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Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I

NCT02702115 · Status: TERMINATED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2023-01-26

Study results available
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Summary

The purpose of the study is to evaluate the safety, tolerability of ascending doses of SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the α-L-iduronidase (IDUA) gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDUA enzyme.

Conditions

Interventions

BIOLOGICAL

SB-318

A single dose of each of the three components of SB-318 \[zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)\] administered via intravenous (IV) infusion.

Sponsors & Collaborators

  • Sangamo Therapeutics

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Sangamo Therapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-05-24
Primary Completion
2021-11-03
Completion
2021-11-03
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02702115 on ClinicalTrials.gov