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）
- 入组人数
- 120
- 试验地点
- 8
- 主要终点
- Annualized bleeding rate (ABR)
研究概览
简要总结
The purpose of this study is to determine the frequency of bleeding episodes in adult and adolescent patients receiving fitusiran as prophylactic treatment of hemophilia compared with patients who are assigned to continue with their regular medication. In addition, the study will assess safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Open Label
入排标准
- 年龄范围
- 12.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- Male
入选标准
- •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.
- •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.
- •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
- •Known co-existing bleeding disorders other than hemophilia A or B, ie, Von Willebrand’s disease, additional factor deficiencies, or platelet disorders.
- •Current use of factor concentrates as regularly administered prophylaxis designed to prevent spontaneous bleeding episodes.
- •AT activity <60% at Screening as determined by central laboratory measurement.
- •Presence of clinically significant liver disease, or as indicated by any of the conditions below: a.
- •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
- •Hepatitis C virus antibody positive, except patients with a history of HCV infection who meet both conditions 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.
- •No evidence of cirrhosis
- •Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
- •Presence of acute or chronic hepatitis B infection (IgM anti-HBc antibody positive or HBsAg positive).
- •Platelet count ≤100,000/μL.
- •Presence of acute infection at Screening.
- •Known to be HIV positive with CD4 count <200 cells/μL.
- •Estimated glomerular filtration rate ≤45 mL/min/1.73m2 (using the Modification of Diet in Renal Disease [MDRD] formula).
- •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.
- •Protein S deficiency c.
- •Protein C deficiency d.
- •Prothrombin mutation (G20210A; homozygous or heterozygous)
- •History of antiphospholipid antibody syndrome.
- •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.
- •Completion of a surgical procedure within 14 days prior to Screening, or currently receiving additional factor infusion for postoperative hemostasis.
- •History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
- •Inadequate venous access, as determined by the Investigator, to allow the blood draws required by the study protocol.
- •History of intolerance to SC injection(s).
- •Current or future participation in another clinical study, scheduled to occur during this study, involving an investigational product other than fitusiran or investigational device; in order to participate in this study, patient must discontinue the investigational product at least 30 days (or 5× the investigational product half-life, whichever is longer) prior to dosing (Day 1).
- •History of alcohol abuse within the 12 months before Screening.
结局指标
主要结局
Annualized bleeding rate (ABR)
时间窗: Annualized bleeding rate (ABR) : 9 months
次要结局
- 1. Annualized spontaneous bleeding rate(2. Annualized joint bleeding rate)
研究者
研究点 (8)
Loading locations...
相似试验
已完成
3 期
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With InhibitorsHemophilia BHemophilia ANCT03417102Genzyme, a Sanofi Company60
进行中（未招募）
3 期
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 IXCTRI/2018/07/014698Sanofi Genzyme
已完成
4 期
Bleeding Pattern and User Satisfaction During Second Consecutive MIRENA® in Contraception and Treatment of MenorrhagiaContraceptionMenorrhagiaNCT00393198Bayer204
已完成
不适用
Management of Major Bleeding Events in Atrial Fibrillation Patients Using PradaxaHemorrhageAtrial FibrillationNCT02149303Boehringer Ingelheim191
已完成
3 期
A Study Comparing Upadacitinib (ABT-494) to Placebo and to Adalimumab in Participants With Psoriatic Arthritis Who Have an Inadequate Response to at Least One Non-Biologic Disease Modifying Anti-Rheumatic Drug (DMARD)Psoriatic ArthritisNCT03104400AbbVie1,705