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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: Clinical Trial Phase 1 study, sponsored by Sangamo Therapeutics.
NCT03041324 is a Phase 1 study that was terminated before completion, run by Sangamo Therapeutics. The registered enrollment target is 9 participants, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower). According to ClinicalTrials.gov, the official US trial registry.
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.
Primary Outcome
Number of participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) in subjects who receive SB-913 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)
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 |
Why NCT03041324 stopped before completion
NCT03041324 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 60-participant average among 29,502 other Phase 1 trials with a reported enrollment target (85% lower).
The record links to 0 conditions, and to 1 intervention - of which SB-913 is the first listed.
NCT03041324 does not publish any study locations in the registry export this page uses.
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.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03041324's enrollment target sits among peer trials
9 1955th of 2000 higher than 42 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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