Sickle Cell DiseasePhase 3REDRESSNCT05392894
RElated Haplo-DonoR Haematopoietic stEm Cell Transplantation for Adults With Severe Sickle Cell Disease
A Phase 3 study testing Haploidentical stem cell transplantation and Standard medical care in people with Sickle Cell Disease, run by King's College Hospital NHS Trust at 1 site in United Kingdom.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Haploidentical stem cell transplantation and Standard medical care
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 120 participants
- Where
- 1 siteUnited Kingdom
- Study dates
- 23 Feb 2023to Mar 2027
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United Kingdom1 site, 1 recruiting
- Recruiting
King's College Hospital
London
Victoria Potteer, victoriapotter@nhs.net
Victoria Potter
About this study
Official title: 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
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.
- Conditions
- Sickle Cell Disease
- Being tested
- Haploidentical stem cell transplantation, Standard medical care
- Keywords
- Sickle, Haploidentical, Stem cell, Transplant
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Haploidentical stem cell transplantationProcedure or surgery
Stem cell transplant from bone marrow or peripheral blood from haploidentical donor using standard nationally approved transplant procedure.
Standard medical careOther
Standard medical care may include any currently available therapies for SCD patients. These may or may not include regular elective transfusion therapy or medications such as hydroxycarbamide.
| Study group | Role | What they receive |
|---|---|---|
| Standard of care | Active comparator | The comparator arm is standard medical care for this patient population. Standard medical care may include all currently available non-trial therapies for SCD. Receives: Other: Standard medical care |
| Haploidentical stem cell transplantation | Experimental | Participants receiving Haploidentical Stem Cell Transplantation will receive the transplant conditioning regimen as per the standard transplant protocol. Stem cells from a haploidentical donor will be infused on Day 0 according to standard institutional practices. Bone marrow is the preferred stem cell source however peripheral blood may be used as an alternative where required due to donor reasons. Receives: Procedure: Haploidentical stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment failure or mortality
Treatment failure is defined as occurrence of vaso-occlusive crisis, or transfusion from 6 months post-randomisation.
Measured: 24 months post-randomisation
Secondary outcomes (13)
Health related quality of life
Quality of life as measured by EQ-5D-5L
Measured: At 3, 6, 9, 12, 15, 18, 21 and 24 months post-randomisation
All cause mortality
Death by any cause
Measured: 24 months post-randomisation
Sickle Cell Disease-related mortality (excluding transplant related complications)
Death due to any sickle cell disease related cause
Measured: 24 months post-randomisation
Sickle type haemoglobin percentage (HbS%)
Sickle type haemoglobin as measured by haemoglobin electrophoresis
Measured: At 6, 12 and 24 months post-randomisation
Sickle cell disease related complications
Defined as transfusion requirement, painful VOC, stroke, pulmonary hypertension
Measured: 24 months post-randomisation
Haemoglobin levels, Reticulocyte count, LDH, Bilirubin
Measured: At 6, 12 and 24 months post-randomisation
Pulmonary Function
As measured by FEV1 %, FEV1/FVC ratio, TLCO %
Measured: At 12 months and 24 months post-randomisation
Renal Function
As measured by urea, creatinine and eGFR
Measured: At 6, 12 and 24 months post-randomisation
Iron overload
As measured by Ferritin and FerriScan (R2-MRI)
Measured: 24 months post-randomisation
Cardiac function and pulmonary hypertension
As measured by echocardiogram/TRV
Measured: At 12 and 24 months post-randomisation
Cerebrovascular progression
As measured by clinical stroke or evidence of progression on MRI/MRA
Measured: 24 months post-randomisation
Evidence of hepatic progression
As measured by liver function (ALT, AST, ALP, GGT, Bilirubin) and FibroScan
Measured: 24 months post-randomisation
Percentage of participants requiring opioid use for pain related to vaso-occlusive sickle related crisis
Measured: At 12 and 24 months post-randomisation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2022
- Study start23 Feb 2023
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by King's College Hospital NHS TrustThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 120 participants (estimated)
Sponsor and investigators
- Lead sponsor
- King's College Hospital NHS Trust
- Sponsor type
- University, hospital or other
- Collaborators
- King's College London, University of Sheffield, Sheffield Teaching Hospitals NHS Foundation Trust, Imperial College Healthcare NHS Trust, Guy's and St Thomas' NHS Foundation Trust, University College London Hospitals, Manchester University NHS Foundation Trust, St George's University Hospitals NHS Foundation Trust, Barts & The London NHS Trust, National Institute for Health Research, United Kingdom
- Responsible party
- Sponsor
- Investigators
- Ann-Marie Murtagh, Study director, King's College Hospitals NHS Foundation Trust
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05392894
- Sponsor's protocol ID
- IRAS: 312212
- Acronym
- REDRESS
- Status last verified
- May 2023
- First submitted
- 23 May 2022
- Last updated
- 10 May 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 10 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05392894
- Acronym / sponsor ID
- REDRESS / IRAS: 312212
- Conditions
- Sickle Cell Disease
- Keywords
- Sickle, Haploidentical, Stem cell, Transplant
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- King's College Hospital NHS TrustUniversity, hospital or other
- Collaborators
- King's College London, University of Sheffield, Sheffield Teaching Hospitals NHS Foundation Trust, Imperial College Healthcare NHS Trust, Guy's and St Thomas' NHS Foundation Trust, University College London Hospitals, Manchester University NHS Foundation Trust, St George's University Hospitals NHS Foundation Trust, Barts & The London NHS Trust, National Institute for Health Research, United Kingdom
- Study start
- 23 Feb 2023 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 26 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard of care | Active comparator | Other: Standard medical careThe comparator arm is standard medical care for this patient population. Standard medical care may include all currently available non-trial therapies for SCD. |
| Haploidentical stem cell transplantation | Experimental | Procedure: Haploidentical stem cell transplantationParticipants receiving Haploidentical Stem Cell Transplantation will receive the transplant conditioning regimen as per the standard transplant protocol. Stem cells from a haploidentical donor will be infused on Day 0 according to standard institutional practices. Bone marrow is the preferred stem cell source however peripheral blood may be used as an alternative where required due to donor reasons. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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).
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Haploidentical stem cell transplantationStem cell transplant from bone marrow or peripheral blood from haploidentical donor using standard nationally approved transplant procedure. | Procedure or surgery | Haploidentical stem cell transplantation |
| Standard medical careStandard medical care may include any currently available therapies for SCD patients. These may or may not include regular elective transfusion therapy or medications such as hydroxycarbamide. | Other | Standard of care |
Primary outcome
Treatment failure or mortality
Time frame: 24 months post-randomisation
Treatment failure is defined as occurrence of vaso-occlusive crisis, or transfusion from 6 months post-randomisation.
13 secondary outcomes
Health related quality of life
Time frame: At 3, 6, 9, 12, 15, 18, 21 and 24 months post-randomisation
All cause mortality
Time frame: 24 months post-randomisation
Sickle Cell Disease-related mortality (excluding transplant related complications)
Time frame: 24 months post-randomisation
Sickle type haemoglobin percentage (HbS%)
Time frame: At 6, 12 and 24 months post-randomisation
Sickle cell disease related complications
Time frame: 24 months post-randomisation
Haemoglobin levels, Reticulocyte count, LDH, Bilirubin
Time frame: At 6, 12 and 24 months post-randomisation
Pulmonary Function
Time frame: At 12 months and 24 months post-randomisation
Renal Function
Time frame: At 6, 12 and 24 months post-randomisation
Iron overload
Time frame: 24 months post-randomisation
Cardiac function and pulmonary hypertension
Time frame: At 12 and 24 months post-randomisation
Cerebrovascular progression
Time frame: 24 months post-randomisation
Evidence of hepatic progression
Time frame: 24 months post-randomisation
Percentage of participants requiring opioid use for pain related to vaso-occlusive sickle related crisis
Time frame: At 12 and 24 months post-randomisation
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 May 2023.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Ann-Marie Murtagh study director
King's College Hospitals NHS Foundation Trust
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Haploidentical stem cell transplantation and Standard medical care in people with Sickle Cell Disease, run by King's College Hospital NHS Trust at 1 site in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Victoria Potter, BSc, MBBS, FRACP, FRCPA (+44 20 3299 3730, victoriapotter@nhs.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment failure or mortality (time frame: 24 months post-randomisation). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 May 2023.