NCT05392894RecruitingPhase 3
RElated Haplo-DonoR Haematopoietic stEm Cell Transplantation for Adults With Severe Sickle Cell Disease
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
Source: ClinicalTrials.gov · Last updated 10 May 2023 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 120 (estimated)
- First submitted
- 23 May 2022
- Start date
- 23 Feb 2023
- Primary completion date
- Mar 2027
- Lead sponsor
- King's College Hospital NHS Trust
- Diseases
- Sickle cell disease
- NCT number
- NCT05392894
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 120
- From 23 Feb 2023 to the primary completion date, Mar 2027
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous bone marrow transplant.
- Clinically significant donor specific HLA antibodies.
Other illness
- HIV infection or active Hepatitis B or C.
- Uncontrolled infection including bacterial, fungal and viral.
- Pre-existing condition deemed to significantly increase the risk of Haploidentical SCT by the local Principal Investigator.
Other
- Fully matched sibling donor.
- Pregnancy or breast feeding.
- Participants able to conceive a child that are unprepared to use effective contraception.
- Participation in another interventional trial in the last three months.
Who the study is looking for
- 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:
- Clinically significant neurologic event (stroke) or deficit lasting \> 24 hours.
- History of ≥2 acute chest syndromes in a 2-year period preceding enrolment despite optimum treatment, e.g. with hydroxycarbamide (HC).
- 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).
- Administration of regular transfusion therapy (=8 packed red blood transfusions per year for 1 year to prevent vaso-occlusive complications).
- Patients assessed as requiring transfusion but with red cell allo-antibodies/very rare blood type, rendering it difficult to continue/commence chronic transfusion.
- Patients requiring HC/transfusion for treatment of SCD complications who cannot tolerate either therapy due to significant adverse reactions.
- 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.
- 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.
- Written informed consent.
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| King's College Hospital Coordinator of this site? Confirm its status | London | United Kingdom | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.