NCT02702115View on ClinicalTrials.gov

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

PHASE1/PHASE2TerminatedINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

May 24, 2017

Primary Completion Date

November 3, 2021

Study Completion Date

November 3, 2021

Conditions

MPS I

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.

Trial Locations (1)

94609

UCSF Benioff Children's Hospital Oakland, Oakland