CTRI/2024/11/077511进行中（未招募）2/3 期
An Adaptive, Randomized, Placebo-controlled, Double -blind, Multicenter Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease.
试验速览
- 阶段
- 2/3 期
- 状态
- 进行中（未招募）
- 入组人数
- 450
- 试验地点
- 6
- 主要终点
- Hemoglobin response rate at Week 24 (increase of greater than 1 g/dL [grater than 10 g/L] from baseline) during the blinded treatment period
研究概览
简要总结
This clinical trial is a Phase 2/3 study that will evaluate the efficacy and safety of FT-4202 and test how well FT-4202 works compared to placebo to improve the amount of hemoglobin in the blood and to reduce the number of vaso-occlusive crises (times when the blood vessels become blocked and cause pain).
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 盲法
- Participant, Investigator, Outcome Assessor and Date-entry Operator Blinded
入排标准
- 年龄范围
- 12.00 Year(s) 至 65.00 Year(s)（—）
- 性别
- All
入选标准
- •Patient has provided documented informed consent or assent (the informed consent form [ICF] must be reviewed and signed by each patient; in the case of adolescent patients, both the consent of the patient’s legal representative or legal guardian, and the patient’s assent must be obtained).
- •Patient has a confirmed diagnosis of sickle cell disease Documentation of SCD genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing. Molecular genotyping is not required. SCD genotype may be determined from the results of Hb electrophoresis, HPLC, or similar testing. Note that Hb electrophoresis is performed by the central laboratory at Screening.
- •Patient has 2–15 episodes of documented VOC (defined in protocol Section 8.2.2) within the 12 months prior to screening. Documentation must exist in the patient’s medical record prior to screening. Events based solely on patient recall without supporting documentation should not be counted towards eligibility.
- •Hemoglobin ≥ 5.5 and ≤ 10.5 g/dL (≥ 55 and ≤ 105 g/L) during screening.
- •For participants taking HU, the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator.
- •Patients on crizanlizumab or l-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they: ï‚· Have been on a stable dose for ≥ 12 months at the time of consent (i.e., no changes to the dose except for changes to weight or for safety reasons) ï‚· Have been ≥ 80% compliant with the planned regimen during the 12 months prior to the time of consent ï‚· Meet the VOC eligibility requirement in Inclusion Criterion
- •Patients, who if female and of childbearing potential, are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and who if male are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug.
排除标准
- •Medical Conditions
- •More than 15 VOCs (defined in protocol Section 8.2.2) within the past 12 months prior to screening
- •Hospitalized for sickle cell crisis or other vaso-occlusive event within 14 days of Screening
- •Female who is breast feeding or pregnant
- •Hepatic dysfunction characterized by.
- •Alanine aminotransferase (ALT) greater than 4.0 × upper limit of normal (ULN) OR.
- •Direct bilirubin greater than 3.0 × ULN
- •Patients with clinically significant bacterial, fungal, parasitic, or viral infection requiring systemic therapy.
- •Patients with acute bacterial, fungal, parasitic, or viral infection requiring systemic therapy should delay screening or enrollment until active therapy has been completed Note: Infection prophylaxis is allowed (see concomitant medication restrictions)
- •Known human immunodeficiency virus (HIV) positivity
- •Active infection with hepatitis B virus (hepatitis B surface antigen [HepBsAg] and hepatitis B core antibody [HepBcAb] positive)
- •Active hepatitis C infection
- •Severe renal dysfunction (estimated glomerular filtration rate at the Screening visit; calculated by the central laboratory less than 30 mL/min/1.73 m2) or on chronic dialysis
- •History of malignancy within the past 2 years prior to treatment Day 1 requiring systemic chemotherapy and/or radiation ï‚· Patients with malignancy considered surgically cured are eligible (e.g., non-melanoma skin cancer, cancer of the cervix in-situ, ductal carcinoma in situ [stage 1], grade 1 endometrial cancer).
结局指标
主要结局
Hemoglobin response rate at Week 24 (increase of greater than 1 g/dL [grater than 10 g/L] from baseline) during the blinded treatment period
时间窗: 24 Weeks | 52 Weeks
Annualized vaso-occlusive crisis rate during the 52-week blinded treatment period based on adjudicated vaso-occlusive crisis review
时间窗: 24 Weeks | 52 Weeks
次要结局
- Change from baseline in hemoglobin at Week 24 during the blinded treatment period(24 Weeks)
- Change from baseline in hemoglobin at Week 52 during the blinded treatment period(52 Weeks)
- Change in absolute reticulocyte count from baseline at Week 24 during the blinded treatment period(24 Weeks)
- Change in unconjugated bilirubin from baseline at Week 24 during the blinded treatment period(24 Weeks)
- Change in lactate dehydrogenase from baseline at Week 24 during the blinded treatment period(24 Weeks)
- Time to first vaso-occlusive crisis during the blinded treatment period(52 Weeks)
- Change in PROMIS Fatigue Scale from baseline in adult patients at Week 24 during the blinded treatment period(24 Weeks)
- Change in PROMIS Fatigue Scale from baseline in adult patients at Week 52 during the blinded treatment period(52 Weeks)
研究者
Dr Meera Chakra
Victoria hospital
研究点 (6)
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