Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
Mucopolysaccharidosis II, MPS II
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis II focused on measuring MPS ll, Mucopolysaccharidosis II, Hunter syndrome, Gene Editing, Gene therapy, Zinc Finger, ZFN, SB-913, Rare, Genetic, DNA, Sangamo, Genome editing, Champions, Hunter, Gene Specific Targeted Insertion
Eligibility Criteria
Inclusion Criteria:
- Male or female 5 years to 65 years of age.
- Clinical diagnosis of MPS II (based on evidence of hepatosplenomegaly, dysostosis multiplex by X-ray, valvular heart disease, or obstructive airway disease) IDS deficiency confirmed by gene sequencing.
Exclusion Criteria:
- Known to be unresponsive to ERT
- Neutralizing antibodies to AAV 2/6
- Serious intercurrent illness or clinically significant organic disease (unless secondary to MPS II)
- Receiving antiviral therapy for hepatitis B or C, or with active hepatitis B or hepatitis C or HIV 1/2
- Lack of tolerance to idursulfase treatment with significant IARs or occurrence of anaphylaxis
- Markers of hepatic dysfunction
- Creatinine ≥ 1.5 mg/dL
- Contraindication to the use of corticosteroids for immunosuppression
- Current treatment with systemic (IV or oral) immunomodulatory agent or steroid use (topical treatment allowed)
- Participation in prior investigational drug or medical device study within the previous 3 months
- Prior treatment with a gene therapy product
- Elevated or abnormal circulating α-fetoprotein (AFP)
- Weight < 20 kg at Screening Visit
Sites / Locations
- UCSF Benioff Children's Hospital Oakland
- Ann & Robert H. Lurie Children's Hospital of Chicago
- NYU School of Medicine, Neurogenetics Division
- University of North Carolina
- Cincinnati Children's Hospital Medical Center
Arms of the Study
Arm 1
Arm 2
Arm 3
Experimental
Experimental
Experimental
Cohort 1 SB-913 Starting Dose 5.00E+12 vg/kg
Cohort 2 SB-913 at Next Ascending Dose 1.00E+13 vg/kg
Cohort 3 SB-913 at Next Ascending Dose 5.00E+13 vg/kg
A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion.
A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion.
A single dose of each of the three components of SB-913 [zinc finger nucleases (ZFN1, ZFN2, and hIDUA Donor)] administered via intravenous (IV) infusion.