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)
NCT ID: NCT05736419
Last Updated: 2025-05-20
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
24 participants
INTERVENTIONAL
2023-02-09
2026-02-09
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Participants with Sickle Cell Disease or β-Thalassemia
Participants will have severe sickle cell disease or transfusion-dependent β-thalassemia.
Fludarabine
PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.
Cyclophosphamide
Cyclophosphamide will be administered Post-Transplant
Tacrolimus
Tacrolimus will be administered beginning on day +5
Mycophenolate Mofetil
Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.
Rabbit ATG
The dose and schedule of ATG will be determined according to the nomogram in Appendix A
Dexamethasone
Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.
Bortezomib
Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33
Rituximab
Rituximab on days -71, -58, -43, and -30.
Interventions
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Fludarabine
PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.
Cyclophosphamide
Cyclophosphamide will be administered Post-Transplant
Tacrolimus
Tacrolimus will be administered beginning on day +5
Mycophenolate Mofetil
Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.
Rabbit ATG
The dose and schedule of ATG will be determined according to the nomogram in Appendix A
Dexamethasone
Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.
Bortezomib
Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33
Rituximab
Rituximab on days -71, -58, -43, and -30.
Eligibility Criteria
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Inclusion Criteria
* 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
* 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 2x 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 Criteria
* 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
* HIV positive
* Active hepatitis B or C.
* Other uncontrolled infections.
* BMI \> 40.
* 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 cell product, including a known history of allergic reactions to DMSO.
2 Years
50 Years
ALL
No
Sponsors
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Memorial Sloan Kettering Cancer Center
OTHER
Responsible Party
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Principal Investigators
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Maria Cancio, MD
Role: PRINCIPAL_INVESTIGATOR
Memorial Sloan Kettering Cancer Center
Locations
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Memorial Sloan Kettering at Basking Ridge (Consent only)
Basking Ridge, New Jersey, United States
Memorial Sloan Kettering Monmouth (Consent only)
Middletown, New Jersey, United States
Memorial Sloan Kettering Bergen (Consent only)
Montvale, New Jersey, United States
Memorial Sloan Kettering Suffolk - Commack (Consent only)
Commack, New York, United States
Memorial Sloan Kettering Westchester (Consent only)
Harrison, New York, United States
Memorial Sloan Kettering Nassau (All protocol activities)
Rockville Centre, New York, United States
Countries
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Central Contacts
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Facility Contacts
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Maria Cancio, MD
Role: primary
Maria Cancio, MD
Role: primary
Maria Cancio, MD
Role: primary
Maria Cancio, MD
Role: primary
Maria Cancio, MD
Role: primary
Maria Cancio, MD
Role: primary
Related Links
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Memorial Sloan Kettering Cancer Center
Other Identifiers
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23-009
Identifier Type: -
Identifier Source: org_study_id
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