AVR-RD-02 for Gaucher Disease

Phase 1 & 2

Waitlist Available

Research Sponsored by AvroBio

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up at weeks 5, 13, 26, 39, and 52

Awards & highlights

No Placebo-Only Group

Summary

This trial tested AVR-RD-02, a gene therapy for adults with Type 1 Gaucher disease. It aims to help patients produce a missing enzyme by correcting their genetic defect. The process involves collecting and modifying the patient's cells and then infusing them back into the patient. Gaucher disease is a lysosomal storage disorder caused by a deficiency of the enzyme glucocerebrosidase (GC), making it an excellent candidate for gene therapy.

Eligible Conditions

Gaucher Disease

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ at weeks 5, 13, 26, 39, and 52

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~at weeks 5, 13, 26, 39, and 52

This trial's timeline: 3 weeks for screening, Varies for treatment, and at weeks 5, 13, 26, 39, and 52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change From Baseline in Hemoglobin Concentration

Change From Baseline in Liver Volume Assessed by Abdominal MRI

Change From Baseline in Plasma Lyso-Gb1 Levels by Liquid Chromatography Tandem Mass Spectrometry (LC/MS/MS)

+5 more

Secondary study objectives

Change From Baseline in Bone Marrow Burden (BMB) Score as Assessed by Bone Magnetic Resonance Imaging (MRI)

Change From Baseline in Bone Mineral Density (BMD) Assessed by Bone Density Scan (DXA)

Change From Baseline in GCase Enzyme Activity Level in Peripheral Blood Leukocytes

+4 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Treatment-naïveExperimental Treatment1 Intervention

Treatment-naïve arm: Subjects with Type 1 Gaucher disease who had never received either ERT or SRT for Gaucher disease or had not received either ERT or SRT for Gaucher disease within 12 months of Screening (i.e., treatment-naïve subjects). Enrollment followed a similar scheme as for the switch-stable subjects. Note: No subjects enrolled in this arm.

Group II: Switch StableExperimental Treatment1 Intervention

Switch-stable arm: Subjects who had undergone ERT ≥15 U/kg and ≤60 U/kg every other week (or equivalent; i.e., any combination of infusions resulting in a total monthly ERT dose of \>30 U/kg and \<120 U/kg) for ≥24 consecutive months for Type 1 Gaucher disease at the time of Screening. Switch-stable subjects must have discontinued ERT at least 2 weeks before the scheduled AVR-RD-02 infusion. Switch-stable subjects who had been and substrate reduction therapy (SRT) must not have received SRT within 12 months of Screening.

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