Hemophilia B, Mucopolysaccharidosis I, Mucopolysaccharidosis II
Conditions
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
Brief 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.
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.
Interventions
BIOLOGICALSB-318
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.
BIOLOGICALSB-913
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.
BIOLOGICALSB-FIX
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.
Sponsors
Sangamo Therapeutics
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. 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 2. Subjects who have provided consent to participate in the LTFU study.
Exclusion criteria
1. Unable to comply with study visit schedule or study visit procedures. 2. Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for participation in the study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Long-term safety | 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. |
Countries
United States
Contacts
STUDY_DIRECTORMedical Monitor, MD
Sangamo Therapeutics
Outcome results
None listed