CTRI/2023/06/054541尚未招募3 期
A Phase 3, single-arm, multicenter, multinational, openlabel, one-way crossover study to investigate the efficacy and safety of fitusiran prophylaxis in male participants aged â?¥ 12 years with severe hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX - ATLAS-NEO
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
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
- •Participants are eligible to be included in the study only if all of the following criteria apply:
- •I 01. Males (adult and adolescent) >= 12 years of age at the time of signing the informed consent
- •and/or assent as applicable.
- •Type of participant and disease characteristics
- •I 02. Diagnosis of severe congenital hemophilia A or B (FVIII < 1% or FIX level <= 2%) as
- •evidenced by a central laboratory measurement at screening or documented medical record
- •I 03. For participants currently not on prophylaxis (CFC or BPA on-demand): A minimum of
- •4 bleeding episodes requiring BPA (inhibitor participants) or CFC (non-inhibitor
- •participants) treatment within the last 6 months prior to screening.
- •I 04. Must meet either the definition of inhibitor or non-inhibitor patient as below:
- •Requiring use of BPA/ emicizumab (Hemlibra®) for prophylaxis or BPA as on-demand
- •therapy for any bleeding episodes for at least the last 6 months prior to screening, and
- •meet one of the following Nijmegen-modified Bethesda assay results criteria:
- •- Inhibitor titer of >= 0.6 Bethesda units (BU)/mL at Screening, or
- •- Inhibitor titer of < 0.6 BU/mL at Screening with medical record evidence of
- •2 consecutive titers >= 0.6 BU/mL, or
- •- Inhibitor titer of < 0.6 BU/mL at Screening with medical record evidence of 1 inhibitor
- •titer >= 0.6 BU/mL and a history of severe allergic reaction (eg, anaphylaxis) or
- •nephrotic syndrome
- •Non-inhibitor:
- •Requiring use of CFC/ emicizumab (Hemlibra®) for prophylaxis or CFC as on-demand
- •therapy for any bleeding episodes for at least the last 6 months prior to screening, and
- •meet each of the following criterion:
- •- Nijmegen-modified Bethesda assay inhibitor titer of <0.6 BU/mL at Screening and
- •- No use of BPA to treat bleeding episodes for at least the last 6 months prior to Screening and
- •- No history of ITI therapy within the past 3 years prior to Screening.
- •Not Applicable
- •Sex, contraceptive/barrier method and pregnancy testing requirements
- •I 05. Male.
- •There are no contraceptive requirements for this study except where required by local
- •regulations.
- •Informed Consent
- •I 06. Willing and able to comply with the study requirements and to provide written informed
- •consent and assent in the case of participants under the age of legal consent, per local and national requirements
排除标准
- •Participants are excluded from the study if any of the following criteria apply:
- •Medical conditions
- •E 01. Known co-existing bleeding disorders other than congenital hemophilia A or B, ie, Von
- •Willebrand disease, additional factor deficiencies, acquired hemophilia or platelet
- •E 02. History of antiphospholipid antibody syndrome.
- •E 03. History of arterial or venous thromboembolism, atrial fibrillation, significant valvular
- •disease, myocardial infarction, angina, transient ischemic attack, or stroke. Participants
- •who have experienced thrombosis associated with indwelling venous access may be
- •E 04. History of malignancy within 2 years prior to screening, except for basal or squamous cell
- •carcinoma of the skin that has been successfully treated.
- •E 05. Completion of a surgical procedure within 14 days prior to screening, or currently
- •receiving additional CFC or BPA infusion for postoperative hemostasis
- •E 06. At screening, anticipated need of surgery during the study or planned surgery scheduled to
- •occur during the study.
- •E 07. History of intolerance to SC injection(s).
- •E 08. History of alcohol abuse within the 12 months before Screening. Alcohol abuse in this
- •study is defined as regular weekly intake of more than 14 units (1 unit equals to 1 glass of wine
- •(Approximately 125 Ml) equals to 1 measure of spirits (approximately 1 fluid ounce) equals to half pint of
- •beer (approximately 284 mL).
- •E 09. Presence of clinically significant liver disease or any of the conditions below:
- •History of portal hypertension, esophageal varices, or hepatic encephalopathy;
- •Presence of ascites by physical exam
研究者
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