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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Hemophilia B Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Carbohydrate Metabolism, Inborn Errors Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders Heredodegenerative Disorders, Nervous System Intellectual Disability Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis I Mucopolysaccharidosis II Nervous System Diseases Neurobehavioral Manifestations Neurologic Manifestations Nutritional and Metabolic Diseases Skin and Connective Tissue Diseases X-Linked Intellectual Disability

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

UCSF Benioff Children's Hospital Oakland, Oakland, California, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

SB-318

Biological

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.

SB-913

Biological

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.

SB-FIX

Biological

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.

Primary outcomes

Long-term safety

Time frame: 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.

Sponsors and collaborators

Lead sponsor

Sangamo Therapeutics

Industry

Registry information

Official study 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

Acronym: LTFU

Important dates

Study start: 2020
Primary completion: 2030
Study completion: 2030
First posted: Nov 16, 2020
Registry last updated: May 3, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed