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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04628871
Recruitment Status : Active, not recruiting
First Posted : November 16, 2020
Last Update Posted : September 26, 2023
Sponsor:
Information provided by (Responsible Party):
Sangamo Therapeutics

Study Description
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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
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
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Study Type : Observational
Estimated 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 : November 3, 2020
Estimated Primary Completion Date : January 1, 2030
Estimated Study Completion Date : January 1, 2030

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort 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
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Primary Outcome Measures :
  1. 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.


Biospecimen Retention:   Samples With DNA
plasma, serum

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04628871


Locations
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United States, California
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
United States, New York
New York University Grossman School of Medicine
New York, New York, United States, 10016
United States, North Carolina
University of North Carolina
Chapel Hill, North Carolina, United States, 27514
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Sangamo Therapeutics
Investigators
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Study Director: Medical Monitor, MD Sangamo Therapeutics
More Information
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Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT04628871    
Other Study ID Numbers: ST-IVPRP-LT01
First Posted: November 16, 2020    Key Record Dates
Last Update Posted: September 26, 2023
Last Verified: January 2023

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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:
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Mucopolysaccharidosis II
Hemophilia A
Hemophilia B
Mucopolysaccharidoses
Mucopolysaccharidosis I
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
 Metabolism, Inborn Errors
Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases
Genetic Diseases, X-Linked
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System