Adults 5 to 65, any sex, with Mucopolysaccharidosis II or MPS II. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) [ Time Frame: Up to 36 Months After the SB-913 InfusionPrimary· Up to 36 months after the SB-913 infusion
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)
Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg
9.9
± 25.5
Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
-0.5
± 6.13
AAV2/6 Clearance in Plasma, Saliva, Urine, Stool, and SemenSecondary· Up to 36 months after the SB-913 infusion
Subjects with AAV2/6 clearance in plasma, saliva, urine, stool, and semen by PCR by Week 24.
All the subjects had AAV2/6 clearance in all the samples assessed (i.e., plasma, saliva, urine, stool, and semen) by week 24.
Subjects were only tested until Week 24 because, by that time, they all had 3 consecutive negative tests in all body fluids.
Experimental: Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg
2
Experimental: Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
5
Adverse events — posted to ClinicalTrials.gov
Time frame: Adverse event data was collected from the subject's date of screening until their end of study visit which ranged from 24 to 36 months..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
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.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT04628871 — Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
· active not recruiting
Other recruiting trials for Mucopolysaccharidosis II
Currently open trials in the same condition.
NCT05619900 — Registry of Patients Diagnosed With Lysosomal Storage Diseases
· recruiting
NCT04573023 — A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
· Phase 3
· active not recruiting
NCT04628871 — Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
· active not recruiting
NCT04348136 — An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
· Phase 2, PHASE3
· active not recruiting
NCT03708965 — An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
· Phase 2
· active not recruiting
Other Sangamo Therapeutics trials
Trials by the same sponsor.
NCT05987527 — Long-Term Follow-Up of TX200-TR101 (STEADFAST Long Term)
· active not recruiting
NCT05145062 — Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.
· active not recruiting
NCT04817774 — Safety & Tolerability Study of Chimeric Antigen Receptor T-Reg Cell Therapy in Living Donor Renal Transplant Recipients
· Phase 1, PHASE2
· active not recruiting
NCT04628871 — Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
· active not recruiting
NCT04046224 — Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)
· Phase 1, PHASE2
· completed
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Sangamo Therapeutics
Last refreshed: 25 October 2022
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03041324.