AVROBIO, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-10-31
Primary Completion
2027-12-31
Completion
2027-12-31
FDA Drug
Yes

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05815004 on ClinicalTrials.gov