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 now · Phase 2

Conditions studied: Sickle Cell Disease, Thalassemia, Beta, Thalassemia

In brief

Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.

Key facts

Study ID: NCT05736419
Run by: Memorial Sloan Kettering Cancer Center
People needed: 24
Starts: 2023-02-09
Expected to finish: 2027-02-09
Last updated by the study team: 2026-06-03

Who can join

Age: 2 and older, up to 50. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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

You may not qualify if…

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.

Where it is running

Memorial Sloan Kettering at Basking Ridge (Consent only) — Basking Ridge, New Jersey, United States (enrolling)
Memorial Sloan Kettering Monmouth (Consent only) — Middletown, New Jersey, United States (enrolling)
Memorial Sloan Kettering Bergen (Consent only) — Montvale, New Jersey, United States (enrolling)
Memorial Sloan Kettering Suffolk - Commack (Consent only) — Commack, New York, United States (enrolling)
Memorial Sloan Kettering Westchester (Consent only) — Harrison, New York, United States (enrolling)
Memorial Sloan Kettering Nassau (All protocol activities) — Rockville Centre, New York, United States (enrolling)

Full record on ClinicalTrials.gov

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