NCT04741542终止1 期
Phase 1, Open-Label, Dose Escalation Study to Assess the Safety of SP-420 in the Treatment of Transfusional Iron Overload
The University of Texas Health Science Center at San Antonio1 个研究点 分布在 1 个国家目标入组 2 人开始时间: 2021年3月9日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 2
- 试验地点
- 1
- 主要终点
- Completion at original dose
研究概览
简要总结
This study enrolls patients with myelodysplastic syndrome (MDS) and myelofibrosis (MFS), with transfusional iron overload and treats them with the investigational iron chelator, SP-420. SP-420 may be better tolerated and safer than commercially available iron chelators. Iron chelation therapy (ICT) has been shown to improve outcomes in iron overload, but adherence is poor due to problems related to ease of administration, tolerability, and safety.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of MDS or MF with transfusional iron overload
- •Patients with MDS, will include only those with MDS Revised international prognostic scoring system (IPSS-R) risk group of intermediate, high, or very high.
- •Patients with MF, will include only those with Dynamic International Prognostic Scoring System-Plus (DIPSS=Plus) risk category of intermediate-1, intermediate-2, and high risk.
- •Patients with sickle cell disease and transfusional iron overload
- •Not appropriate for other iron chelation therapy, per physician
- •Received 10 or more units of packed red blood cells in the preceding 24 months and remains red cell transfusion dependent
- •ALT ≤ 3 times the upper limit of the normal range
- •Estimate glomerular filtration rate calculated using Cockroft Gault of ≥ 60 mL/min/1.73m2
- •Serum ferritin ≥1000 ng/ml
- •Willing to comply with all study procedures and be available for the duration of the study
- •Able to take oral medication and be willing to adhere to study medication for 28 days
- •Female patient must be post-menopausal (no menses for > 12 consecutive months) or surgically sterile (i.e., bilateral oophorectomy, hysterectomy, or tubal sterilization; must agree to completely abstain for heterosexual intercourse; or, if sexually active, must agree to use 1 of the following methods for birth control from the date she signs the consent form until 30 days after final dose of the study drug.
- •Progesterone implant
- •Intrauterine device
- •Combination of 2 highly effective birth control methods (e.g., diaphragm/or cervical cap with spermicide plus a condom, hormonal contraception plus a barrier method, partner with vasectomy conducted >60 days before screening visit plus a hormone or barrier method
- •Male patients must agree to use 1 of the following methods for birth control from the date he signs the consent form until 30 days after final dose of the study drug: be surgically sterile by vasectomy conducted > 60 days before screening visit plus use a barrier method, or, must agree to completely abstain from heterosexual intercourse, or must agree to use a combination of 2 highly effective birth control methods (e.g., diaphragm/or cervical cap with spermicide plus a condom, hormonal contraception plus a barrier method), or have a post-menopausal partner plus barrier method.
排除标准
- •History of kidney disease including the renal Fanconi syndrome
- •Proteinuria on urine dipstick greater than trace positive
- •Pregnant, intending to become pregnant during the study, or breastfeeding
- •Receiving another investigational drug within 30 days or 3 half-lives of the discontinued investigational agent, whichever is greater, of signing consent
- •History of significant hepatic impairment, defined by Child-Pugh class C
- •Active hepatitis B or C disease, evidenced by positive viral PCR
- •Symptomatic heart failure
- •Receiving active cytotoxic chemotherapy or radiation therapy for a second malignancy (hormonal therapy or topical therapy for squamous cell/basal cell cutaneous tumors are allowed). Treatment of the underlying hematologic malignancy with azacytidine, decitabine, venetoclax, lenalidomide, or ruxolitinib is permitted. Treatment with the supportive care agents luspatercept or erythropoietin agonists is permitted.
- •Concurrent treatment with Exjade/Jadenu (deferasirox), Desferal (deferoxamine), or Ferriprox (deferiprone) are not permitted. Patients are allowed to stop these chelators and participate in this trial 14 days after discontinuation of the other chelator.
研究组 & 干预措施
Group A
Experimental
Study subjects will receive a 14mg/kg starting dose of SP-420 three times a week
干预措施: SP-420 (Drug)
Group B
Experimental
Study subjects will receive a 28mg/kg starting dose of SP-420 three times a week
干预措施: SP-420 (Drug)
Group C
Experimental
Study subjects will receive a 42mg/kg starting dose of SP-420 three times a week
干预措施: SP-420 (Drug)
Group D
Experimental
Study subjects will receive a 56mg/kg starting dose of SP-420 three times a week
干预措施: SP-420 (Drug)
结局指标
主要结局
Completion at original dose
时间窗: 28 Days
Number of subjects that completed the study at the original starting dose of that group
Number of adverse events
时间窗: 28 Days
Count of adverse events induced by SP-420
次要结局
未报告次要终点
研究者
研究点 (1)
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