EUCTR2016-001464-11-HU进行中（未招募）1 期
ATLAS-A/B: A Phase 3 study to evaluate the efficacy and safety of fitusiran in patients with hemophilia A or B, without inhibitory antibodies to factor VIII or IX
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 90
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
- •Males =12 years of age.
- •Severe hemophilia A or B (evidenced by a central laboratory FVIII <1% or FIX level =2% at Screening) without inhibitors (evidenced by inhibitor titer of <0.6 BU/mL and supported by medical records)
- •AT activity =60% at Screening
- •A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months
- •Willing and able to comply with the study requirements and to provide written informed consent and assent in the case of patients under the age of legal consent
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 10
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 105
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •1. Known co-existing bleeding disorders other than hemophilia A or B, ie, Von Willebrand's disease, additional factor deficiencies, or platelet disorders.
- •2. Current use of factor concentrates as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes.
- •3. AT activity <60% at Screening as determined by central laboratory measurement.
- •4. Presence of clinically significant liver disease, or as indicated by any of the conditions below:
- •a. INR >1.2;
- •b. ALT and/or AST >1.5× upper limit of normal reference range (ULN);
- •c. Total bilirubin >ULN (>1.5 ULN in patients with Gilbert's Syndrome);
- •d. History of portal hypertension, esophageal varices, or hepatic encephalopathy;
- •e. Presence of ascites by physical exam
- •5. Hepatitis C virus antibody positive, except patients with a history of HCV infection who meet both conditions a. and b.:
- •a. Completed curative treatment at least 12 weeks prior to enrollment and attained sustained virologic response as documented by a negative HCV RNA at screening, or they have spontaneously cleared infection as documented by negative HCV RNA at Screening.
- •b. No evidence of cirrhosis according to one of the following assessments:
- •? FibroScan <12.5 kPa (where available), or
- •? FibroTest score <0.75 and APRI <2 (if FibroScan unavailable)
- •6. Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
- •7. Presence of acute or chronic hepatitis B infection (IgM anti-HBc antibody positive or HBsAg positive).
- •8. Platelet count =100,000/µL.
- •9. Presence of acute infection at Screening.
- •10. Known to be HIV positive with CD4 count <200 cells/µL.
- •11. Estimated glomerular filtration rate =45 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula).
- •12. Co-existing thrombophilic disorder, as determined by presence of any of the below as identified at central laboratory (or via historical results, where available):
- •a. FV Leiden (homozygous or heterozygous)
- •b. Protein S deficiency
- •c. Protein C deficiency
- •d. Prothrombin mutation (G20210A; homozygous or heterozygous)
- •13. History of antiphospholipid antibody syndrome.
- •14. History of arterial or venous thromboembolism, atrial fibrillation, significant valvular disease, myocardial infarction, angina, transient ischemic attack, or stroke. Patients who have experienced thrombosis associated with indwelling venous access may be enrolled.
- •15. Had a malignancy within 2 years, except for basal or squamous cell carcinoma of the skin that has been successfully treated.
- •16. Any condition (eg, medical concern), which in the opinion of the Investigator, would make the patient unsuitable for dosing on Day 1 or which could interfere with the study compliance, the patient's safety and/or the patient's participation in the completion of the treatment period of the study. This includes significant active and poorly controlled (unstable) cardiovascular, neurologic, gastrointestinal, endocrine, renal or psychiatric disorders unrelated to hemophilia identified by key laboratory abnormalities or medical history.
- •17. At Screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study.
- •18. Completion of a surgical procedure within 14 days prior to Screening, or currently receiving additional factor infusion for postoperative hemostasis.
- •19. History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
- •20. Inadequate venous access, as determined by t
研究者
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