EUCTR2019-003505-96-GR进行中（未招募）1 期
A Phase 2a, Randomized, Open-Label Study to Evaluate theEfficacy, Safety, Tolerability, Pharmacokinetics andPharmacodynamics of ISIS 702843 Administered Subcutaneously toPatients with Non-Transfusion Dependent ß-ThalassemiaIntermedia
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 29
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 must have given written informed consent and be able to comply with all study
- •requirements
- •2. Aged 18-65 years old, inclusive, at the time of informed consent
- •3. Clinical diagnosis of Beta-Thalassemia Intermedia with genotypic confirmation of beta-globin gene mutations including but not limited to Hemoglobin E (HbE)/beta-thalassemia
- •4. Patient must be non-transfusion dependent as defined by: No more than 6 transfusions in the past 12-month period, and no transfusions in the 8-week period prior to Day 1
- •5. Mean Hb within the range 6.0–10.0 g/dL, inclusive, with this mean based on all Hb measurements taken in the Screening Period that are at least 6 weeks after the most recent transfusion for that patient. This mean must be based on at least two Hb measurements.
- •6. LIC within the range of 3.0–20.0 mg Fe/g dry weight, inclusive
- •7. Chelators will be permitted provided the patient has been on a stable dose for at least
- •3 months prior to Day 1, with LIC > 5.0 mg Fe/g dry weight and serum ferritin
- •> 300 ng/mL
- •8. Females must be non-pregnant and non-lactating, one of the following: (i) surgically sterile (e.g., tubal occlusion, hysterectomy, bilateral salpingectomy, bilateral oophorectomy), (ii) postmenopausal (defined as 12 months of spontaneous amenorrhea without an alternative medical cause and follicle stimulating hormone [FSH] levels in the
- •postmenopausal range for the laboratory involved), (iii) abstinent*, or (iv) if engaged in
- •sexual relations of child-bearing potential, the patient must be using a highly effective contraceptive method from the time of signing the informed consent form until at least 13 weeks after the last dose of ISIS 702843.
- •Males must be one of the following: (i) surgically sterile, (ii) abstinent*, or (iii) if engaged in sexual relations with a female of child-bearing potential, the patient must be using a highly effective contraceptive method from the time of signing the
- •informed consent form until at least 13 weeks after the last dose of ISIS 702843.
- •* Abstinence is only acceptable as true abstinence, i.e., when this is in line with the
- •preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar,
- •ovulation, symptothermal, post-ovulation methods), declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.
- •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 32
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •1. Genotypic confirmation of either a-globin gene triplication or sickle hemoglobin (HbS)/beta-thalassemia, as determined by genetic assessment of blood-related disorders
- •2. Clinically significant abnormalities in medical history or physical examination, which at the discretion of the PI will pose significant additional risk to the patient in participating in the study
- •3. Clinically significant abnormalities in Screening laboratory values that would render a patient unsuitable for inclusion, at the discretion of the PI
- •4. Current use of iron-chelation therapy if LIC is 3.0–5.0 mg Fe/g dry weight, inclusive, or if serum ferritin = 300 ng/mL
- •5. Symptomatic splenomegaly, including abdominal pain or organ obstruction, or evidence of hypersplenism, such as low white blood cell (WBC) count and/or low platelets
- •6. Platelet count < LLN, or platelet count > 1,000 x 109/L
- •7. Significant concurrent/recent coagulopathy; history of non-traumatic significant bleeding; history of immune thrombocytopenic purpura (ITP); current use of SC anti-coagulants; history of thrombotic events, including stroke or DVT
- •8. Clinically significant renal dysfunction which at the discretion of the PI will pose significant additional risk to the patient in participating in the study
- •9. eGFR < 45 mL/min/1.73 m2, using CKD-EPI
- •10. Clinically significant liver function test (LFT) abnormalities
- •11. Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) > 3.0 × ULN
- •12. Historical diagnosis of cirrhosis, or current signs and symptoms of cirrhosis
- •13. Fasting blood glucose > 2.0 × ULN
- •14. Significant PHT defined as tricuspid regurgitation > 3.0 meters per second (m/s) on echocardiography and/or requiring treatment
- •15. Uncontrolled hypertension (which for this protocol is considered > 140 mm Hg systolic or > 90 mm Hg diastolic)
- •16. Heart failure class 3 or higher (New York Heart Association, NYHA)
- •17. Ejection fraction < 50% by echocardiogram, multigated acquisition (MUGA), or cardiac magnetic resonance imaging (MRI)
- •18. Patients unable to have MRI performed, for example, because of a pacemaker or implantable cardioverter-defibrillator. (MRI is being used to measure LIC.)
- •19. Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Day 1
- •20. Known history of or positive test for human immunodeficiency virus (HIV), hepatitis C (unless treatment has caused the patient to test negative for hepatitis C), or chronic hepatitis B
- •21. Unwillingness to comply with study procedures, including follow-up, as specified by this protocol, or unwillingness to cooperate fully with the Investigator
- •22. Recent introduction of hydroxyurea (6 months prior to Day 1)
- •23. Treatment with or exposure to another investigational drug, biological agent, ASO, small interfering ribonucleic acid (siRNA), or device within 1 month of Screening, or 5 half-lives of investigational agent, whichever is longer; or:
- •- Treatment with or exposure to sotatercept (ACE-011), luspatercept (ACE-536), or ruxolitinib within 4 months of Screening
- •- Treatment with or exposure to hematopoietic stimulating agents (e.g., EPOs) or any hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHI) (e.g., roxadustat,
- •vadadustat, daprodustat, molidustat, desidustat) within 8 weeks of Day 1
- •- Prior bone marrow transplant, stem cell transplant, or gene therapy
- •24. Regular use of alcohol within 6 months prior to Screening (> 7 drinks/wk for females, > 14 drinks/wk for ma
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