NL-OMON53637招募中不适用
A randomised, placebo controlled, double blind, multicentre proof of concept study to assess the safety and efficacy of two doses of VAD044 in patients with hereditary haemorrhagic telangiectasia (HHT) - VAD044C002
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 45
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
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Patients aged >=18 years.
- •2. Able to understand and comply with the requirements for the study, including
- •the epistaxis app completion using a smartphone.
- •3. Patients with definite diagnosis of HHT by the Curaçao criteria defined as
- •having at least 3 of the following criteria:
- •a. Spontaneous and recurrent epistaxis;
- •b. Multiple telangiectases at characteristic sites: lips, oral cavity, fingers,
- •c. Visceral lesions: GI telangiectasia, pulmonary, hepatic, cerebral or spinal
- •d. A first degree relative with HHT according to these criteria.
- •4. Patients with typically several (>=5) epistaxis per week with some episodes
- •of epistaxis reported to exceed 5 mins duration supported by clinical
- •judgement, and an ESS >4 during the month prior to Screening.
- •5. Patients with anaemia (haemoglobin levels <11 g/L in men and <10 g/L in
- •women) or parenteral infusion of at least 250 mg of iron in the preceding 6
- •6. Glycosylated haemoglobin (HbA1c) <=6.0% in patients without established
- •diagnosis of diabetes.
- •7. Fasting plasma glucose (FPG) or non-fasting plasma glucose <=5.6 mmol/L in
- •patients without established diagnosis of diabetes mellitus. Note: a single
- •value measured between signing informed consent and entering the treatment
- •period satisfies this criterion (this criterion therefore does not need to be
- •met to enter the observation period)..
- •8. Patients with COVID-19 vaccination(s) (with or without booster) or positive
- •COVID-19 antibody test (spike or nucleocapsid antibody). The last
- •vaccine/booster shot should have been performed at least 14 days before the
- •first dose of the IMP.
- •9. A female patient is eligible to participate if she is neither pregnant nor
- •nursing, and of non-childbearing potential or agrees to use highly effective
- •methods of birth control throughout the Treatment period until 30 days after
- •last IMP administration (for details on contraception refer to Appendix 6).
- •Women are considered post-menopausal and of non-childbearing potential if they
- •meet any of the following: (1) are above age 60; (2) have had 12 months of
- •natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g.,
- •age appropriate, history of vasomotor symptoms) and with follicle stimulating
- •hormone (FSH) >40UI/L (as measured once during or before screening);, or (3)
- •have had surgical bilateral oophorectomy (with or without hysterectomy) or
- •tubal ligation at least 6 weeks before screening. In the case of bilateral
- •oophorectomy alone, female patient is considered of non-childbearing potential
- •only when the reproductive status of the patient has been confirmed by FSH
- •hormone level assessment.
- •10. Male patients must agree to use two (2) reliable and acceptable methods of
- •contraception with their female partner and refrain from donating sperm
- •throughout the Treatment period until 30 days after last IMP administration
- •(for details on contraception refer to Appendix 6).
- •11. The patient has given written informed consent, prior to any study-related
- •procedures that are not part of normal medical care.
- •(as in part 1 except for the HHT severity criteria and COVID rules):
- •1. Completion of Part I of the study, through the End of Study Visit (Visit
- •12), within the previous 8 months.
- •2. All adverse events or serious adverse events occurring
排除标准
- •1. Patients with type 1 diabetes or uncontrolled type II diabetes (insulin or
- •non-insulin dependent).
- •2. History or current diagnosis of ECG abnormalities indicating significant
- •risk of safety for patients participating in the study such as:
- •a. Concomitant clinically relevant cardiac findings, e.g., sustained
- •ventricular tachycardia, and clinically significant second- or third-degree
- •atrioventricular block without a pacemaker;
- •b. History of familial long QT syndrome or known family history of Torsades de
- •c. Resting QTcF >=450 msec (male) or >=460 msec (female);
- •d. Concomitant use of agents known to prolong the QT interval.
- •3. Grade 2 hypertension untreated (systolic blood pressure >=140 mmHg and/or
- •diastolic blood pressure >=90 mmHg). Note that hypertension should be diagnosed
- •according to standard clinical criteria and isolated blood pressure
- •measurements above these values is not exclusionary.
- •4. Active COVID-19 infection confirmed by either polymerase chain reaction
- •(PCR) or rapid antigen test. COVID-19 testing is not required for eligibility
- •unless, in the opinion of the investigator, the patient is displaying symptoms
- •concerning for acute COVID-19 infection.
- •Note: The COVID test does not need to be repeated at Screening in case a test
- •was performed in last 72 hours and lab results/data source shared with the
- •site. In case of positive test, a negative test is needed within 2 weeks for
- •the patient to be eligible. If active infection (confirmed by PCR or rapid
- •antigen test) persists after 2 weeks, patients will not be eligible
- •5. Administration of live attenuated vaccine within 12 weeks of first IMP dose.
- •6. Administration of other vaccines, excluding COVID-19 and live-attenuated
- •vaccines, within 14 days of first IMP dose.
- •7. Patients with active uncontrolled infection or known to be serologically
- •positive for human immunodeficiency virus (HIV), hepatitis B (except after
- •vaccination) or hepatitis C infection.
- •8. Patients with any other severe, progressive, or uncontrolled acute or
- •chronic medical or psychiatric condition or clinical laboratory abnormalities
- •that may increase the risk associated with study participation/treatment or may
- •interfere with interpretation of study results, and, in the Investigator*s
- •opinion, would make the patient inappropriate for entry in this study.
- •9. History of malignancy of any organ system, within the past 5 years,
- •regardless of whether there is evidence of local recurrence or metastases; with
- •the exception of patients with removal of uncomplicated basal cell carcinoma,
- •who may take part in the study.
- •10. Presence of ANY of the following laboratory abnormalities:
- •a. Platelets <=100 × 109/L;
- •b. Absolute neutrophil count <=1.5 × 109/L;
- •c. Substantive renal disease (estimated Glomerular Filtration Rate [eGFR] <=60
- •mL/min/1.73m2 calculated using the MDRD Glomerular Filtration Rate [GFR]
- •d. Abnormal liver function tests such as aspartate transaminase (AST), alanine
- •transaminase (ALT), alkaline phosphatase, or serum bilirubin. The Investigator
- •should be guided by the following criteria: ALT, AST, alkaline phosphatase, or
- •serum bilirubin must NOT exceed 1.5 times upper limit of normal (ULN).
- •11. Any surgical or medical condition which might significantly alter the
- •absorption of the study drug
研究者
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