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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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1 November 2021 |
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Main ID: |
NCT02702115 |
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Date of registration:
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29/02/2016 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
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Scientific title:
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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) |
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Date of first enrolment:
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May 24, 2017 |
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Target sample size:
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3 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT02702115 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Medical Monitor |
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Address:
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Telephone:
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Email:
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Affiliation:
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Sangamo Therapeutics |
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Key inclusion & exclusion criteria
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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 a-fetoprotein (AFP)
- Weight <20 kg at Screening Visit
Age minimum:
5 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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MPS I
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Intervention(s)
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Biological: SB-318
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Primary Outcome(s)
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Incidence of Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
[Time Frame: Up to 36 months after the SB-318 infusion]
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Secondary Outcome(s)
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Effect of SB-318 on IDUA activity
[Time Frame: Up to 36 months after the SB-318 infusion]
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AAV2/6 clearance in plasma, saliva, urine, stool, and semen
[Time Frame: Up to 36 months after the SB-318 infusion]
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Effect of SB-318 on urine glycosaminoglycans (GAG) levels
[Time Frame: Up to 36 months after the SB-318 infusion]
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Secondary ID(s)
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SB-318-1502
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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