NCT05736419 · Memorial Sloan Kettering Cancer Center
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)
What this study is about
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.
View original scientific description
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.
Interventions
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.
Primary outcome measures
Number of participants with treatment related mortality/TRM or primary graft failure
Time frame: 1 year
The primary outcome is to estimate treatment-related mortality (TRM) or primary graft failure at 1 year post-HCT.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Age \> 12 and \< 35 years. Patients of age 2-12 and 35-50 years will be included after 8 patients reach day 100 without TRM and if ≥ 4 of the first 8 patients reach day 100 without graft failure or grade III-IV acute GvHD.
- 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 hemo
Where
- Basking Ridge, New Jersey
- Middletown, New Jersey
- Montvale, New Jersey
- Commack, New York
- Harrison, New York
- Rockville Centre, New York
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Jun 3, 2026 · Source of record for eligibility and locations