Sickle Cell DiseasePhase 2Sickle-AIDNCT03214354
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
A Phase 2 study testing Alemtuzumab, Total Body Irradiation and Sirolimus in people with Sickle Cell Disease and Stem Cell Transplant Complications, run by University of Calgary at 1 site in Canada.
Trial summary
The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. Read more about this study
- Who is it for?
- Ages 1 year to 19 years
- Conditions the study is for
- Sickle Cell Disease, Stem Cell Transplant Complications, Red Blood Cell Disorder
- What is being tested?
- Alemtuzumab, Total Body Irradiation and Sirolimus
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteCanada
- Study dates
- 5 Jul 2017to Jul 2028
- Phase
- Phase 2
- 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 1 year to 19 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be ≥ 12 months and < 19 years of age at the time of study enrollment.
- Patients must have sickle cell disease as defined by hemoglobin electropheresis, as follows:
- homozygous Hb S disease (HbSS),
- sickle-Hb C disease (HbSC),
- sickle beta-plus-thalassemia (HbS/β+), or
- sickle beta-null-thalassemia (HbS/βo)
Show all 16Show fewer
- Patients must meet standard eligibility criteria to undergo HSCT, including but not limited to one or more of the following:
- history of repeated (more than 1) bony (vaso-occlusive) crisis
- history of stroke
- elevated transcranial Doppler velocity not eligible for hydroxyurea, as per TWiTCH trial (ie. severe vasculopathy)
- history of acute chest crisis or splenic sequestration crisis
- history of priapism in males
- history of osteonecrosis
- pulmonary hypertension as documented by tricuspid regurgitation jet velocity (TRV) > 2.5 m/s on echocardiogram
- red cell allo-immunization (≥ 2 antibodies) during long term transfusion therapy
- Sickle complications should be present despite the use of hydroxyurea, but this is not an absolute requirement, if the treating team considers the patient to be at high risk for further crisis episodes.
Who cannot join
- Patients who are unable to comply with or follow the study protocol.
- Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Greg Guilcher, MD contact
Canada1 site, 1 recruiting
- Recruiting
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Tony Truong, MD, MPH, 403-955-7272, tony.truong@ahs.ca
Greg Guilcher, MD, 403-955-7272
About this study
Official title: A Phase II Pilot Study of Nonmyeloablative Conditioning Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease Who Have a Matched Related Major ABO-Incompatible Donor (Sickle-AID)
The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.
Read the detailed description
Sickle cell disease (SCD) is a debilitating chronic blood disorder with multi-system end-organ damage that leads to morbidity and early mortality. The only cure for SCD is hematopoietic stem cell transplantation (HSCT), which given the risks with unrelated HSCT, is only an option for a minority of patients who have a matched sibling donor.
In the field of HSCT, blood group ABO incompatibility between donor and recipient is not a contraindication and several studies do not show compromised outcomes. However, in the context of nonmyeloablative (NMA) conditioning and major ABO-incompatibility, when the recipient has existing antibodies to donor red blood cells, pure red cell aplasia (PRCA) may occur.
This phase II pilot study will enroll SCD patients with a matched related major ABO-incompatible donor to determine the safety and efficacy of NMA-HSCT. Biological studies will include a plan to study and monitor red cell engraftment in this population to facilitate early detection and interventional measures to prevent and treat PRCA.
- Conditions
- Sickle Cell Disease, Stem Cell Transplant Complications, Red Blood Cell Disorder, Pure Red Cell Aplasia
- Being tested
- Alemtuzumab, Total Body Irradiation, Sirolimus
- Keywords
- sickle cell disease, stem cell transplant, red blood cell engraftment, nonmyeloablative, pure red cell aplasia
- Medical subject headings
- Anemia, Sickle Cell, Red-Cell Aplasia, Pure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlemtuzumabDrug
Also called Campath
Alemtuzumab, Day -7 to -3. Dose: 0.2mg/kg/dose SC once daily x 5 days
Total Body IrradiationRadiation
Also called TBI
TBI 300 cGy on Day -2
SirolimusDrug
Sirolimus is used for GVHD prophylaxis
| Study group | Role | What they receive |
|---|---|---|
| Non-myeloablative conditioning | Experimental | Non-myeloablative conditioning Receives: Drug: Alemtuzumab; Radiation: Total Body Irradiation; Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of pure red cell aplasia (PRCA)
Clinical definition: reticulocytopenia < 10x109/L (< 1%) lasting more than 60 days after HSCT, or Pathological definition: the absence of erythroid precursors in the marrow in the setting of adequate myeloid, lymphoid and megakaryocytic precursors
Measured: 6 months from enrollment
Secondary outcomes (7)
RBC chimerism measured by peripheral blood flow cytometry
Peripheral blood for RBC chimerism on flow sorted erythroid precursor cells
Measured: 12 months
RBC chimerism measured by bone marrow BFU-erythroid forming colonies
Bone marrow will be performed between Day +45 and +60
Measured: 2 months
Primary graft failure
Measured by donor chimerism from peripheral blood and bone marrow
Measured: 6 weeks
Secondary graft failure
Measured by donor chimerism in peripheral blood and bone marrow
Measured: 24 months
Disease recurrence
Measured by peripheral blood Hb S level
Measured: 24 months
Incidence and severity of acute GVHD
Acute GVHD grade will be accessed using modified CIBMTR criteria
Measured: 100 days
Incidence and severity of chronic GVHD
Chronic GVHD will be accessed using the NIH consensus criteria
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jul 2017
- Study start5 Jul 2017
- Primary completionJul 2028 (estimated)
- Study completionJul 2028 (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 University of CalgaryThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Phase II pilot, non-randomized, prospective study to evaluate the safety and efficacy of a nonmyeloablative conditioning allogeneic stem cell transplantation for patients with sickle cell disease who have a matched related major ABO-incompatible donor.
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Calgary
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Tony H. Truong, University of Calgary
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03214354
- Sponsor's protocol ID
- TRU-17-001
- Acronym
- Sickle-AID
- Status last verified
- Apr 2026
- First submitted
- 5 Jul 2017
- Last updated
- 1 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Pilot Study of Nonmyeloablative Conditioning Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease Who Have a Matched Related Major ABO-Incompatible Donor (Sickle-AID)
- ClinicalTrials.gov ID
- NCT03214354
- Acronym / sponsor ID
- Sickle-AID / TRU-17-001
- Conditions
- Sickle Cell Disease, Stem Cell Transplant Complications, Red Blood Cell Disorder, Pure Red Cell Aplasia
- Keywords
- sickle cell disease, stem cell transplant, red blood cell engraftment, nonmyeloablative, pure red cell aplasia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- University of CalgaryUniversity, hospital or other
- Study start
- 5 Jul 2017 (actual)
- Primary completion
- Jul 2028 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 11 Jul 2017 (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 aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.
Detailed description
Sickle cell disease (SCD) is a debilitating chronic blood disorder with multi-system end-organ damage that leads to morbidity and early mortality. The only cure for SCD is hematopoietic stem cell transplantation (HSCT), which given the risks with unrelated HSCT, is only an option for a minority of patients who have a matched sibling donor.
In the field of HSCT, blood group ABO incompatibility between donor and recipient is not a contraindication and several studies do not show compromised outcomes. However, in the context of nonmyeloablative (NMA) conditioning and major ABO-incompatibility, when the recipient has existing antibodies to donor red blood cells, pure red cell aplasia (PRCA) may occur.
This phase II pilot study will enroll SCD patients with a matched related major ABO-incompatible donor to determine the safety and efficacy of NMA-HSCT. Biological studies will include a plan to study and monitor red cell engraftment in this population to facilitate early detection and interventional measures to prevent and treat PRCA.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Phase II pilot, non-randomized, prospective study to evaluate the safety and efficacy of a nonmyeloablative conditioning allogeneic stem cell transplantation for patients with sickle cell disease who have a matched related major ABO-incompatible donor.
| Arm | Type | Receives |
|---|---|---|
| Non-myeloablative conditioning | Experimental | Drug: Alemtuzumab, Radiation: Total Body Irradiation, Drug: SirolimusNon-myeloablative conditioning |
Who may qualify
- Ages
- 1 year to 19 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (16 listed)
- Patients must be ≥ 12 months and < 19 years of age at the time of study enrollment.
- Patients must have sickle cell disease as defined by hemoglobin electropheresis, as follows:
- Patients must meet standard eligibility criteria to undergo HSCT, including but not limited to one or more of the following:
- Sickle complications should be present despite the use of hydroxyurea, but this is not an absolute requirement, if the treating team considers the patient to be at high risk for further crisis episodes.
Key exclusion criteria (2 listed)
- Patients who are unable to comply with or follow the study protocol.
- Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components.
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 |
|---|---|---|
| AlemtuzumabAlso: CampathAlemtuzumab, Day -7 to -3. Dose: 0.2mg/kg/dose SC once daily x 5 days | Drug | Non-myeloablative conditioning |
| Total Body IrradiationAlso: TBITBI 300 cGy on Day -2 | Radiation | Non-myeloablative conditioning |
| SirolimusSirolimus is used for GVHD prophylaxis | Drug | Non-myeloablative conditioning |
Primary outcome
Incidence of pure red cell aplasia (PRCA)
Time frame: 6 months from enrollment
Clinical definition: reticulocytopenia < 10x109/L (< 1%) lasting more than 60 days after HSCT, or Pathological definition: the absence of erythroid precursors in the marrow in the setting of adequate myeloid, lymphoid and megakaryocytic precursors
7 secondary outcomes
RBC chimerism measured by peripheral blood flow cytometry
Time frame: 12 months
RBC chimerism measured by bone marrow BFU-erythroid forming colonies
Time frame: 2 months
Primary graft failure
Time frame: 6 weeks
Secondary graft failure
Time frame: 24 months
Disease recurrence
Time frame: 24 months
Incidence and severity of acute GVHD
Time frame: 100 days
Incidence and severity of chronic GVHD
Time frame: 24 months
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 1 May 2026.
- Individual participant dataSharing planned: No
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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.
Greg Guilcher, MD contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Alemtuzumab, Total Body Irradiation and Sirolimus in people with Sickle Cell Disease and Stem Cell Transplant Complications, run by University of Calgary at 1 site in Canada.
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 16 inclusion and 2 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 1 May 2026. 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 Canada. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Tony Truong, MD, MPH (403-955-7272, tony.truong@ahs.ca), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of pure red cell aplasia (PRCA) (time frame: 6 months from enrollment). 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 1 May 2026.