NL-OMON55249已完成2 期
A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease - IMR-SCD-301
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- IMARA, Inc.,
- 入组人数
- 18
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
- •Each subject must meet all the following criteria to be enrolled in the study:
- •1. Male or female aged *18 to *65 years at the time of informed consent form
- •(ICF) signing.
- •2. Confirmed diagnosis of SCD (HbSS, HbS*0 thalassemia, or HbS*+ thalassemia)
- •in the medical record; if not available, the diagnosis must be confirmed at the
- •site*s local laboratory instead.
- •3. Subjects must have had at least 2 and no more than 12 documented episodes of
- •VOC in the past 12 months at the time of ICF signing and at randomization (Day
- •For study eligibility, VOC is defined as a documented episode of an acute
- •painful crisis (for which there was not an explanation other than VOC) that
- •involved moderate to severe pain lasting for at least 2 hours and at least one
- •of the following:
- •* Use of escalated analgesia (including healthcare professional-instructed use
- •of an analgesic prescription)
- •* A hospital, emergency department, or clinic visit and/or healthcare telephone
- •consultation at the time of occurrence
- •* Diagnosis of acute chest syndrome (ACS) (defined as an acute illness
- •characterized by fever and/or respiratory symptoms, accompanied by a new
- •pulmonary infiltrate on a chest X ray), hepatic sequestration, splenic
- •sequestration, or priapism (in males)
- •4. Hb of >5.5 and <10.5 g/dL; Hb values within 21 days post-transfusion will be
- •5. This inclusion criterion has been removed.
- •6. Subjects receiving HU must have received it continuously for at least 6
- •months prior to signing the ICF, and must have been on a stable dose for at
- •least 3 months prior to signing the ICF, with no anticipated need for dose
- •adjustments during the study including the screening period, in the opinion of
- •the investigator.
- •7. Female subjects must not be pregnant or breastfeeding and be highly unlikely
- •to become pregnant. Male subjects must be unlikely to impregnate a partner.
- •Male or female subjects must meet at least one of the following criteria:
- •* A female subject who is not of reproductive potential is eligible without
- •requiring the use of contraception. A female subject who is not of reproductive
- •potential is defined as one who: (1) has reached natural menopause (defined as
- •12 months of spontaneous amenorrhea without an alternative medical cause, and
- •can be confirmed with serum follicle-stimulating hormone levels in the
- •postmenopausal range as determined by the central laboratory); (2) is 6 weeks
- •post surgical bilateral oophorectomy with or without hysterectomy; or (3) has
- •undergone bilateral tubal ligation. Spontaneous amenorrhea does not include
- •cases for which there is an underlying disease that causes amenorrhea (e.g.,
- •anorexia nervosa).
- •* A female of reproductive potential must have 2 negative pregnancy tests as
- •verified by the investigator prior to starting study therapy. She must agree to
- •ongoing pregnancy testing during the course of the study, at the EOT visit, and
- •at the EOS visit. This applies even if the subject practices true abstinence
- •from heterosexual contact.
- •* A male subject who is not of reproductive potential is eligible without
- •requiring the use of contraception. A male subject who is not of reproductive
- •potential is defined as one who has undergone a successful vasectomy. A
- •successful vasectomy is defined as (1) microscopic documentation of azoospermia
排除标准
- •Subjects who meet any of the following criteria will be excluded from the study:
- •1. Hospital discharge for sickle cell crisis or other vaso occlusive event
- •within the 4 days prior to randomization (Day 1).
- •2. Subjects participating in a chronic/prophylactic RBC transfusion program
- •(i.e., regularly scheduled RBC transfusions); any transfusions within 21 days
- •of screening or baseline Hb measurements.
- •3. Subjects with HbF >25% at screening.
- •4. Subjects with known active hepatitis A, hepatitis B, or hepatitis C, with
- •active or acute event of malaria, or who are known to be positive for human
- •immunodeficiency virus (HIV).
- •5. For female subjects of childbearing potential, a positive serum human
- •chorionic gonadotropin (hCG) test (screening) or a positive urine hCG test at
- •randomization (Day 1).
- •6. Significant kidney disease as indicated by, for example, estimated
- •glomerular filtration rate (eGFR) <45 mL/min as calculated by the equation from
- •the Modification of Diet in Renal Disease (MDRD) Study using creatinine, age,
- •sex, and ethnicity (modified MDRD formula).
- •7. Alanine aminotransferase or aspartate aminotransferase >3× the upper limit
- •8. Body mass index (BMI) <17.0 kg/m2 or >35 kg/m2; or total body weight <45 kg.
- •9. Current or history of malignancies (solid tumors and hematological
- •malignancies), unless the subject has been free of the disease (including
- •completion of any active or adjuvant treatment for prior malignancy) for *5
- •years. However, subjects with the following history of/concurrent conditions
- •are allowed if, in the opinion of the investigator, the condition has been
- •adequately diagnosed and is determined to be clinically in remission, and the
- •subject*s participation in the study would not represent a safety concern:
- •a. Basal or squamous cell carcinoma of the skin
- •b. Carcinoma in situ of the cervix
- •c. Carcinoma in situ of the breast
- •d. Incidental histologic finding of prostate cancer (T1a or T1b using the
- •tumor, nodes, metastasis clinical staging system)
- •10. History of a clinically significant allergic reaction or hypersensitivity,
- •as judged by the investigator, to any drug or any component of the study drug
- •formulations used in the study (see the Investigator*s Brochure).
- •11. History of unstable or deteriorating cardiac or pulmonary disease within 6
- •months before signing the ICF, including but not limited to the following:
- •a. Unstable angina pectoris or myocardial infarction or elective coronary
- •intervention
- •b. Congestive heart failure requiring hospitalization
- •c. Uncontrolled clinically significant arrhythmias
- •12. Any condition affecting drug absorption, such as major surgery involving
- •the stomach or small intestine (prior cholecystectomy is acceptable).
- •13. On ECG testing at ICF signing and/or randomization (Day 1), a corrected QT
- •interval, Fridericia*s formula (QTcF) >450 ms in men and >470 ms in women on 2
- •or more of the triplicate ECGs, or the presence of clinically significant ECG
- •abnormalities as determined by the investigator.
- •14. Major surgery within 8 weeks or minor surgery within 2 weeks of
- •randomization (Day 1).
- •15. Stroke requiring medical intervention within 24 weeks prior to
- •randomization (Day 1).
- 另有 2 项未显示
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