EUCTR2020-003445-11-IT进行中（未招募）1 期
A Phase II, double-blind, placebo-controlled, Randomized, cross-over, dose-ranging study of oral PHA-022121 for Acute treatment of angioedema attacks in Patients with hereditary angioedema due to C1-Inhibitor Deficiency type I and II - RAPIDe-1
Pharvaris Netherlands B.V.0 个研究点目标入组 54 人开始时间: 2021年6月15日最近更新:
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 54
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
- All
入选标准
- •1. Provision of signed and dated informed consent form
- •2. Male or female, aged = 18 and = 75 years at enrollment
- •3. Diagnosis of HAE (type I or II) based upon all of the following:
- •a. Documented clinical history consistent with HAE (subcutaneous or mucosal, nonpruritic swelling without accompanying urticaria)
- •b. At least one of the following:
- •- Age at reported onset of first angioedema symptoms = 40 years
- •- Family history consistent with HAE type I or II
- •- C1q within normal range
- •c. Diagnostic testing results to confirm HAE type I or II:
- •- C1-INH functional level < 50% of the normal level
- •The diagnosis may be established by local laboratory values documented in the medical records or by genotyping of the C1-INH gene (SERPING1).
- •Before entering Part II of the study (home treatment), the diagnosis needs to be confirmed by a central laboratory assessment or by genotyping of the C1-INH gene (SERPING1).
- •4. Documented history of at least three HAE attacks in the last 4 months, or at least two HAE attacks in the last 2 months.
- •5. Reliable access and experience to use standard of care treatment to effectively manage acute HAE attacks
- •6. Capable to record PRO data using the ePRO device
- •7. Female patients of childbearing potential must agree to be abstinent or to use highly effective forms of contraception methods from enrollment through the end of the study. This includes progestin-only oral contraceptive associated with inhibition of ovulation (oral, injectable, or implantable), intrauterine device (IUD, all types) or intrauterine hormone releasing systems (IUS). A female of childbearing potential whose male partner has had a vasectomy must agree to use one additional form of medically acceptable contraception.
- •Male patients, including males who are surgically sterile (post vasectomy), who have a female partner of childbearing potential must agree to be sexually abstinent or use a medically acceptable form of barrier contraception for 2 weeks after each administration of study drug. In addition, they must agree to not donate sperm during study participation.
- •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 40
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 14
排除标准
- •1. Pregnancy or breast-feeding
- •2. Clinically significant abnormal ECG, most notably a QTcF > 470 ms (for females) or > 450 ms (for males)
- •3. Any clinically significant history of angina, myocardial infarction, syncope, stroke, left ventricular hypertrophy or cardiomyopathy, or any other cardiovascular abnormality within the previous year
- •4. Any other systemic disease (e.g., gastrointestinal, renal, respiratory, neurological) or significant disease or disorder that would interfere with the patient's safety or ability to participate in the study
- •5. Use of:
- •a. long-term prophylactic therapy for HAE (C1-INH, oral kallikrein inhibitors, attenuated androgens, or anti-fibrinolytics) within 2 weeks prior to enrollment
- •b. long-term prophylactic monoclonal therapy for HAE (e.g., lanadelumab) within 12 weeks prior to enrollment
- •c. acute C1-INH treatment or short-term prophylaxis for HAE within 7 days prior to enrollment
- •Short-term prophylaxis is defined as C1-INH, attenuated androgens, or anti-fibrinolytics to avoid angioedema complications from medically indicated procedures.
- •6. Clinical history of resistance to B2 receptor antagonists
- •7. Positive serology for human immunodeficiency virus (HIV) or active infection with hepatitis B virus (HBV) or hepatitis C virus (HCV)
- •8. Abnormal hepatic function (AST > 2×ULN, ALT > 2×ULN, or total bilirubin > 1.5×ULN)
- •9. Abnormal renal function (eGFR CKD-EPI < 60 mL/min/1.73 m2)
- •10. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse (self-reported alcoholic intake > 3 drinks/day)
- •11. History of documented severe hypersensitivity to any medicinal product
- •12. Participation in any other investigational drug study currently, within the last 30 days or within 5 half-lives of study drug at enrollment (whichever was longer)
- •13. Regular use of corticosteroids, antihistamines, narcotics, and other pain relief medications for acute HAE attack treatment
- •14. Use of concomitant medication that are moderate or potent inhibitors/inducers of CYP3A4 or are metabolized by CYP3A4 and have a narrow therapeutic range, such as clarithromycin, erythromycin, diltiazem, itraconazole, ketoconazole, ritonavir, verapamil, goldenseal and grapefruit as well as phenobarbital, phenytoin, rifampicin, St. John's Wort, and glucocorticoids (not for topical use or inhalation)
研究者
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