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Gene Therapy
Gene Therapy for Fabry Disease
Phase 1 & 2
Waitlist Available
Research Sponsored by Sangamo Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Additional Inclusion Criteria for Cardiac Cohort: Left ventricular hypertrophy (LVH) in 2D echocardiography or CMR, OR presentation with cardiac changes indicative of disease progression
≥ 18 years of age
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 12 months after the st-920 infusion
Awards & highlights
Study Summary
This trial is testing a new treatment for Fabry disease using a virus to deliver a working copy of the gene for alpha-Gal A. The goal is to see if it's safe and tolerable in humans, and if it can provide long-term production of alpha-Gal A.
Who is the study for?
Adults with Fabry disease can join this trial. For the cardiac group, they need heart changes like left ventricular hypertrophy or other signs of worsening disease. The renal group requires specific kidney function levels and a history of declining function. All participants must be COVID-19 vaccinated at least one month before treatment.Check my eligibility
What is being tested?
The trial is testing ST-920, a gene therapy designed to produce the enzyme α-Gal A in people with Fabry disease. It's given as a single intravenous dose, aiming to reduce or clear harmful substances in the body caused by the disease over a 52-week period.See study design
What are the potential side effects?
Potential side effects are not detailed but may include reactions related to immune response against the viral vector (AAV6) used for gene delivery or components of ST-920 formulation.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My heart shows signs of thickening or disease progression.
Select...
I am 18 years old or older.
Select...
I have symptoms like vision changes, pain in hands/feet, less sweating, or skin spots.
Select...
I have been diagnosed with Fabry disease.
Select...
My kidney function is moderately reduced and getting worse each year.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 12 months after the st-920 infusion
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 12 months after the st-920 infusion
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence of treatment-emergent adverse events (TEAEs)
Trial Design
2Treatment groups
Experimental Treatment
Group I: Sequential dose escalationExperimental Treatment1 Intervention
ST-920 is administered as a single infusion:
Cohort 1: 0.5e13 vg/kg
Cohort 2: 1.0e13 vg/kg
Cohort 3: 3.0e13 vg/kg
Cohort 4: 5.0e13 vg/kg
Group II: Expansion CohortsExperimental Treatment1 Intervention
Anti Alpha-Gal A Antibody Positive Cohort
Anti Alpha-Gal A Antibody Negative Cohort
Female Cohort
Renal Cohort
Cardiac Cohort
Find a Location
Who is running the clinical trial?
Sangamo TherapeuticsLead Sponsor
28 Previous Clinical Trials
915 Total Patients Enrolled
1 Trials studying Fabry Disease
48 Patients Enrolled for Fabry Disease
Medical MonitorStudy DirectorSangamo Therapeutics, Inc.
1,649 Previous Clinical Trials
979,691 Total Patients Enrolled
13 Trials studying Fabry Disease
808 Patients Enrolled for Fabry Disease
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Eligibility Criteria:
This trial includes the following eligibility criteria:- You have had problems with alcohol or drugs in the recent past.My heart shows signs of thickening or disease progression.You had a bad reaction to ERT in the last 6 months.My heart condition severely limits my daily activities.I have previously received gene therapy.I am 18 years old or older.I have not had liver disease in the last 6 months, except for Gilbert's syndrome.You have had kidney dialysis or transplantation, recent kidney problems, or have started or changed certain medications for kidney issues. Your urine test shows high protein levels and you are not taking specific medications for it.You have significant heart scarring shown on a special heart imaging test, or you cannot have that test based on the hospital's rules. You also started or changed certain heart medications in the last 4 weeks, or you are in a severe stage of heart failure.I have had cancer before, but only skin cancer (not melanoma) or prostate cancer that was treated to cure it.I have symptoms like vision changes, pain in hands/feet, less sweating, or skin spots.Your blood has higher levels of AFP than normal.You are allergic to any of the ingredients in the ST-920 medication.You have antibodies that can stop the AAV6 treatment from working.I have been diagnosed with Fabry disease.My kidney function is reduced with an eGFR below 40.My kidney function is moderately reduced and getting worse each year.I cannot take corticosteroids due to health reasons.I do not have an active infection with hepatitis A, B, C, HIV, or TB.I haven't used immunomodulators, biologics, or steroids in the last 6 months.
Research Study Groups:
This trial has the following groups:- Group 1: Sequential dose escalation
- Group 2: Expansion Cohorts
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How many people will be taking part in this clinical trial at most?
"That is accurate. As of now, this study is looking for 48 individuals who meet the requirements and are willing to participate at one of the 12 locations across clinicaltrials.gov."
Answered by AI
How many clinical sites are conducting this trial?
"There are many enrolling sites for this clinical trial, but they include Vanderbilt University Medical Center in Nashville, Tennessee, M.A.G.I.C. Clinic Ltd. in Calgary, Alberta, and Cincinnati Children's Hospital Medical Center in Cincinnati, Ohio among 12 others."
Answered by AI
Are new participants being sought for this experiment?
"Yes, according to the information available on clinicaltrials.gov, this trial is still looking for participants. The trial was first posted on July 23rd, 2019 and was most recently updated on November 2nd, 2020. There are 12 locations where the study is being conducted and they are looking for 48 participants in total."
Answered by AI
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