EUCTR2019-004210-33-GB进行中（未招募）1 期
A Phase 1/2 Open-Label, Dose-Escalation, Safety, Tolerability, and Efficacy Study of SIG-001 in Adult Patients with Severe or Moderately-Severe Haemophilia A Without Inhibitors
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 18
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
- 性别
- Male
入选标准
- •1. Males aged 18 years or older
- •2. Diagnosis of Haemophilia A as follows:
- •a) Severe (<1% FVIII activity) receiving FVIII prophylaxis or on-demand treatment; or
- •b) Moderately-severe (=1% - =2% FVIII activity) receiving FVIII prophylaxis
- •3. Greater than 150 exposure days to treatment with FVIII products
- •4. Fertile males (not surgically sterilized) who have sexual partners that are women of childbearing potential must be willing to use a barrier method of contraception (e.g. condoms) for at least 90 days post-SIG-001 administration
- •5. Normal levels of von Willebrand factor (VWF) antigen (=50 IU/dL)
- •6. Able and willing to provide informed consent
- •7. Willing to withdraw from FVIII prophylaxis during specified periods in the study
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 16
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •1. Morbid obesity defined as body mass index (BMI) =35
- •2. Current FVIII inhibitors (>0.6 NBU/mL) or prior Immune Tolerance Induction (ITI)
- •Note: Patients with a history of low and/or transient inhibitors require Sponsor review and approval.
- •3. History of allergic reaction or anaphylaxis to recombinant FVIII products, alginate (including seaweed and algae), or barium and barium products (including barium contrast agents)
- •4. Evidence of any bleeding disorder in addition to haemophilia A
- •5. Abnormal laboratory values, as follows:
- •a) Platelet count <100 × 109/L
- •b) Creatinine =1.5 mg/dL
- •c) Haemoglobin <11 g/dL
- •d) Elevated levels (i.e. greater than 2 × upper limit of normal (ULN) of aspartate aminotransferase (AST), alanine aminotransferase (ALT), or total bilirubin
- •Note: Patients with a known history of Gilbert’s disease and bilirubin levels above ULN are not excluded.
- •6. Active infection with Hepatitis B or Hepatitis C virus, defined as a positive HBsAg, HBeAg, HBV DNA, or HCV RNA results, or currently managed with antiviral medications for Hepatitis B or C
- •7. Uncontrolled HIV infection, defined as CD4+ counts =200/µL or by a viral load of >200 copies/mL
- •8. Active alcoholism or drug addiction during the 12 months before the screening visit
- •9. Active malignancy or history of malignancy in the 5 years prior to study entry, exclusive of surgically removed non-melanoma skin cancer
- •10. Participation in another investigational medicine or device study within 90 days of screening or 5 investigational product half-lives, whichever is longer
- •11. Prior administration of a gene therapy product
- •12. Treatment with emicizumab less than 6 months prior to screening.
- •Note: Patients with emicizumab exposure may be eligible before 6 months of prior exposure if FVIII activity assessed by one stage assay is back to pre-emicizumab treatment levels.
- •13. Unable to tolerate general anaesthesia required for the laparoscopic procedure
- •14. History of:
- •a) Abdominal adhesions due to prior large, median laparotomy(ies)
- •Note: Uncomplicated prior laparoscopic procedures are not exclusionary (e.g. cholecystectomy, laparoscopic appendectomy or diagnostic laparoscopy).
- •b) Septic peritonitis
- •c) Intraperitoneal mesh
- •d) Inflammatory Bowel Disease (IBD), including ulcerative colitis or Crohn’s disease
- •15. Other comorbidities that in the opinion of the Investigator increase the risk of abdominal adhesions
- •16. Any disease or medical condition that in the Investigator’s opinion is a contraindication for anaesthesia or the laparoscopic procedure (e.g. prior significant cardiovascular events)
- •17. In the Investigator’s judgment, the patient is unlikely to complete all protocol-required study visits, procedures, or comply with the study requirements for participation
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