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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
Drug Product 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
Therapeutic Modality Gene editing
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAV2/6
Editor Type ZFN
Dose 1 1E13 vg/kg (n=1)
Dose 2 5E13 vg/kg (n=2)

Study Record Dates


Current Phase 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