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

Active, not recruiting

• Conditions: Mucopolysaccharidosis I; Hemophilia B; Mucopolysaccharidosis II
• Interventions: Biological: SB-913; Biological: SB-318; Biological: SB-FIX

• Registration Number: NCT04628871
• Lead Sponsor: Sangamo Therapeutics

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.

Study Timeline

• Study Start: 2020-11-03
• Study First Submitted: 2020-11-09
• Study First Submitted QC: 2020-11-09
• Study First Posted: 2020-11-16
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-01
• Last Update Posted: 2024-05-03
• Primary Completion: 2030-01-01
• Study Completion: 2030-01-01

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 13

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Subjects who received SB-913	SB-913	Subjects who received SB-913 in clinical study SB-913-1602.
Subjects who received SB-318	SB-318	Subjects who received SB-318 in clinical study SB-318-1502
Subjects who received SB-FIX	SB-FIX	Subjects who received SB-FIX in clinical study SB-FIX

Primary Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (5)

UCSF Benioff Children's Hospital Oakland; 🇺🇸 Oakland, California, United States

New York University Grossman School of Medicine; 🇺🇸 New York, New York, United States

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

University of North Carolina; 🇺🇸 Chapel Hill, North Carolina, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States