NCT06286046尚未招募2 期
A Phase 2, Open-label, Multicenter Study of Mitapivat in Subjects With Sickle Cell Disease and Nephropathy (RESIST)
适应症
干预措施
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 入组人数
- 40
- 试验地点
- 7
- 主要终点
- Percentage of Participants With Albumin Creatinine Ratio (ACR) Response
研究概览
简要总结
The primary purpose of this study is to evaluate the effect of mitapivat on albumin creatinine ratio (ACR) response in participants with sickle cell disease (SCD) and nephropathy.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 65+ years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age of 16 years or older (except in France where participants must be aged 18 years or older);
- •Females must be post-menarche;
- •Documented diagnosis of sickle cell disease (Homozygosity for hemoglobin S [HbSS] or Hemoglobin S/Beta 0 [HbS/β0]-thalassemia);
- •Hemoglobin concentration ≥ 5.5 and ≤ 10.5 grams per deciliter (g/dL) during the Screening Period. If more than one measurement is collected during the Screening Period, the average must be ≥ 5.5 and ≤ 10.5 g/dL;
- •If taking hydroxyurea, the dose of hydroxyurea must have been stable for at least 90 days before Study Day 1 with no planned dose adjustment during the study and no sign of hematologic toxicity;
- •Discontinuation of hydroxyurea requires a 90-day washout before providing informed consent/assent;
- •Two urine ACR results collected during the Screening Period, both of which must be ≥ 100 and < 2000 milligrams per gram (mg/g). One ACR result can be from an untimed urine sample collected as part of a clinic visit. The other ACR result must be from a urine sample that is the first (or second) morning void on another day;
- •One ACR result > 100 mg/g within 24 weeks before providing informed consent/assent;
- •If taking Angiotensin-converting enzyme (ACE) inhibitor or Angiotensin receptor blockers (ARB) therapy, must have been on stable dose for at least 90 days before providing informed consent/assent with no planned dose adjustment during the study;
- •Women of childbearing potential (WOCBP) must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle or agree to use 2 forms of contraception, one of which must be considered highly effective, from the time of providing informed consent/assent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can include an acceptable barrier method.
排除标准
- •Pregnant, breastfeeding, or parturient;
- •Currently receiving regularly scheduled red RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or vaso-occlusive crisis (VOC) is permitted;
- •Have received an RBC transfusion within 60 days before providing informed consent/assent or during the Screening Period;
- •Hospitalized within 14 days before providing informed consent/assent or during the Screening Period either for a sickle cell disease pain crisis (SCPC) or other vaso-occlusive event;
- •More than 10 SCPCs in the 52 weeks before providing informed consent/assent;
- •History of stroke or meeting criteria for primary stroke prophylaxis (history of 2 transcranial Doppler [TCD] measurements ≥ 200 centimeters per second (cm/s) by nonimaging TCD or ≥ 185 cm/s by imaging TCD) at any time;
- •Renal dysfunction as defined by an eGFR < 45 milliliters per minute (mL/min)/1.73 meters per square (m^2) by the Chronic Kidney Disease Epidemiology Collaboration creatinine equation (National Kidney Foundation, 2021b) at Screening;
- •History of renal disease due to another disorder (e.g., diabetes, hypertension, primary focal segmental glomerulosclerosis, autoimmune) unrelated to SCD at any time;
- •Evidence of acute kidney injury (in the opinion of the Investigator) within 4 weeks before informed consent/assent or during Screening Period.
- •Currently undergoing renal replacement therapy (i.e., hemodialysis, peritoneal dialysis, hemofiltration, kidney transplantation);
- •History of kidney transplant at any time;
- •Currently receiving treatment with a disease-modifying therapy for SCD (eg, voxelotor, crizanlizumab, L-glutamine), except for hydroxyurea. The last dose of such therapies must have been administered at least 90 days before starting study drug;
- •Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before starting study drug;
研究组 & 干预措施
Mitapivat 100 mg
Experimental
Participants will receive mitapivat 100 milligrams (mg) tablet, orally, twice daily (BID) for up to 24 months.
干预措施: Mitapivat (Drug)
结局指标
主要结局
Percentage of Participants With Albumin Creatinine Ratio (ACR) Response
时间窗: Baseline up to 6 months
The ACR response is defined as a decrease of 30% or more in ACR from the baseline to Month 6.
次要结局
- Change From Baseline in Cystatin C and Creatinine-based Estimated Glomerular Filtration Rate (eGFRcr-cys)(Baseline up to 25 months)
- Change From Baseline in Albumin Creatinine Ratio (ACR)(Baseline up to 25 months)
- Percentage of Participants With Stable ACR(Baseline up to 6 months)
- Annualized Rate of Emergency Room (ER) Visits(Up to 24 months)
- Annualized Rate of Days of Hospitalizations(Up to 24 months)
- Type, Frequency, Severity, and Relationship to Study Drug of Adverse Events (AEs) and Serious Adverse Events (SAEs)(Up to 25 months)
研究者
Director, Scientific Communications
Scientific
Agios Pharmaceuticals Inc.
研究点 (7)
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