EUCTR2022-002395-36-GR招募中1 期
An open-label, dose-escalation, dose-finding, and proof-of-concept trial of SP-420 in subjects with transfusion-dependent ß-thalassemia
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 90
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
- 性别
- All
入选标准
- •1. Women and men aged =18 years
- •2. Transfusion-dependent ß-thalassemia including HbE/ß-thalassemia requiring iron chelation therapy (ß-thalassemia with mutation and/or multiplication of a-globin is allowed)
- •3. On a stable dose of iron chelation for at least 4 weeks prior to screening
- •4. Weight =35 kg at screening
- •5. Willing to discontinue current iron chelation therapy 7 days (± 3 days) prior to the first dose of SP-420 and for the duration of the trial
- •6. Transfusion iron overload defined as LIC =5 and =20 mg/g dw on the R2-MRI obtained within 2 weeks prior to baseline
- •7. Subject has been treated and followed for at least the past 6 months in a specialised centre that maintained detailed medical records, including transfusion and iron chelation histories
- •8. Willingness to participate and signing the informed consent form
- •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 85
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •1. ß-thalassemia with the structural Hb variants HbS and HbC
- •2. Cardiac MRI-T2 score <10 msec obtained within 2 weeks prior to baseline
- •3. S-ferritin <500 or >4000 ng/mL
- •4. Current malignancy with the exceptions of localised basal cell or squamous cell skin cancer or localised prostate cancer or is receiving immunotherapy, chemotherapy, or radiation therapy for a malignancy
- •5. Current myelodysplastic syndrome
- •6. Current biliary disorder
- •7. ALAT >4 times the upper limit of normal, decompensated cirrhosis, or ascites at screening
- •8. Past or ongoing history of clinically significant kidney disease
- •9. Creatinine greater than the upper limit of normal at screening
- •10. Estimated glomerular filtration rate eGFR <60 mL/min/1.73 m2
- •11. Urine protein to creatinine ratio >0.5 mg/mg at screening
- •12. Heart failure grade II, III and IV by NYHA
- •13. Left ventricular ejection fraction (LVEF) on MRI <56 % (echocardiography allowed if MRI not available)
- •14. A QTcF >450 ms, 2nd or 3rd degree atrioventricular block, complete left bundle branch block, or the presence of clinically significant abnormalities as determined by the Investigator at screening
- •15. Hypertransfused defined as more than 6 units/month in average for the last 6 months prior to screening
- •16. Ongoing symptoms of neuropathy, including peripheral sensory neuropathy, peripheral motor neuropathy, or paresthesia at screening
- •17. Platelet count <100×109/L at screening
- •18. History of hypersensitivity to an iron chelator (investigational or marketed) or excipients
- •19. Documented history of non-compliance to chelation therapy within past 2 years
- •20. Received another investigational drug within 30 days or investigational antibody within 90 days before screening
- •21. Treatment with prohibited medication: iron, aluminium containing antacid therapies, systemic corticosteroids (topical and pulmonary corticosteroids are allowed), oral bisphosphonates, chronic use of high dose NSAIDs (as needed and low dose acetylsalicylic acid are allowed), drugs with known renal toxicity, drugs with known QTc prolongation, potent UGT inducers (e.g. rifampicin, phenytoin, phenobarbital, ritonavir) within 7 days prior to baseline
- •22. Initiation of treatment with luspatercept within 6 months prior to screening (luspatercept is allowed if initiated and dose is stable at least 6 months prior to screening)
- •23. Subject unable to undergo trial assessments including MRI, e.g. who are claustrophobic to MRI, have a cardiac pacemaker, ferromagnetic metal implants other than those approved as safe for use in MR scanners (e.g. some types of aneurysm clips, and shrapnel), and subjects who are obese (exceeding the equipment limits)
- •24. Pregnant or nursing women. In order to avoid pregnancy, women of childbearing potential (i.e., fertile, following menarche and until
- •becoming post-menopausal unless permanently sterile. Permanent
- •sterilisation methods include hysterectomy, bilateral salpingectomy, and
- •bilateral oophorectomy) have to use highly efficient contraception (e.g.,
- •combined [estrogen and progestogen containing] hormonal
- •contraception associated with inhibition of ovulation [oral, intravaginal,
- •or transdermal], progestogen-only hormonal contraception associated
- •with inhibition of ovulation [oral, injectable, or implantable],
- •intrauterine device, intrauterine hormone-releasing system, bilateral
- •tubal occlusion, or vasectomised partner) during the whole trial period
- •and 4 weeks post-dosing. A sterile sole partner (i.e., perman
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