Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

  • End date
    Dec 11, 2023
  • participants needed
  • sponsor
Updated on 16 February 2022
platelet count
a hemoglobin
type 1 gaucher disease


This is a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are 18 and 45 years of age and postpubertal at Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on clinical phenotype, genotyping, and deficient GCase enzyme activity in whole blood).


Five study periods (Screening, Baseline, Pre-transplant, Transplant, and Post-transplant Follow-up) comprise the study. During the Screening Period (up to 60 days), written informed consent will be obtained and the subject will complete other Screening procedures to confirm study eligibility. Once study eligibility is confirmed, subjects will enter the Baseline Period (up to 3 days) during which time assessments will be performed to establish pre-transplant baseline. Once baseline assessments are complete, the subject will enter the Pre-transplant Period (approximately 6 to 8 weeks) during which time mobilization, apheresis, AVR-RD-02 investigational product preparation and testing for release, conditioning regimen administration, and conditioning washout period will take place. Enzyme replacement therapy must be discontinued at least 2 weeks before the scheduled transplant day. Following completion of the Pre-transplant Period, the subject will enter the Transplant Period (1 day) during which AVR-RD-02 transplant will take place. After AVR-RD-02 transplant, the subject will enter the Post-transplant Follow-up Period (approximately 52 weeks) during which time periodic safety and efficacy assessments will be performed to assess measures of safety, engraftment, and clinical response post-transplant. During the post-transplant period, subjects will not receive ERT unless pre-specified laboratory and clinical criteria, which suggest the need for ERT initiation, are met.

Condition Gaucher Disease
Treatment AVR-RD-02
Clinical Study IdentifierNCT04145037
Last Modified on16 February 2022


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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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