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 (REDRESS V1.0)

King's College Hospital NHS Foundation Trust0 个研究点目标入组 120 人2022年10月25日

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who.int

开始日期

2022年10月25日

结束日期

2027年6月30日

最后更新

3年前

研究类型

Interventional

性别

All

发起方

•1\. Adult patients aged \> \= 18 years
•2\. Confirmed haploidentical donor
•3\. Severe SCD phenotype who are at high risk for morbidity and mortality. Severe SCD is defined by at least one of the following:
•3\.1\. Clinically significant neurologic event (stroke) or deficit lasting \> 24 hours
•3\.2\. History of \> \= 2 acute chest syndromes in a 2\-year period preceding enrolment despite optimum treatment, e.g. with hydroxycarbamide (HC)
•3\.3\. 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)
•3\.4\. Administration of regular transfusion therapy (\=8 packed red blood transfusions per year for 1 year to prevent vaso\-occlusive complications).
•3\.5\. Patients assessed as requiring transfusion but with red cell alloantibodies/very rare blood type, rendering it difficult to continue/commence chronic transfusion
•3\.6\. Patients requiring HC/transfusion for treatment of SCD complications who cannot tolerate either therapy due to significant adverse reactions
•3\.7\. 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.

•1\. Fully matched sibling donor.
•2\. Previous bone marrow transplant.
•3\. Pregnancy or breast feeding.
•4\. Participants who are able to conceive a child and are unprepared to use effective contraception.
•5\. Clinically significant donor\-specific HLA antibodies.
•6\. HIV infection or active Hepatitis B or C.
•7\. Uncontrolled infection including bacterial, fungal and viral.
•8\. Participation in another interventional trial in the last three months.
•9\. Pre\-existing condition deemed to significantly increase the risk of Haploidentical SCT by the local Principal Investigator.