Immune Suppression Treatment for Sickle Cell Disease or Thalassemia Before Stem Cell Transplant
This study is looking at a new way to prepare people with Sickle Cell Disease or Thalassemia for a hematopoietic cell transplant (HCT), also known as a stem cell transplant. Researchers are testing if giving drugs like fludarabine, cyclophosphamide, tacrolimus, mycophenolate mofetil, and rabbit ATG, which lower your body's immune system activity (immune suppression), before the transplant can help prevent serious side effects like graft failure. The study plans to enroll 24 participants between 2 and 50 years old who have a suitable donor. The main goal is to see how many participants experience treatment-related death or graft failure within one year after the transplant. The current status of this study is unclear.
- Study design
- This study is an interventional trial with a planned enrollment of 24 participants. It is testing a specific treatment approach for people undergoing a stem cell transplant.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Participants will be followed for at least one year to monitor for treatment-related mortality or primary graft failure.
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NCT05736419
A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
Recruiting
PHASE2Ages 2–50InterventionalTreatmentMemorial Sloan Kettering Cancer CenterInvestigator-initiated
~24 participants
Updated 2026-06-03 on ClinicalTrials.gov
What's tested:FludarabineCyclophosphamideTacrolimusMycophenolate MofetilRabbit ATGDexamethasone
At a glance
Recruiting sites
6 of 6 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of participants with treatment related mortality/TRM or primary graft failure
Measured over 1 year
Conditions
Where it's being run
6 sites across 2 statesNew Jersey3
New York3
Study leadership
- Maria Cancio, MD · PRINCIPAL_INVESTIGATOR · Memorial Sloan Kettering Cancer Center
Who to contact
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Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
Age ≥ 2 and ≤ 50 years
Suitable haploidentical donor.
Performance score ≥ 70% by Karnofsky Performance Scale or 0 to 1 by ECOG (age \> 16 years), or Lansky Play-Performance Scale ≥ 70% (age ≤ 16 years).
Adequate major organ system function as demonstrated by:
For patients ≥ 18 years of age:
eGFR ≥ 50 mL/min by Cockcroft-Gault formula Formula: ((140 - Age) x Weight (kg)) / (72 x Serum Creatinine (mg/dL) Female Adjustment: Multiply result by 0.85
For patients \< 18 years of age:
Serum creatinine clearance: glomerular filtration rate \[GFR\]) must be \>50 mL/min/1.73 m2 as calculated by the Schwartz formula
Conjugated (direct) bilirubin less than 3x upper limit of normal.
ALT or AST ≤ 3 times institutional upper limit of normal.
Left ventricular ejection fraction ≥ 50%.
Diffusing capacity for carbon monoxide (DLCO) ≥ 50% predicted, corrected for hemoglobin. For children \< 7 years of age who are unable to perform PFT, oxygen saturation \> 92% on room air by pulse oximetry.
For SCD patients: HbSS, HbSC, HbS/β° with one or more of the following complications:
Acute chest syndrome: 2 or more episodes in the 2 years preceding enrollment
Vaso-occlusive episodes: 3 or more episodes in the 2 years preceding enrollment
Recurrent priapism: 2 or more episodes in the 2 years preceding enrollment
History of osteomyelitis or osteonecrosis
Cerebrovascular disease:
Imaging evidence of prior overt or silent stroke
History of a neurologic event resulting in focal neurologic deficits lasting \> 24 hours
Abnormal transcranial Doppler: Timed average maximum mean velocity ≥ 200 cm/sec in terminal portion of the carotid or proximal portion of the middle cerebral artery or \> 185 cm/sec plus evidence of intracranial vasculopathy if imaging TCD is used
Pulmonary hypertension: Confirmed by right heart catheterization with mean pulmonary arterial pressure ≥ 25 mmHg or mean pulmonary vascular resistance \> 2 Wood units
Red blood cell alloimmunization (\> 3 alloantibodies)
For thalassemia patients: Any genotype, with all of the following:
Onset of red blood cell transfusion dependence during the first 3 years of life
RBC transfusion history \> 225 mL/kg/year or \> 15 lifetime RBC transfusions
Pre-transfusion hemoglobin ≤ 7 g/dL
Hepatosplenomegaly
Patient or the patient's legal representative, parent(s) or guardian should be able to provide written informed consent. Assent of a minor if participant's age is at least seven and less than eighteen years.
For sexually active men and women of childbearing potential, must agree to use a form of contraception considered effective and medically acceptable by the Investigator.
Exclusion
Prior myeloablative allogeneic HCT.
Overt stroke or CNS instrumentation (e.g. for Moyamoya disease) within 6 months of enrollment.
Liver cirrhosis. Mild fibrosis will be permitted, i.e. fine reticulin or grade 1 of 4, with bridging fibrosis.
Hepatic iron content ≥ 3 mg Fe/g liver dry weight, if applicable
Active hepatitis B or C.
Other uncontrolled infections.
Other malignancy/cancer diagnosis unless in remission after definitive therapy for a minimum of 2 years. Exceptions: Ductal carcinoma in situ, basal cell carcinoma, cervical intraepithelial neoplasia.
Positive pregnancy test in a woman with child-bearing potential, defined as not post-menopausal for 12 months or no previous surgical sterilization.
Inability to comply with medical therapy or follow-up.
Known history of allergic reactions to any constituents of the stem cell product, including a known history of allergic reactions to DMSO.
What this trial measures
- Number of participants with treatment related mortality/TRM or primary graft failure1 year
The primary outcome is to estimate treatment-related mortality (TRM) or primary graft failure at 1 year post-HCT.