Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

Completed · Phase 1/Phase 2

Conditions studied: Fabry Disease

In brief

This is the first in human treatment with ST-920, an adeno-associated virus (AAV2/6) vector encoding the complementary deoxyribonucleic acid (cDNA) for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single dose of ST-920 and followed for a period of 52 weeks.

Key facts

Study ID: NCT04046224
Run by: Sangamo Therapeutics
People needed: 36
Starts: 2019-07-23
Expected to finish: 2025-04-10
Last updated by the study team: 2026-04-14

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

≥ 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

You may not qualify if…

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

Where it is running

University of California, Irvine — Irvine, California, United States
University of South Florida — Tampa, Florida, United States
Emory University School of Medicine — Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
University of Iowa Hospital and Clinics — Iowa City, Iowa, United States
University of Minnesota Medical Center — Minneapolis, Minnesota, United States
Mt. Sinai School of Medicine — New York, New York, United States
Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
Lysosomal and Rare Disorders Research and Treatment Center (LDRTC) — Fairfax, Virginia, United States
The Royal Melbourne Hospital — Parkville, Victoria, Australia
M.A.G.I.C. Clinic Ltd. — Calgary, Alberta, Canada
University Medical Center Hamburg-Eppendorf — Hamburg, Germany
University Hospital of Würzburg — Würzburg, Germany
Azienda Ospedaliero-Universitaria Careggi — Florence, Tuscany, Italy
National Taiwan University Hospital — Taipei, Taiwan
Queen Elizabeth Hospital — Birmingham, United Kingdom
Addenbrooke's Hospital — Cambridge, United Kingdom
Royal Free Hospital — London, United Kingdom

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.