Hemophilia BLTFUNCT04628871
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
An observational study of Hemophilia B and Mucopolysaccharidosis I, run by Sangamo Therapeutics at 5 sites in United States.
Trial summary
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial 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 10 years following exposure to SB-318, SB-913, or SB-FIX. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
- What is being tested?
- SB-318, SB-913 and SB-FIX
- How many people
- 13 participants
- Where
- 5 sitesUnited States
- Study dates
- 3 Nov 2020to 17 Jun 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
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 SB-318 in Study Protocol SB-318-1502, SB-913 in Study Protocol SB-913-1602 or SB-FIX in Study Protocol SB-FIX-1501 and who have consented to participate in this Long Term Follow-up study.
Who may be able to join
- Subjects who received SB-318 under Study Protocol SB-318-1502, SB-913 under Study Protocol SB-913-1602, or SB-FIX under Study Protocol SB-FIX-1501
- Subjects who have provided consent to participate in the LTFU study.
Who cannot join
- Unable to comply with study visit schedule or study visit procedures.
- Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for participation in the study.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
University of North Carolina
Chapel Hill, North Carolina, 27514
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
New York University Grossman School of Medicine
New York, New York, 10016
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
About this study
Official title: Long-Term Follow-up of Subjects Who Were Treated With SB-318, SB-913, or SB-FIX, for Targeted Genome Editing Into the Albumin Gene in the Liver
Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial 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 10 years following exposure to SB-318, SB-913, or SB-FIX.
Read the detailed description
Non-interventional, multi-center, long-term follow-up (LTFU) study of subjects dosed with SB-318 in the clinical study SB-318-1502, SB-913 in the clinical study SB-913-1602, and SB-FIX in clinical study SB-FIX-1501. All subjects dosed in the studies and 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 10 years following exposure to the respective investigational products.
- Being tested
- SB-318, SB-913, SB-FIX
- Keywords
- Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Mucopolysaccharidosis II, Hunter Syndrome, Gene Editing, Gene Therapy, Zinc Finger, SB-318, SB-913, SB-FIX, Rare, Genetic, DNA, Sangamo, ZFN, Long Term, Hemophilia B, MPS
- Medical subject headings
- Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SB-318Biological
No study drug is administered in this study. Subject who received SB-318 in a previous trial will be evaluated in this trial for long-term safety.
SB-913Biological
No study drug is administered in this study. Subject who received SB-913 in a previous trial will be evaluated in this trial for long-term safety.
SB-FIXBiological
No study drug is administered in this study. Subject who received SB-FIX in a previous trial will be evaluated in this trial for long-term safety.
| Study group | Role | What they receive |
|---|---|---|
| Subjects who received SB-318 | Subjects who received SB-318 in clinical study SB-318-1502 Receives: Biological: SB-318 | |
| Subjects who received SB-913 | Subjects who received SB-913 in clinical study SB-913-1602. Receives: Biological: SB-913 | |
| Subjects who received SB-FIX | Subjects who received SB-FIX in clinical study SB-FIX Receives: Biological: SB-FIX |
What is being measured
The primary outcome is the main question the study is designed to answer.
Long-term safety
Long-term safety of SB-318, SB-913, and SB-FIX by assessment of any newly-diagnosed or worsening of existing medical conditions.
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Nov 2020
- Study start3 Nov 2020
- Primary completion17 Jun 2026
- Study completion17 Jun 2026
Why it stopped: Sangamo declared Chapter 11 Bankruptcy on 23Jun2026 and terminated the study on 21Jul2026.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies 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
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sangamo Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, MD, Study director, Sangamo Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04628871
- Sponsor's protocol ID
- ST-IVPRP-LT01
- Acronym
- LTFU
- Status last verified
- Aug 2026
- First submitted
- 9 Nov 2020
- Last updated
- 13 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 13 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-Term Follow-up of Subjects Who Were Treated With SB-318, SB-913, or SB-FIX, for Targeted Genome Editing Into the Albumin Gene in the Liver
- ClinicalTrials.gov ID
- NCT04628871
- Acronym / sponsor ID
- LTFU / ST-IVPRP-LT01
- Keywords
- Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Mucopolysaccharidosis II, Hunter Syndrome, Gene Editing, Gene Therapy, Zinc Finger, SB-318
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Sangamo declared Chapter 11 Bankruptcy on 23Jun2026 and terminated the study on 21Jul2026.
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Sangamo TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 3 Nov 2020 (actual)
- Primary completion
- 17 Jun 2026 (actual)
- Study completion
- 17 Jun 2026 (actual)
- First posted
- 16 Nov 2020 (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 SB-318, SB-913, or SB-FIX in a previous trial 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 10 years following exposure to SB-318, SB-913, or SB-FIX.
Detailed description
Non-interventional, multi-center, long-term follow-up (LTFU) study of subjects dosed with SB-318 in the clinical study SB-318-1502, SB-913 in the clinical study SB-913-1602, and SB-FIX in clinical study SB-FIX-1501. All subjects dosed in the studies and 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 10 years following exposure to the respective investigational products.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Subjects who received SB-318 | – | Biological: SB-318Subjects who received SB-318 in clinical study SB-318-1502 |
| Subjects who received SB-913 | – | Biological: SB-913Subjects who received SB-913 in clinical study SB-913-1602. |
| Subjects who received SB-FIX | – | Biological: SB-FIXSubjects who received SB-FIX in clinical study SB-FIX |
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 SB-318 in Study Protocol SB-318-1502, SB-913 in Study Protocol SB-913-1602 or SB-FIX in Study Protocol SB-FIX-1501 and who have consented to participate in this Long Term Follow-up study.
Key inclusion criteria (2 listed)
- Subjects who received SB-318 under Study Protocol SB-318-1502, SB-913 under Study Protocol SB-913-1602, or SB-FIX under Study Protocol SB-FIX-1501
- Subjects who have provided consent to participate in the LTFU study.
Key exclusion criteria (2 listed)
- Unable to comply with study visit schedule or study visit procedures.
- Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for participation in the study.
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 |
|---|---|---|
| SB-318No study drug is administered in this study. Subject who received SB-318 in a previous trial will be evaluated in this trial for long-term safety. | Biological | Subjects who received SB-318 |
| SB-913No study drug is administered in this study. Subject who received SB-913 in a previous trial will be evaluated in this trial for long-term safety. | Biological | Subjects who received SB-913 |
| SB-FIXNo study drug is administered in this study. Subject who received SB-FIX in a previous trial will be evaluated in this trial for long-term safety. | Biological | Subjects who received SB-FIX |
Primary outcome
Long-term safety
Time frame: 10 years
Long-term safety of SB-318, SB-913, and SB-FIX by assessment of any newly-diagnosed or worsening of existing medical conditions.
Results and safety
The study was stopped early (reason given: Sangamo declared Chapter 11 Bankruptcy on 23Jun2026 and terminated the study on 21Jul2026.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2026.
Related trials for Hemophilia B
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia
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, MD study director
Sangamo Therapeutics
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia B and Mucopolysaccharidosis I, run by Sangamo Therapeutics at 5 sites in United States.
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 2 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: stopped early, as of the sponsor's last update on 13 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 5 sites in United States. 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?
The study was stopped early (reason given: Sangamo declared Chapter 11 Bankruptcy on 23Jun2026 and terminated the study on 21Jul2026.). Results are not available in the source record.
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?
Long-term safety (time frame: 10 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 13 Aug 2026.