NCT05392894招募中3 期
A Multi-centre Open Randomised Controlled Trial to Assess the Effect of Related Haplo-donor Haematopoietic Stem Cell Transplantation Versus Standard of Care (no Transplant) on Treatment Failure at 24 Month in Adults With Severe Sickle Cell Disease
King's College Hospital NHS Trust1 个研究点 分布在 1 个国家目标入组 120 人开始时间: 2023年2月23日最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 120
- 试验地点
- 1
- 主要终点
- Treatment failure or mortality
研究概览
简要总结
The purpose of this clinical trial is to evaluate the clinical and cost effectiveness of Haploidentical Stem Cell Transplantation (SCT) for adults with severe sickle cell disease (SCD), who have failed other therapies or are intolerant of existing therapies or require chronic transfusions to prevent on-going complications of SCD.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 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
- •Adult patients age ≥ 18 years
- •Confirmed haploidentical donor
- •Severe SCD phenotype who are at high risk for morbidity and mortality. Severe SCD is defined by at least one of the following:
- •i. Clinically significant neurologic event (stroke) or deficit lasting > 24 hours.
- •ii. History of ≥2 acute chest syndromes in a 2-year period preceding enrolment despite optimum treatment, e.g. with hydroxycarbamide (HC).
- •iii. History of ≥3 severe pain crises per year in a 2-year period preceding enrolment despite the institution of supportive care measures (e.g. optimum treatment with HC).
- •iv. Administration of regular transfusion therapy (=8 packed red blood transfusions per year for 1 year to prevent vaso-occlusive complications).
- •v. Patients assessed as requiring transfusion but with red cell allo-antibodies/very rare blood type, rendering it difficult to continue/commence chronic transfusion.
- •vi. Patients requiring HC/transfusion for treatment of SCD complications who cannot tolerate either therapy due to significant adverse reactions.
- •vii. Established end organ damage relating to SCD, including but not limited to progressive sickle vasculopathy and hepatopathy. End-organ sufficient for entry to this trial shall be ratified at the UK NHP.
- •d) Patients must be fit to proceed to Haploidentical SCT as defined below: i. Karnofsky score ≥60 ii. Cardiac function: LVEF ≥45% or shortening fraction ≥25% iii. Lung Function: FEV1, FVC and TLCO ≥50% iv. Renal function: EDTA GFR ≥40 ml/min/1.73m2 v. Hepatic function: ALT <x3 ULN and bilirubin <x2 the upper limit of normal, those with hyperbilirubinemia due to sickle related haemolysis will not be excluded. No radiological evidence of cirrhosis.
- •e) Written informed consent.
排除标准
- •Fully matched sibling donor.
- •Previous bone marrow transplant.
- •Pregnancy or breast feeding.
- •Participants able to conceive a child that are unprepared to use effective contraception.
- •Clinically significant donor specific HLA antibodies.
- •HIV infection or active Hepatitis B or C.
- •Uncontrolled infection including bacterial, fungal and viral.
- •Participation in another interventional trial in the last three months.
- •Pre-existing condition deemed to significantly increase the risk of Haploidentical SCT by the local Principal Investigator.
结局指标
主要结局
Treatment failure or mortality
时间窗: 24 months post-randomisation
Treatment failure is defined as occurrence of vaso-occlusive crisis, or transfusion from 6 months post-randomisation.
次要结局
- Sickle cell disease related complications(24 months post-randomisation)
- Renal Function(At 6, 12 and 24 months post-randomisation)
- Sickle type haemoglobin percentage (HbS%)(At 6, 12 and 24 months post-randomisation)
- Sickle Cell Disease-related mortality (excluding transplant related complications)(24 months post-randomisation)
- Haemoglobin levels, Reticulocyte count, LDH, Bilirubin(At 6, 12 and 24 months post-randomisation)
- All cause mortality(24 months post-randomisation)
- Pulmonary Function(At 12 months and 24 months post-randomisation)
- Cerebrovascular progression(24 months post-randomisation)
- Health related quality of life(At 3, 6, 9, 12, 15, 18, 21 and 24 months post-randomisation)
- Iron overload(24 months post-randomisation)
- Cardiac function and pulmonary hypertension(At 12 and 24 months post-randomisation)
- Evidence of hepatic progression(24 months post-randomisation)
- Percentage of participants requiring opioid use for pain related to vaso-occlusive sickle related crisis(At 12 and 24 months post-randomisation)
研究者
研究点 (1)
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