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NCT03041324 · ClinicalTrials.gov registry record · Phase 1
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
A Phase 1 study, sponsored by Sangamo Therapeutics.
- Terminated
- Registry status
- Phase 1
- Development phase
- 9
- Enrollment target
NCT03041324 is a Phase 1 study that was terminated before completion, run by Sangamo Therapeutics. The registered enrollment target is 9 participants.
The verdict
NCT03041324, a Phase 1 study, was terminated before completion, sponsored by Sangamo Therapeutics.
- TERMINATED
- Registry status
- Phase 1
- Development phase
- 9 participants
- Enrollment target
Study Summary
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the IDS gene into the Albumin locus in hepatocytes with the goal of lifelong therapeutic production of the IDS enzyme.
Interventions
- BIOLOGICAL SB-913
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2017-05-11 |
| Est. Completion | 2021-05-07 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT03041324
The ClinicalTrials.gov registry entry for NCT03041324 describes a study currently listed as terminated, categorized as Phase 1. The registered enrollment target is 9 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sangamo Therapeutics, which has 25 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which SB-913 is the first listed.
NCT03041324 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT03041324 about?
NCT03041324 is a clinical study titled "Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II". The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913. SB-913 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of t...
What is the current status of trial NCT03041324?
This trial is currently terminated. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2017-05-11. Estimated completion is 2021-05-07.
What interventions are being tested in trial NCT03041324?
The interventions under investigation include: SB-913 (BIOLOGICAL).
Who is sponsoring clinical trial NCT03041324?
This trial is sponsored by Sangamo Therapeutics, which has 25 total clinical trials registered on ClinicalTrials.gov.
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