A Phase I / 2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-318, a rAAV2/6-based Gene Transfer in Subjects With Mucopolysaccharidosis I (MPS I)

Who is this study for? Patients with mucopolysaccharidosis
What treatments are being studied? SB-318
Status: Terminated
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Healthy Volunteers: No
View:

• Male or female ≥ 5 years of age

• Clinical diagnosis of attenuated MPS I deficiency (Hurler-Scheie, Scheie, or Hurlers status post-HSCT)

Locations
United States
California
UCSF Benioff Children's Hospital Oakland
Oakland
Time Frame
Start Date: May 24, 2017
Completion Date: November 3, 2021
Participants
Target number of participants: 3
Treatments
Experimental: Cohort 1: SB-318: Starting Dose
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.
Experimental: Cohort 2: SB-318 at Next Ascending Dose
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.
Experimental: Cohort 3: SB-318 at Next Ascending Dose
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
Leads: Sangamo Therapeutics

This content was sourced from clinicaltrials.gov

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