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Gene Therapy

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

Study Summary

This trial is testing a new treatment for Gaucher disease, a disorder where people don't have enough of an enzyme that breaks down fats. The trial will test the safety and effectiveness of the new treatment in 8 to 16 people with Gaucher disease.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
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 outcome measures
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

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.

Find a Location

Who is running the clinical trial?

AvroBioLead Sponsor
3 Previous Clinical Trials
24 Total Patients Enrolled
1 Trials studying Gaucher Disease
4 Patients Enrolled for Gaucher Disease
AVROBIOLead Sponsor
6 Previous Clinical Trials
79 Total Patients Enrolled
2 Trials studying Gaucher Disease
4 Patients Enrolled for Gaucher Disease
Medical DirectorStudy DirectorAvroBio
2,777 Previous Clinical Trials
8,063,472 Total Patients Enrolled
2 Trials studying Gaucher Disease
12 Patients Enrolled for Gaucher Disease

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the current enrollment limit for this research project?

"This study requires 16 suitable patients to partake in the experiment, and two choice clinical trial sites are Hackensack University Medical Center (NJ) and University of Calgary (Alberta)."

Answered by AI

How many establishments are offering the clinical trial?

"Currently, 4 different trial sites are enrolling patients. These include Hackensack, Calgary and Pittsburgh as well as another four locations. To reduce travel needs, it's recommended to select the nearest site when registering for this clinical trial."

Answered by AI

Is this experiment currently welcoming participants?

"According to the information sourced from clinicaltrials.gov, this investigation is still enrolling volunteers with a start date of May 30th 2019 and most recent update on October 20th 2022."

Answered by AI

Are individuals aged 18 and over eligible for participation in this experimentation?

"This medical trial has stipulated that participants must be aged 18 to 50. For those under the age of 18, there are 7 alternative studies; conversely, 10 trials are available for people over 65 years old."

Answered by AI

Is there any possibility I could be a participant in the current medical experiment?

"Individuals aged 18 to 50 with a diagnosis of Gaucher disease are eligible for this study, which requires 16 participants in total."

Answered by AI

Who else is applying?

How old are they?
18 - 65
What site did they apply to?
Hackensack University Medical Center
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
2019. "Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04145037.
~1 spots leftby Apr 2025
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