Contact SCGE




Gene Therapy Trial Report

Summary

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


NCTID NCT02702115 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Mucopolysaccharidosis Type I (Hurler Syndrome)
Disease Ontology Term DOID:12802
Compound Name SB-318
Sponsor Sangamo Therapeutics
Funder Type Industry
Recruitment Status
Terminated
Enrollment Count 3 (ACTUAL)
Results Posted View Results

Therapy Information


Target Gene/Variant IDUA
Therapy Type Gene editing
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV2/6
Editor Type ZFN
Dose 1 1E13 vg/kg (n=1)
Dose 2 5E13 vg/kg (n=2)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2016-02-29
Completion Date 2021-11-03
Last Update 2023-01-26

Participation Criteria


Eligible Age >=5 Years
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Male or female ≥ 5 years of age * Clinical diagnosis of attenuated MPS I deficiency (Hurler-Scheie, Scheie, or Hurlers status post-HSCT) 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 I) * Receiving antiviral therapy for hepatitis B or C, or with active hepatitis B or hepatitis C or HIV 1/2 * Lack of tolerance to laronidase 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
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 1
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
Recent Updates Only 3 subjects were enrolled prior to study termination, Sangamo announced they had stopped development in 2022

Resources/Links