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

• Sponsor: Sangamo Therapeutics (Industry)
• Overall Status: Enrolling by invitation
• CT.gov ID: NCT04628871
• Collaborator: (none)
• Enrollment: 13
• Locations: 5
• Anticipated Duration (Months): 109.9
• Patients Per Site: 2.6
• Patients Per Site Per Month: 0

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Hemophilia B; Mucopolysaccharidosis I; Mucopolysaccharidosis II	Biological: SB-318; Biological: SB-913; Biological: 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 Design

• Study Type: Observational
• Anticipated Enrollment: 13 participants
• Observational Model: Case-Only
• Time Perspective: Prospective
• 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
• Actual Study Start Date: Nov 3, 2020
• Anticipated Primary Completion Date: Jan 1, 2030
• Anticipated Study Completion Date: Jan 1, 2030

Arms and Interventions

Arm	Intervention/Treatment
Subjects who received SB-318 Subjects who received SB-318 in clinical study SB-318-1502	Biological: SB-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.
Subjects who received SB-913 Subjects who received SB-913 in clinical study SB-913-1602.	Biological: SB-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.
Subjects who received SB-FIX Subjects who received SB-FIX in clinical study SB-FIX	Biological: SB-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.

Outcome Measures

Primary Outcome Measures

1. 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.

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	UCSF Benioff Children's Hospital Oakland	Oakland	California	United States	94609
2	Ann & Robert H. Lurie Children's Hospital of Chicago	Chicago	Illinois	United States	60611
3	New York University Grossman School of Medicine	New York	New York	United States	10016
4	University of North Carolina	Chapel Hill	North Carolina	United States	27514
5	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229

Sponsors and Collaborators

• Sangamo Therapeutics

Investigators

• Study Director: Medical Monitor, Sangamo Therapeutics

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Sangamo Therapeutics
• ClinicalTrials.gov Identifier: NCT04628871
• Other Study ID Numbers: ST-IVPRP-LT01
• First Posted: Nov 16, 2020
• Last Update Posted: Oct 25, 2021
• Last Verified: Oct 1, 2021

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No
• Keywords provided by Sangamo Therapeutics: 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
• Additional relevant MeSH terms: Mucopolysaccharidosis II; Hemophilia A; Hemophilia B; Mucopolysaccharidoses; Mucopolysaccharidosis I

Study Results

No Results Posted as of Oct 25, 2021