Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

Study Overview
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Brief Summary
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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
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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.

Official Title
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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

Conditions
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Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II

Intervention / Treatment
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* Biological: SB-318
* Biological: SB-913
* Biological: SB-FIX


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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. Exclusion Criteria: 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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Subjects who received SB-318<br>Subjects who received SB-318 in clinical study SB-318-1502 | Biological: SB-318<br>* 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.<br>|
| Subjects who received SB-913<br>Subjects who received SB-913 in clinical study SB-913-1602. | Biological: SB-913<br>* 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.<br>|
| Subjects who received SB-FIX<br>Subjects who received SB-FIX in clinical study SB-FIX | Biological: SB-FIX<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 10 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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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