Clinical trial NCT04628871: Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
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
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hemophilia B
Mucopolysaccharidosis I
Mucopolysaccharidosis II
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
Lead sponsor: Sangamo Therapeutics
Sponsor class: INDUSTRY
First posted: 2020-11-16
Enrollment: 13
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Long-term safety
timeFrame: 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: Oakland
country: United States
facility: UCSF Benioff Children's Hospital Oakland
state: California
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital of Chicago
state: Illinois
city: New York
country: United States
facility: New York University Grossman School of Medicine
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Sangamo Therapeutics
name: Medical Monitor, MD
role: STUDY_DIRECTOR
References: