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NCT02702115 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study, sponsored by Sangamo Therapeutics.

Terminated
Registry status
Phase 1
Development phase
3
Enrollment target

NCT02702115: Clinical Trial Phase 1 study, sponsored by Sangamo Therapeutics.

NCT02702115 is a Phase 1 study that was terminated before completion, run by Sangamo Therapeutics. The registered enrollment target is 3 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (95% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT02702115, a Phase 1 study, was terminated before completion, sponsored by Sangamo Therapeutics.

TERMINATED
Registry status
Phase 1
Development phase
3 participants
Enrollment target

Study 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.

Primary Outcome

Number of Participants with Treatment-Emergent Adverse Events

Interventions

  • BIOLOGICAL SB-318

Trial Details

FieldValue
Enrollment Target 3 participants
Start Date 2017-05-24
Est. Completion 2021-11-03
Phase Phase 1
Sangamo Therapeutics

25 total trials

Why NCT02702115 stopped before completion

NCT02702115 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 3 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (95% lower).

The record links to 0 conditions, and to 1 intervention - of which SB-318 is the first listed.

NCT02702115 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT02702115 about?

NCT02702115 is a clinical study titled "Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I". 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 g...

What is the current status of trial NCT02702115?

This trial is currently terminated. It is a Phase 1 study. The enrollment target is 3 participants. The study started on 2017-05-24. Estimated completion is 2021-11-03.

What interventions are being tested in trial NCT02702115?

The interventions under investigation include: SB-318 (BIOLOGICAL).

Who is sponsoring clinical trial NCT02702115?

This trial is sponsored by Sangamo Therapeutics, which has 25 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT02702115's enrollment target sits among peer trials

3 1999th of 2000 higher than 1 of 2,000 other Phase 1 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 1 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT02702115, the US trial registry maintained by the National Library of Medicine. NCT02702115 (small enrollment · none site footprint · terminated) retrieved and formatted by PlainTrial, see methodology.