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)
Pre-Transplant Immune Suppression With Hematopoietic Cell Transplantation From Haploidentical Donors for Adults and Children With Sickle Cell Disease or ß-Thalassemia (Haplo PTCy)
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: To join this study, participants generally need to meet the listed inclusion criteria (30 items) and avoid the listed exclusions (11 items).
Location: Memorial Sloan Kettering at Basking Ridge (Consent only) — Basking Ridge, New Jersey
Age range: 2 Years to 50 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistCheck every box below. If you can check all of them, you may meet the study's basic screening requirements (final eligibility is confirmed by the site team).
Usually NOT eligible if any of the following apply:
- 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.
🔍 Extracted verbatim from the official NIH eligibility criteria. Always confirm with the study coordinator.
📍 All Participating Trial Locations (6 Sites)
- Memorial Sloan Kettering at Basking Ridge (Consent only) — Basking Ridge, New Jersey
- Memorial Sloan Kettering Monmouth (Consent only) — Middletown, New Jersey
- Memorial Sloan Kettering Bergen (Consent only) — Montvale, New Jersey
- Memorial Sloan Kettering Suffolk - Commack (Consent only) — Commack, New York
- Memorial Sloan Kettering Westchester (Consent only) — Harrison, New York
- Memorial Sloan Kettering Nassau (All protocol activities) — Rockville Centre, New York
📞 Contact the Study Coordinator
Phone: (212) 639-2446
Email: canciom@mskcc.org
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
- 💵 Compensation indicated: 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.
More studies in Sickle Cell Disease
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- Kidney Function in Sickle Cell Anemia — Chapel Hill, North Carolina
- BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease — Birmingham, Alabama
- Virtual Reality for SCD VOC — Baltimore, Maryland
- Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease — Bethesda, Maryland
- Acupressure in Patients With Sickle Cell Disease — Irvine, California
Explore by condition: All Sickle Cell Disease clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.