Fabry DiseaseNCT05039866
Long-Term Follow-up of Subjects Who Were Treated With ST-920
An observational study of Fabry Disease and Fabry Disease, Cardiac Variant, run by Sangamo Therapeutics at 13 sites in 5 countries.
Trial summary
Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 5 years following ST-920 infusion. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease, Fabry Disease, Cardiac Variant
- What is being tested?
- ST-920
- How many people
- 32 participants
- Where
- 13 sitesin 5 countries
- Study dates
- 16 Aug 2021to Mar 2039
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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
Study population: All subjects who have received ST-920 in separate parent trial and who have consented to participate in this Long Term Follow-up study.
Who may be able to join
- Subjects who received ST-920 therapy in a separate parent trial
- Subjects who have consented to participate in this LTFU study.
Who cannot join
- -This study has no exclusion criteria
Trial locations and contacts
13 sites in 5 countries.
13 sites in 5 countries
United States8 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Lysosomal & Rare Disorders Research & 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 Hospital
New York, New York, 10029
University of South Florida
Tampa, Florida, 33620
United Kingdom2 sites
Addenbrooke's Hospital
Cambridge, CB2 0QQ
Royal Free Hospital
London
About this study
Official title: Long-Term Follow-up of Fabry Disease Subjects Who Were Treated With ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy
Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 5 years following ST-920 infusion.
Read the detailed description
Non-interventional, multi-center, long-term follow-up (LTFU) study of subjects dosed with ST-920 in the clinical study ST-920-201. All subjects dosed in the study who completed at least 52 weeks post-infusion follow-up in their primary protocol will be offered to participate. Subjects who enroll will be monitored for a total of up to 5 years following ST-920 infusion.
- Conditions
- Fabry Disease, Fabry Disease, Cardiac Variant
- Being tested
- ST-920
- Keywords
- Fabry Disease, Gene Therapy, ST-920, Rare, Genetic, DNA, Sangamo, Long Term
- Medical subject headings
- Fabry Disease, Fabry Disease, Cardiac Variant
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ST-920Biological
No study drug is administered in this study. Subjects who received ST-920 in a separate parent trial will be evaluated in this trial for long-term safety.
| Study group | Role | What they receive |
|---|---|---|
| Subjects who received ST-920 | Subjects who received ST-920 in a separate parent trial Receives: Biological: ST-920 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate long-term safety of ST-920
To evaluate long-term safety of ST-920 by assessment of incidence and severity of adverse events (AEs)
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Sep 2021
- Study start16 Aug 2021
- Primary completionMar 2029 (estimated)
- Study completionMar 2039 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 32 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
- NCT05039866
- Sponsor's protocol ID
- ST-920-LT01
- Status last verified
- Aug 2026
- First submitted
- 20 Aug 2021
- Last updated
- 20 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-Term Follow-up of Fabry Disease Subjects Who Were Treated With ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy
- ClinicalTrials.gov ID
- NCT05039866
- Acronym / sponsor ID
- ST-920-LT01
- Conditions
- Fabry Disease, Fabry Disease, Cardiac Variant
- Keywords
- Fabry Disease, Gene Therapy, ST-920, Rare, Genetic, DNA, Sangamo, Long Term
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 16 Aug 2021 (actual)
- Primary completion
- Mar 2029 (estimated)
- Study completion
- Mar 2039 (estimated)
- First posted
- 10 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 5 years following ST-920 infusion.
Detailed description
Non-interventional, multi-center, long-term follow-up (LTFU) study of subjects dosed with ST-920 in the clinical study ST-920-201. All subjects dosed in the study who completed at least 52 weeks post-infusion follow-up in their primary protocol will be offered to participate. Subjects who enroll will be monitored for a total of up to 5 years following ST-920 infusion.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Subjects who received ST-920 | – | Biological: ST-920Subjects who received ST-920 in a separate parent trial |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- All subjects who have received ST-920 in separate parent trial and who have consented to participate in this Long Term Follow-up study.
Key inclusion criteria (2 listed)
- Subjects who received ST-920 therapy in a separate parent trial
- Subjects who have consented to participate in this LTFU study.
Key exclusion criteria (1 listed)
- -This study has no exclusion criteria
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-920No study drug is administered in this study. Subjects who received ST-920 in a separate parent trial will be evaluated in this trial for long-term safety. | Biological | Subjects who received ST-920 |
Primary outcome
To evaluate long-term safety of ST-920
Time frame: 4 years
To evaluate long-term safety of ST-920 by assessment of incidence and severity of adverse events (AEs)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Aug 2026.
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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.
13 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Fabry Disease and Fabry Disease, Cardiac Variant, run by Sangamo Therapeutics at 13 sites in 5 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 2 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 20 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in United States, United Kingdom, Australia, Canada, Germany. 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
To evaluate long-term safety of ST-920 (time frame: 4 years). 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 20 Aug 2026.