NL-OMON54921已完成2 期
A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia - IMR-BTL-201
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
- 15
研究概览
简要总结
Trial ended prematurely
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •Subjects must meet all of the following inclusion criteria to be eligible for
- •1. Subjects must understand and voluntarily provide informed consent and sign
- •an informed consent form (ICF) prior to any study-related
- •assessments/procedures being conducted. Although RBC transfusions and
- •associated Hb laboratory measurements prior to Screening are not study related,
- •the ICF will specifically request subject consent to collect these data.
- •2. Subjects must be >=18 to <=65 years of age at the time of signing the ICF.
- •3. Subjects must have documented diagnosis of β-thalassemia or HbE/β-
- •thalassemia in their medical history. Concomitant alpha gene deletion,
- •duplication, or triplication is allowed.
- •4. For TDT subjects only: Subjects must be regularly transfused, defined as >3
- •to 10 pRBC units (one RBC unit refers to one bag of packed RBCs) in the 12
- •weeks prior to the Baseline (Day 1) visit and no transfusion-free period for
- •>35 days during that period.
- •For NTDT subjects only: Subjects must be transfusion independent, defined as 0
- •to <=3 units1 of pRBCs received during the 12-week period prior to the Baseline
- •(Day 1) visit, must not be on a regular transfusion program, must be RBC
- •transfusion-free for at least >= 4 weeks prior to randomization, and must not be
- •scheduled to start a regular hematopoietic stem cell transplantation within 9
- •5. Subjects must have documentation of the dates of transfusion events and the
- •number of pRBC units per event within the 12 weeks prior to the Baseline (Day
- •6. Subjects must be willing and able to complete all study assessments and
- •procedures, and to communicate effectively with the investigator and site staff.
- •7. Subjects must have Eastern Cooperative Oncology Group (ECOG) performance
- •score of 0 to 1 (Appendix 1).
- •8. 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 end of
- •treatment visit, and at the end of study 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 succe
排除标准
- •Subjects meeting any of the following criteria must be excluded from the study:
- •1. Any significant medical condition, laboratory abnormality, or psychiatric
- •illness that would prevent the subject from participating in the study,
- •including the presence of laboratory abnormalities that may place the subject
- •at unacceptable risk if he/she were to participate in the study.
- •2. Any situation or condition that confounds the ability to interpret data from
- •the study (e.g., subjects also receiving RBC transfusions at centers not able
- •to obtain laboratory samples for central processing).
- •3. Diagnosis of a-thalassemia (e.g., hemoglobin H [HbH]) or hemoglobin S (HbS)/
- •β thalassemia.
- •4. Body mass index (BMI) <17.0 kg/m2 or a total body weight <45 kg; or BMI >35
- •5. 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).
- •6. Stroke requiring medical intervention <=24 weeks prior to randomization.
- •7. Subjects taking direct acting oral anti-coagulants (DOACs) apixaban,
- •dabigatran, rivaroxaban, edoxaban, or ticagrelor, or taking warfarin, are
- •excluded due to the possibility of a cytochrome P450 (CYP)3A-mediated drug
- •interaction, unless they stopped the treatment at least 28 days prior to
- •randomization (Day 1); other oral anti-coagulants and anti-platelet drugs are
- •permitted. Anti-coagulant therapies for prophylaxis of venous thromboembolism,
- •including pulmonary emboli including when undergoing surgery or high risk
- •procedures, are allowed if low molecular weight heparins are used in the peri
- •operative period. Aspirin use (<100 mg per day) is allowed before and during
- •8. Participated in another clinical study of an investigational agent (or
- •medical device) within 30 days or 5 half-lives of date of informed consent,
- •whichever is longer, or is currently participating in another study of an
- •investigational agent (or medical device).
- •9. Platelet count >1000 × 109/L.
- •10. For subjects on iron chelation therapy (ICT) at the time of ICF signing,
- •initiation of ICT less than 24 weeks before the predicted randomization date.
- •ICT can be initiated at any time during treatment and should be used according
- •to the label.
- •11. Subjects who have had treatment with erythropoietin-stimulating agents <=24
- •weeks prior to randomization.
- •12. Uncontrolled hypertension as defined by systolic BP >=160 mm Hg or diastolic
- •BP >=100 mm Hg, medical intervention indicated, and more than one drug or more
- •intensive therapy than previously used indicated.
- •13. Poorly controlled diabetes mellitus, in the opinion of the investigator,
- •for example 1) Hb A1c >9.0% within 12 weeks prior to randomization (in the
- •medical history); 2) short-term hyperglycemia leading to hyperosmolar or
- •ketoacidotic crisis; and/or 3) history of diabetic cardiovascular complications.
- •14. Subjects who have major organ damage, including:
- •a. Liver disease with ALT or AST >3× ULN; direct bilirubin >3× ULN with
- •proportion of direct/total bilirubin >0.3; or history/evidence of cirrhosis,
- •liver transplant, or presence of clinically significant mass/tumor.
- •b. Heart disease, heart failure as classified by the New York Heart Association
- •classification 3 or higher, or significant arrhythmia requiring treatm
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