NCT04628871

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
13

participants targeted

Target at below P25 for all trials

Timeline
45mo left

Started Nov 2020

Longer than P75 for all trials

Geographic Reach
1 country

5 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress60%
Nov 2020Jan 2030

Study Start

First participant enrolled

November 3, 2020

Completed
6 days until next milestone

First Submitted

Initial submission to the registry

November 9, 2020

Completed
7 days until next milestone

First Posted

Study publicly available on registry

November 16, 2020

Completed
9.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2030

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2030

Last Updated

May 3, 2024

Status Verified

May 1, 2024

Enrollment Period

9.2 years

First QC Date

November 9, 2020

Last Update Submit

May 1, 2024

Conditions

Keywords

Mucopolysaccharidosis IHurler-Scheie SyndromeMucopolysaccharidosis IIHunter SyndromeGene EditingGene TherapyZinc FingerSB-318SB-913SB-FIXRareGeneticDNASangamoZFNLong TermHemophilia BMPS

Outcome Measures

Primary Outcomes (1)

  • Long-term safety

    Long-term safety of SB-318, SB-913, and SB-FIX by assessment of any newly-diagnosed or worsening of existing medical conditions.

    10 years

Study Arms (3)

Subjects who received SB-318

Subjects who received SB-318 in clinical study SB-318-1502

Biological: SB-318

Subjects who received SB-913

Subjects who received SB-913 in clinical study SB-913-1602.

Biological: SB-913

Subjects who received SB-FIX

Subjects who received SB-FIX in clinical study SB-FIX

Biological: SB-FIX

Interventions

SB-318BIOLOGICAL

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-318
SB-913BIOLOGICAL

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-913
SB-FIXBIOLOGICAL

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.

Subjects who received SB-FIX

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (5)

UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

New York University Grossman School of Medicine

New York, New York, 10016, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27514, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

plasma, serum

MeSH Terms

Conditions

Hemophilia BMucopolysaccharidosis IMucopolysaccharidosis II

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-LinkedMucopolysaccharidosesCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsLysosomal Storage DiseasesMucinosesConnective Tissue DiseasesSkin and Connective Tissue DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesX-Linked Intellectual DisabilityIntellectual DisabilityNeurobehavioral ManifestationsNeurologic ManifestationsNervous System DiseasesHeredodegenerative Disorders, Nervous System

Study Officials

  • Medical Monitor, MD

    Sangamo Therapeutics

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 9, 2020

First Posted

November 16, 2020

Study Start

November 3, 2020

Primary Completion (Estimated)

January 1, 2030

Study Completion (Estimated)

January 1, 2030

Last Updated

May 3, 2024

Record last verified: 2024-05

Locations