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.
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:
Show all 10Show fewer
- 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)
Show all 26Show fewer
- 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
United Kingdom3 sites
Queen Elizabeth Hospital
Birmingham, B15 2TH
Addenbrooke's Hospital
Cambridge, CB2 0QQ
Royal Free Hospital
London
Germany2 sites
University Medical Center Hamburg-Eppendorf
Hamburg
University Hospital of Würzburg
Würzburg
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 group | Role | What they receive |
|---|---|---|
| Sequential dose escalation | Experimental | ST-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 Cohorts | Experimental | 1. 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.
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
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
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
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)
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.
- First posted6 Aug 2019
- Study start23 Jul 2019
- Primary completion10 Apr 2025
- Study completion10 Apr 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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)
Sponsor and investigators
- Lead sponsor
- Sangamo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Sangamo Therapeutics, Inc.
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
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT04046224
- Acronym / sponsor ID
- ST-920-201
- Conditions
- Fabry Disease
- Keywords
- Sangamo, Rare, Lysosomal Storage Disease, Gene Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 23 Jul 2019 (actual)
- Primary completion
- 10 Apr 2025 (actual)
- Study completion
- 10 Apr 2025 (actual)
- First posted
- 6 Aug 2019 (actual)
- Results
- Posted 14 Apr 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sequential dose escalation | Experimental | Biological: ST-920ST-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 |
| Expansion Cohorts | Experimental | Biological: ST-9201. Anti Alpha-Gal A Antibody Positive Cohort 2. Anti Alpha-Gal A Antibody Negative Cohort 3. Female Cohort 4. Renal Cohort 5. Cardiac Cohort |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- ≥ 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
- Screening estimated glomerular filtration rate (eGFR) value between 40-90 mL/min/1.73 m²
Key exclusion criteria (26 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ST-920Single dose of investigational product ST-920 | Biological | Expansion Cohorts, Sequential dose escalation |
Primary outcome
Incidence of Treatment-emergent Adverse Events (TEAEs) - All
Time frame: Up to 12 months after the ST-920 infusion
All incidences of Treatment-Emergent Adverse Events (TEAEs) in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)
Incidence of Treatment-emergent Adverse Events (TEAEs) - Related to ST-920
Time frame: Up to 12 months post ST-920 infusion
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)
Incidence of Treatment-emergent Adverse Events (TEAEs) - Serious
Time frame: Up to 12 month post ST-920 infusion
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)
Incidence of Treatment-emergent Adverse Events (TEAEs) - Any TEAEs Leading to Study Discontinuation or Withdrawal
Time frame: Up to 12 month post ST-920 infusion
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)
1 secondary outcome
To Assess Alpha Gal-A Activity in Plasma Over Time
Time frame: up to 12 months post ST-920 infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Fabry Disease
RecruitingPhase 3
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Fabry Disease
RecruitingObservational study
A Study of Patients With Fabry Disease (US Specific)
Fabry Disease
RecruitingObservational study
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Fabry Disease
Active, not recruiting
Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
Fabry Disease
RecruitingPhase 3
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Fabry Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Sangamo Therapeutics, Inc.
18 sites in 7 countries: see every site and its status.
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.