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Fabry DiseasePhase 1/2NCT04046224

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

A Phase 1/2 study testing ST-920 in people with Fabry Disease, run by Sangamo Therapeutics at 18 sites in 7 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Fabry Disease
What is being tested?
ST-920
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
36 participants
Where
18 sitesin 7 countries
Study dates
23 Jul 2019to 10 Apr 2025
Phase
Phase 1/2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • ≥ 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:
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  • 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

Who cannot join

  • 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)
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  • 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

Trial locations and contacts

18 sites in 7 countries.

18 sites in 7 countries

United States9 sites
  • Emory University School of Medicine

    Atlanta, Georgia, 30322

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • Lysosomal and Rare Disorders Research and Treatment Center (LDRTC)

    Fairfax, Virginia, 22030

  • University of Iowa Hospital and Clinics

    Iowa City, Iowa, 52242

  • University of California, Irvine

    Irvine, California, 92697

  • University of Minnesota Medical Center

    Minneapolis, Minnesota, 55455

  • Mt. Sinai School of Medicine

    New York, New York, 10029

  • University of South Florida

    Tampa, Florida, 33620

Other trials in United States

United Kingdom3 sites
  • Queen Elizabeth Hospital

    Birmingham, B15 2TH

  • Addenbrooke's Hospital

    Cambridge, CB2 0QQ

  • Royal Free Hospital

    London

Other trials in United Kingdom

Germany2 sites
  • University Medical Center Hamburg-Eppendorf

    Hamburg

  • University Hospital of Würzburg

    Würzburg

Other trials in Germany

Australia1 site
  • The Royal Melbourne Hospital

    Parkville, Victoria, 3050

Other trials in Australia

Canada1 site
  • M.A.G.I.C. Clinic Ltd.

    Calgary, Alberta, T2E 7Z4

Other trials in Canada

Italy1 site
  • Azienda Ospedaliero-Universitaria Careggi

    Florence, Tuscany, 50134

Other trials in Italy

Taiwan1 site
  • National Taiwan University Hospital

    Taipei

Other trials in Taiwan

About this study

Official title: A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease (STAAR)

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.

Conditions
Fabry Disease
Being tested
ST-920
Keywords
Sangamo, Rare, Lysosomal Storage Disease, Gene Therapy
Medical subject headings
Fabry Disease, Lysosomal Storage Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ST-920Biological

    Single dose of investigational product ST-920

Study groups in NCT04046224
Study groupRoleWhat they receive
Sequential dose escalationExperimentalST-920 is administered as a single infusion: 1. Cohort 1: 0.5e13 vg/kg 2. Cohort 2: 1.0e13 vg/kg 3. Cohort 3: 3.0e13 vg/kg 4. Cohort 4: 5.0e13 vg/kg
Receives: Biological: ST-920
Expansion CohortsExperimental1. Anti Alpha-Gal A Antibody Positive Cohort 2. Anti Alpha-Gal A Antibody Negative Cohort 3. Female Cohort 4. Renal Cohort 5. Cardiac Cohort
Receives: Biological: ST-920

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence of Treatment-emergent Adverse Events (TEAEs) - All

    All incidences of Treatment-Emergent Adverse Events (TEAEs) in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)

    Measured: Up to 12 months after the ST-920 infusion

  2. Incidence of Treatment-emergent Adverse Events (TEAEs) - Related to ST-920

    Incidences of Treatment-Emergent Adverse Events (TEAEs) directly related to ST-920 in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)

    Measured: Up to 12 months post ST-920 infusion

  3. Incidence of Treatment-emergent Adverse Events (TEAEs) - Serious

    All incidences of serious Treatment-Emergent Adverse Events (TEAEs) in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)

    Measured: Up to 12 month post ST-920 infusion

  4. Incidence of Treatment-emergent Adverse Events (TEAEs) - Any TEAEs Leading to Study Discontinuation or Withdrawal

    All incidences of Treatment-Emergent Adverse Events (TEAEs) that lead to study discontinuation or withdrawal in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)

    Measured: Up to 12 month post ST-920 infusion

Secondary outcomes (1)
  1. To Assess Alpha Gal-A Activity in Plasma Over Time

    Change in alpha Gal-A activity in plasma from baseline at specific time points over the 1-year study period. Two collections occurred during the baseline period and the latter of the two collections was used for the baseline value. The specific time points are Week 24 and Week 52/End of Study (EOS). Plasma α-Gal A activity was measured using a validated fluorometric enzyme activity assay.

    Measured: up to 12 months post ST-920 infusion

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted6 Aug 2019
  2. Study start23 Jul 2019
  3. Primary completion10 Apr 2025
  4. Study completion10 Apr 2025

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
36 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04046224
Sponsor's protocol ID
ST-920-201
Status last verified
Oct 2026
First submitted
1 Aug 2019
Last updated
27 Oct 2026
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing ST-920 in people with Fabry Disease, run by Sangamo Therapeutics at 18 sites in 7 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 26 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in United States, United Kingdom, Germany, Australia, Canada and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 14 Apr 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence of Treatment-emergent Adverse Events (TEAEs) - All (time frame: Up to 12 months after the ST-920 infusion). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Oct 2026.

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