This study is accepting new patients by invitation only

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

a study on Hemophilia Mucopolysaccharidosis

Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Oakland, California and other locations
Dates
study started
estimated completion

Description

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.

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

Details

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.

Keywords

Hemophilia B Mucopolysaccharidosis I Mucopolysaccharidosis II Hurler-Scheie Syndrome Hunter Syndrome Gene Editing Gene Therapy Zinc Finger SB-318 SB-913 SB-FIX Rare Genetic DNA Sangamo ZFN Long Term MPS Hemophilia A Mucopolysaccharidoses

Eligibility

You can join if…

Open to people ages 18 years and up

  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.

You CAN'T join if...

  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.

Locations

  • UCSF Benioff Children's Hospital Oakland
    Oakland California 94609 United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Chicago Illinois 60611 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Sangamo Therapeutics
ID
NCT04628871
Study Type
Observational
Last Updated