EUCTR2017-004805-42-GB进行中（未招募）1 期
A Phase I, Open-Label, Ascending Dose Study to Assess the Safety and Tolerability of AAV2/6 Factor IX Gene Therapy via Zinc Finger Nuclease (ZFN) mediated targeted integration of SB-FIX in Subjects with Severe Haemophilia B
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 16
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
- •Inclusion Criteria
- •1. Signed informed consent
- •2. Male =18 years of age and older (adult cohort); Male 12-17 years of age (pediatric cohort)
- •3. Severe heamophilia B (native circulating FIX activity <1%, with or without cross reactive material)
- •4. FIX mutations confirmed by FIX genome sequencing
- •5. With 150 or more exposure days to FIX concentrates
- •6. Sexually active subject must agree to use double barrier contraceptive (one of them being a condom) or abstinence and all subjects must refrain from donating their sperm until at least 2 consecutive semen samples after SB-FIX are negative for AAV 2/6 and for a minimum of 90 days after SB-FIX administration
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 3
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 2
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Exclusion Criteria
- •1. Presence of neutralizing antibodies to AAV 2/6
- •2. History of hypersensitivity response or allergic reaction to FIX or FIX products
- •3. Currently receiving long acting FIX replacement therapy and unwilling to switch to short acting FIX infusions
- •4. FIX mutations known to be associated with FIX inhibitors, including those with a large gene deletion
- •5. Polymorphisms in the ZFN target region of the albumin locus
- •6. Presence of any liver mass on MRI or equivalent imaging technology, or elevated alpha-fetoprotein (AFP)
- •7. Any contraindication to the use of corticosteroids for immunosuppression
- •8. Currently receiving antiviral therapy for hepatitis B or C or with history of active hepatitis B or hepatitis C or HIV-1 or HIV 1/2 antibody positive. To be considered HCV-negative after an active HCV infection, viral assays in two samples collected at least six months apart must be negative
- •9. Chronic anaemia, leukopenia, or thrombocytopenia
- •10. Past medical history of active tuberculosis or systemic fungal disease
- •11. Symptomatic cardiovascular disease as a co-morbid condition
- •12. Markers of hepatic inflammation or overt or occult cirrhosis as evidenced by one or more of the following:
- •Albumin =3.5 g/dL (35g/L)
- •Total bilirubin >1.5 x ULN and direct bilirubin =0.5 mg/dL (8.55 µmol/L)
- •Alkaline phosphatase >2.0 x ULN
- •ALT or AST >2.0 x ULN
- •13. History of chronic renal disease or creatinine = 1.5 mg/dL (133 µmol/L)
- •14. Systemic (iv or oral) immunomodulatory agent or steroid use (topical treatment is allowed, e.g. for asthma or eczema) within 3 months prior to Screening
- •15. History of chronic infection or other chronic disorder considered an unacceptable risk
- •16. History of malignancy except for treated basal cell or squamous cell carcinoma
- •17. History of alcohol or substance abuse
- •18. Previously received gene therapy product
- •19. Participation in prior investigational drug or medical device study within the previous 3 months
- •20. History of therapeutic non-adherence
- •21. Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for participation in the study
研究者
相似试验
未知
1 期
A Phase I, Open-Label, Dose Escalation Study to Assess the Safety and Tolerability of AZD2281 Following Single and Multiple Oral Doses in Patients in Japan With Advanced Solid MalignanciesAdvanced Solid MalignanciesJPRN-jRCT2080220492AstraZeneca
未知
1 期
A Phase 1, Open-Label, Dose-Escalation Study to Assess Safety, Tolerability & Pharmacokinetics of AZD7762 Administered as a Single Intravenous Agent and in Combination w/ Wkly Standard Dose Gemcitabine in Japanese Patients w/ Advanced Solid MalignanciesJPRN-jRCT2080220802AstraZeneca45
未知
1 期
A Phase I, Open-Label, Dose-Escalation Study to Assess the Safety and Tolerability of AZD0530 in Patients With Advanced Solid Malignanciesadvanced solid malignanciesJPRN-jRCT2080220617AstraZeneca
未知
1 期
A Phase I, Open-Label, Dose-Escalation Study to Assess the Safety, Tolerability and Pharmacokinetics of AZD4877 Administered Weekly in Japanese Adult Patients With Advanced Solid MalignanciesCancer, Tumors, CarcinomaJPRN-jRCT2080220530AstraZeneca
招募中
不适用
A dose-escalation, open label phase I study to assess the safety, feasibility and preliminary efficacy of HA-1H TCR modified T cells, MDG1021, in patients with relapsed or persistent hematologic malignancies after allogeneic hematological stem cell transplantation with or without unmanipulated donor lymphocyte infusion.blood diseaseNL-OMON55022Miltenyi Biomedicine29