CTRI/2018/07/014698进行中（未招募）3 期
ATLAS-A/B: A Phase 3 Study to Evaluate theEfficacy and Safety of Fitusiran in Patients WithHemophilia A or B, Without Inhibitory Antibodiesto Factor VIII or IX
试验速览
- 阶段
- 3 期
- 状态
- 进行中（未招募）
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •1) Males, >=12 years of age
- •2) Severe hemophilia A or B without inhibitors evidenced by:
- •a. A central laboratory measurement or documented medical record evidence of
- •FVIII <1% or FIX level <=2% at Screening.
- •b. On-demand use of factor concentrate to manage bleeding episodes for at least the last
- •6 months prior to Screening
- •3) A minimum of 6 bleeding episodes requiring factor concentrate treatment within the last 6 months prior to Screening.
- •4) 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, per local and
- •national requirements.
排除标准
- •1. Known co-existing bleeding disorders other than hemophilia A or B, ie, Von
- •Willebrandâ??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
- •a. INR >1.2;
- •b. ALT and/or AST >1.5Ã? upper limit of normal reference range (ULN);
- •c. Total bilirubin >ULN ( >1.5 ULN in patients with Gilbertâ??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
- •b. No evidence of cirrhosis
- •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 patientâ??s safety and/or the patientâ??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
- •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 p
研究者
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