EUCTR2019-001899-11-GB进行中（未招募）1 期
A Randomized, Placebo-controlled, Phase 2 Study to Evaluate the Safety and Pharmacodynamics of Once-daily Oral IW-1701 in Patients with Stable Sickle Cell Disease - Strong SCD
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 88
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. Patient has provided written consent before any study-specific procedures are performed; for patients <18 years of age, parental permission and child assent will be obtained.
- •2. Patient is ambulatory male or female 16 to 70 years of age at the Screening Visit.
- •3. Patient has sickle cell disease (SCD), including HbSS, HbSC, HbSß0-thalassemia, or HbSß+-thalassemia, documented in their medical history by hemoglobin electrophoresis or genotyping.
- •4. If receiving hydroxyurea (HU), erythropoietin, L-glutamine, or prophylactic oral antibiotics, patient has had no change in regimen(s) (ie, drug and dose) for at least 8 weeks before the Randomization Visit and has no plans to change regimen(s) during the study. If receiving HU, patient must have been prescribed HU for at least 6 months prior to the Randomization Visit. (Note: Patient is not required to be taking medication[s] for SCD.)
- •Should the patient begin any new chronic treatment/therapy for SCD during the study or alter any current treatment/therapy for SCD during the study, the Sponsor Medical Monitor must be informed.
- •5. If receiving chronic medication(s) for hypertension, patient has had no change in regimen(s) (ie, drug and dose) for at least 8 weeks before the Randomization Visit and has no plans to change regimen(s) during the study.
- •6. Per medical history and/or patient recall, patient has had at least 1 and no more than 10 sickle cell-related pain crises in the 12 months before the Screening Visit and none occurring in the 4 weeks before the Randomization Visit.
- •For assessing study eligibility, an SCD-related pain crisis is defined as an acute episode of new-onset pain that lasts =2 hours with no medically determined cause other than a vaso-occlusive event and requires presentation to a medical facility (eg, acute care setting,
- •Emergency Department, urgent care clinic) and treatment with oral or parenteral opioids, parenteral nonsteroidal anti-inflammatory drugs, or other analgesics, prescribed by a healthcare provider.
- •7. Patient has clinically acceptable (per Investigator discretion) electrocardiogram (ECG) with QT interval corrected using Fridericia’s formula (QTcF interval) <500 ms at the Screening Visit.
- •8. Patient has seated systolic blood pressure (BP) from 90 to 160 mmHg at the Screening Visit. (Note: BP for eligibility will be the average of 3 measurements obtained with an appropriately sized cuff at 2-minute intervals after the patient has been sitting quietly for =5 minutes.)
- •9. Female patient must be postmenopausal (no menses for =12 consecutive months) or surgically sterile (ie, bilateral oophorectomy, hysterectomy, or tubal sterilization [tie, clip, band, or burn]); must agree to completely abstain from heterosexual intercourse; or, if heterosexually active, must agree to use 1 of the following methods of birth control from the date she signs the informed consent form (ICF) until 90 days after the final dose of study drug:
- •- Progesterone implant and/or an intrauterine device (IUD)
- •- Combination of 2 highly effective birth control methods (eg, diaphragm with spermicide plus a condom, condom with spermicide plus a diaphragm or cervical cap, hormonal contraceptive [eg, oral and transdermal patch] plus a barrier method, or partner with vasectomy [conducted =60 days before the Screening Visit or confirmed via sperm analysis] plus a hormone or barrier method).
- •10. Male patient must be surgically sterile by vasectomy (conducted =60 days before the Screening Visit or con
排除标准
- •1. Patient requires a program of prescheduled, regularly administered chronic blood transfusion therapy, has received a transfusion in the 8 weeks before the Randomization Visit, and/or is scheduled to receive a transfusion during the study.
- •2. Patient has been hospitalized for an SCD-related complication in the 4 weeks before the Randomization Visit.
- •3. Patient is planning to undergo major surgery during the trial or has undergone surgery within 4 weeks of the Screening Visit, other than minor dermatologic procedures.
- •4. Patient has used oral or parenteral corticosteroids in the 8 weeks before the Randomization visit. Note: Transient use for =2 days may be acceptable; consult the Medical Monitor for confirmation.
- •5. Patient has taken opioid(s) >200 morphine mg equivalent/day within the 4 weeks before the Randomization Visit.
- •6. Patient is taking aspirin =325 mg daily, any P2Y12 inhibitor, any anticoagulant medication, specific inhibitors of phosphodiesterase 5 (PDE5), nonspecific inhibitors of PDE5 (including dipyridamole and theophylline), any supplements for the treatment of erectile dysfunction, riociguat, or nitrates or nitric oxide donors in any form.
- •Patient is taking moderate or strong cytochrome P450 3A (CYP3A) inhibitors or inducers.
- •These medications are prohibited from the Run-in Visit (which may occur from Day -14 to Day -17) through the duration of the study.
- •See Appendix 1 of the study protocol for a list of prohibited medications, supplements, and foods.
- •7. Patient uses or requires the use of fentanyl.
- •8. Patient has any of the following clinical laboratory values at the Screening Visit:
- •a. Hemoglobin =6 g/dL
- •b. Platelets =100×109/L
- •c. Absolute neutrophils =1.5×109/L
- •d. Alanine aminotransferase >1.5×the upper limit of normal (ULN) as defined by laboratory
- •e. Direct bilirubin >3×ULN as defined by laboratory
- •f. Creatinine clearance <30 mL/minute/1.73 m2 by the Modification of Diet in Renal Disease equation
- •g. Prothrombin time and/or aPTT >1.5×ULN and considered clinically significant by the Investigator, and/or INR>1.5
- •9. Patient has oxygen saturation =90% by pulse oximetry on room air at the Screening Visit and/or at the Randomization Visit.
- •10. Patient has had inpatient hospitalization for alcoholism or drug addiction in the 12 months before the Screening Visit or is positive for any the following at the Screening Visit unless legally prescribed: Amphetamines, Benzodiazepines, Opiates, Propoxyphene, Barbiturates, Cocaine, Phencyclidine
- •11. Patient has major concurrent illness or medical condition that in the opinion of the Investigator would preclude participation in a clinical study, including but not limited to:
- •a. Uncontrolled significant cardiovascular disease or clinically significant cardiac arrhythmia as assessed by the Investigator
- •b. Serious event such as stroke or transient ischemic attack =12 months before Randomization, or deep venous thrombosis or pulmonary embolism =6 months before Randomization
- •c. History of platelet dysfunction, hemophilia, von Willebrand disease, coagulation disorder, other bleeding diathesis condition(s), or significant, nontraumatic bleeding episodes, such as from a gastrointestinal source, even if patient has normal complete blood count, prothrombin time, and activated partial thromboplastin time at the screening Visit
- •d. Known severe central nervous system vasculopathy (eg, Moyamoya disease, arteriovenous malformations)
- •e. Interstitial lung disease requiring continuous oxygen
研究者
相似试验
已完成
2 期
A Randomized, Placebo-controlled, Phase 2 Study to Evaluate the Safety and Pharmacodynamics of Once-daily Oral IW-1701 in Patients with Stable Sickle Cell DiseaseStable sickle cell diseaseSickle Cell Disease (SCD)LBCTR2019091283Cyclerion Therapeutics, Inc.88
进行中（未招募）
1 期
A Study to Investigate the Safety, Tolerability, and Efficacy of TAK-079 in Participants With Generalized Myasthenia GravisMedDRA version: 21.1Level: PTClassification code 10028417Term: Myasthenia gravisSystem Organ Class: 10029205 - Nervous system disordersGeneralized Myasthenia GravisEUCTR2019-003383-47-ITMILLENNIUM PHARMACEUTICALS, INC.36
进行中（未招募）
不适用
A Phase II Randomized, Placebo-Controlled Study to Evaluate the Safety and Tolerability of MK-7009 When Administered Concomitantly With Pegylated Interferon and Ribavirin in Patients With Chronic Hepatitis C InfectioChronic hepatitis C infectionMedDRA version: 9.1Level: LLTClassification code 10019751Term: Hepatitis C virusEUCTR2008-000150-12-SEMerck Sharp & Dohme (Sweden) AB300
进行中（未招募）
不适用
A Phase II Randomized, Placebo-Controlled Study to Evaluate the Safety and Efficacy of MK-7009 Administered Concomitantly With Pegylated-Interferon and Ribavirin for 28 Days in Treatment-Naïve Patients With Chronic Hepatitis C Infection.EUCTR2008-000149-72-DEMerck & Co., Inc.176
进行中（未招募）
1 期
A Phase II Randomized, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of 4 Different Regimens of MK-7009 When Administered Concomitantly with Pegylated Interferon and Ribavirin in Treatment-Experienced Patients with Chronic Genotype 1 Hepatitis C Virus Infection. - Add-on study of MK-7009 to PEG interferon and Ribavirin.Chronic hepatitis C infectionMedDRA version: 9.1Level: LLTClassification code 10019751Term: Hepatitis C virusEUCTR2008-000150-12-BEMerck Sharp & Dohme (Europe) Inc.160