One-Time gene therapy could change fabry disease treatment
NCT ID NCT04046224
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This trial tested a new gene therapy called ST-920 for Fabry disease, a rare genetic condition. The therapy uses a harmless virus to deliver a working gene that helps the body produce an enzyme it's missing. 36 adults with Fabry disease received a single intravenous dose and were monitored for a year to check safety and tolerability. The goal is to see if this one-time treatment can provide long-term benefits.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ST-920 (a gene therapy using a modified virus to deliver a working copy of the alpha-galactosidase A gene)
- What this could lead to
- If successful, this could provide a long-term treatment option for Fabry disease, potentially reducing or eliminating the need for regular enzyme replacement therapy.
- What could go wrong
- This is an early-phase trial (Phase 1/2) with only 36 participants, so safety and effectiveness are not yet proven. Gene therapies can have unexpected side effects, and long-term benefits are uncertain.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1/2
Runs two stages together: safety and dose first, then whether the treatment works.
- Participants
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36 people
The number who actually took part.
- Started
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Jul 2019
- Finished
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Apr 2025
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * ≥ 18 years of age * Documented diagnosis of Fabry disease * One or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma * Subject must be fully vaccinated (as per the Centers for Disease Control and Prevention (CDC) definition in the US and as per local guidelines in other countries) for Coronavirus Disease (COVID-19) at least one month prior to dosing Additional Inclusion Criteria: Renal Cohort: * Screening estimated glomerular filtration rate (eGFR) value between 40-90 mL/min/1.73 m² * Linear negative eGFR slope (estimated from at least 3 serum creatinine values within 18 months, including the value obtained during screening visit) of ≥ 2 mL/min/1.73m²/year Cardiac Cohort: • Left ventricular hypertrophy (LVH) in 2D echocardiography or cardiac magnetic resonance imaging (CMR) defined as an end diastolic septum and posterior wall thickness ≥12 mm with no other explanation for LVH, OR presentation with cardiac changes indicative of disease progression such as decreased global longitudinal strain on 2D strain echocardiography or low native T1 mapping on CMR Exclusion Criteria: * Neutralizing antibodies to AAV6 * eGFR \< 40 ml/min/1.73m2 * New York Heart Association Class III or higher * Active infection with hepatitis A, B or C, human immunodeficiency virus (HIV) or tuberculosis (TB) * History of liver disease such as clinically significant steatosis, fibrosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, biliary disease within 6 months of informed consent; except for Gilbert's syndrome * Elevated circulating serum alpha fetoprotein (AFP) * Recent or recurrent hypersensitivity response to enzyme replacement therapy (ERT) within within 6 months prior to consent * Current or history of systemic (IV or oral) immunomodulatory agents, or biologics or steroid use in the past 6 months prior to consent (topical treatment and inhaled allowed). * Contraindication to use of corticosteroids * History of malignancy except for non-melanoma skin cancer and localized prostate cancer treated with curative intent * Recent history of alcohol or substance abuse * Participation in investigational interventional drug or medical device study throughout the duration of this study and within previous 3 months prior to consent * Prior treatment with a gene therapy product * Known hypersensitivity to components of ST-920 formulation * Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study including but not limited to risk of COVID-19 infection Additional exclusion criteria for: Renal cohort: * History of renal dialysis or transplantation * History of acute kidney insufficiency in the 6 months prior to screening * Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated within 4 weeks prior to screening or changed ACE inhibitor or ARB dose in the 4 weeks prior to screening * Urine protein to creatinine ratio (UPCR) \> 0.5 g/g who are not being treated with an ACE inhibitor or ARB Cardiac cohort: * Significant cardiac fibrosis defined by late gadolinium enhancement on CMR * Any contraindications to CMR as per local hospital/institution guidelines * Angiotensin converting enzyme (ACE) inhibitor or angiotensin receptor blocker (ARB) therapy initiated within 4 weeks prior to screening or changed ACE inhibitor or ARB dose in the 4 weeks prior to screening * New York heart association (NYHA) Class IV
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Addenbrooke's Hospital
Cambridge, CB2 0QQ, United Kingdom
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Azienda Ospedaliero-Universitaria Careggi
Florence, Tuscany, 50134, Italy
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)
Fairfax, Virginia, 22030, United States
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M.A.G.I.C. Clinic Ltd.
Calgary, Alberta, T2E 7Z4, Canada
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Mt. Sinai School of Medicine
New York, New York, 10029, United States
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National Taiwan University Hospital
Taipei, Taiwan
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Queen Elizabeth Hospital
Birmingham, B15 2TH, United Kingdom
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Royal Free Hospital
London, United Kingdom
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The Royal Melbourne Hospital
Parkville, Victoria, 3050, Australia
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University Hospital of Würzburg
Würzburg, Germany
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University Medical Center Hamburg-Eppendorf
Hamburg, Germany
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University of California, Irvine
Irvine, California, 92697, United States
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University of Iowa Hospital and Clinics
Iowa City, Iowa, 52242, United States
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University of Minnesota Medical Center
Minneapolis, Minnesota, 55455, United States
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University of South Florida
Tampa, Florida, 33620, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a One-Time gene therapy fix fabry disease for years?
- Can a new pill stop fatty buildup in fabry disease?
- Gene Therapy's lasting promise: can one infusion safely control fabry disease for years?
- Can early enzyme therapy save kidneys in fabry disease?
- Can continued lucerastat access help fabry patients?
- Can a single gene infusion rewrite the story of fabry disease?