Testing Alemtuzumab with Total Body Irradiation for sickle cell disease
Official title: Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
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)
- Phase 2
- 1 group
- One site, in Calgary
- Recruiting
Interventions
- Medication
Alemtuzumab
Alemtuzumab, Day -7 to -3. Dose: 0.2mg/kg/dose SC once daily x 5 days
- Radiation therapy
Total Body Irradiation
TBI 300 cGy on Day -2
- Medication
Sirolimus
Sirolimus is used for GVHD prophylaxis
Canadian Sites (1)
1 of 1 recruiting
- Recruiting
Alberta Children's Hospital
Calgary, Alberta
Eligibility Criteria
See who this study is looking for18 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Patients must be ≥ 12 months and \< 19 years of age at the time of study enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →.
- +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)
- +Patients must meet standard eligibility criteriaEligibility criteriaThe full list of requirements for taking part in a study.Read more → 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.
Exclusion
- −Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components.
- −Patients who are unable to comply with or follow the study protocolProtocolThe detailed plan a study must follow.Read more →.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more → — a middle-stage study in a few hundred people at most, looking at what the treatment does and watching for side effectsSide effectAn unwanted effect thought to be caused by the treatment itself.Read more →.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
Everyone in this study is in one group, which receives one or more of: Alemtuzumab, Total Body Irradiation and Sirolimus.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
There is only one group in this study, so there is no assignment to different treatments.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists an age range of 1 year to 19 years.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 12 people.
The study is currently expected to finish around July 2028.
The main measurement is taken over: 6 months from enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Incidence of pure red cell aplasia (PRCA) — measured over 6 months from enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT03214354 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About AlemtuzumabDrug
Alemtuzumab, Day -7 to -3. Dose: 0.2mg/kg/dose SC once daily x 5 days
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Total Body IrradiationRadiation
TBI 300 cGy on Day -2
From the trial registry — its own words, unedited.
What a radiation is here: A form of radiotherapy or radiation-based treatment being studied.
Read the full explanation → · in clinical review
About SirolimusDrug
Sirolimus is used for GVHD prophylaxis
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: an age range of 1 year to 19 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
There is only one group in this study, so there is no assignment to different treatments.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists an age range of 1 year to 19 years.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.maximumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: 6 months from enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →.
The study as a whole is currently expected to finish around 2028-07.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 12 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists one location: Calgary, Alberta, Canada.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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.
Think this trial might be right for you?
Complete a quick intake form and we will match you with this and other relevant trials based on your medical profile.
See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.