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

Sangamo Therapeutics

Status

Active, not recruiting

Conditions

Mucopolysaccharidosis I

Hemophilia B

Mucopolysaccharidosis II

Treatments

Biological: SB-318

Biological: SB-FIX

Biological: SB-913

Study type

Observational

Funder types

Industry

Identifiers

NCT04628871

ST-IVPRP-LT01

Details and patient eligibility

About

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.

Full 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.

Enrollment

13 estimated patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

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.