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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 2INTERVENTIONAL

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)

Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)

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) (NCT05736419) is a Phase 2 interventional studying Sickle Cell Disease and Thalassemia, Beta, sponsored by Memorial Sloan Kettering Cancer Center. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Sickle Cell Disease and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 24 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 - 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: ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * 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 * 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 Criteria: * 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.

Treatments Being Tested

DRUG

Fludarabine

PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.

DRUG

Cyclophosphamide

Cyclophosphamide will be administered Post-Transplant

DRUG

Tacrolimus

Tacrolimus will be administered beginning on day +5

DRUG

Mycophenolate Mofetil

Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.

BIOLOGICAL

Rabbit ATG

The dose and schedule of ATG will be determined according to the nomogram in Appendix A

DRUG

Dexamethasone

Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.

DRUG

Bortezomib

Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33

DRUG

Rituximab

Rituximab on days -71, -58, -43, and -30.

Locations (6)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

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

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT05736419), the sponsor (Memorial Sloan Kettering Cancer Center), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT05736419 clinical trial studying?

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 parti… The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT05736419?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT05736419?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT05736419. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT05736419. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.