EUCTR2018-000206-28-GB进行中（未招募）1 期
A Phase 1/2, 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)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 27
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •Subjects must meet all of the following criteria to be included in the study:
- •1. Signed informed consent
- •2. =5 years of age:
- •a) adult cohorts 1 through 3: =18 years of age;
- •b) pediatric cohorts 4 and 5: 12 to 17 years of age; and
- •c) pediatric cohorts 6 and 7: 5 to 11 years of age.
- •3. Clinical diagnosis of MPS I; IDUA deficiency confirmed by gene sequencing
- •4. Sexually mature subjects must agree to use a barrier contraceptive method for prevention of AAV transfer as follows: for female subjects this means that the subjects’ partners must use a condom from dosing with SB-318 until at least 3 consecutive plasma samples after administration of SB-318 are negative for AAV2/6; for male subjects this means that the subjects must use a condom and must refrain from sperm donation from the time of SB-318 until at least 3 consecutive semen samples after administration of SB-318 are negative for AAV2/6. Additionally, female participants of child-bearing potential must consent to use a highly effective method of contraception.
- •5. Magnetic resonance imaging (MRI) negative for liver mass as read by a radiologist.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 16
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 9
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Subjects who meet any of the following criteria will be excluded from participating in the study:
- •1. Known to be unresponsive to ERT.
- •2. Neutralizing antibodies in serum (immune response) to AAV2/6.
- •3. Serious intercurrent illness or clinically significant organic disease (unless secondary to MPS I) such as cardiovascular, hepatic, pulmonary, neurologic, or renal disease.
- •4. Receiving antiviral therapy for hepatitis B or C, or with active hepatitis B (HBV DNA positive or HBV surface antigen positive) or hepatitis C (HCV RNA viral load) or human immunodeficiency virus (HIV)-1/2 (HIV RNA viral load or HIV antibody positive); to be considered negative for hepatitis C after treatment of an active HCV infection, viral assays in 2 samples collected at least 6 months apart must be negative.
- •5. Lack of tolerance to ERT with significant infusion-associated reactions (IARs) or occurrence of anaphylaxis.
- •6. Polymorphisms in the ZFN-targeted region of the albumin locus.
- •7. Liver fibrosis score of 3 or 4 on a 0 to 4 point scale (Desmet et al. 1994) if subject has had a liver biopsy within 2 years of Screening.
- •8. Markers of hepatic dysfunction as evidenced by one or more of the following:
- •a. Platelet count <100,000/µL
- •b. Albumin =3.2 g/dL
- •c. Total bilirubin >1.5 x upper limit of normal (ULN) and direct bilirubin =0.5 mg/dL
- •d. Alkaline phosphatase >2.0 x ULN
- •e. ALT or AST >2.0 x ULN
- •9. Creatinine = 1.5 mg/dL.
- •10. Weight < 20 kg at Screening.
- •11. Pregnant or breastfeeding female.
- •12. Contraindication to the use of corticosteroids.
- •13. Current treatment with systemic (IV or oral) immunomodulatory agent or steroid use (topical treatment allowed, e.g., for asthma or eczema).
- •14. History of active malignancy in past 5 years (non-melanoma skin cancer or cervical cancer in situ permitted).
- •15. Participation in prior investigational drug or medical device study within the previous 3 months.
- •16. Prior treatment with a gene therapy product.
- •17. History of alcohol or substance abuse that in the opinion of the Principal Investigator may interfere with study compliance.
- •18. History of therapeutic non-adherence.
- •19. Elevated or abnormal circulating a-fetoprotein (AFP).
- •20. Any other reason that, in the opinion of the Principal Investigator or Medical Monitor, would render the subject unsuitable for participation in the study.
研究者
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