Actively Recruiting
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)
Led by Memorial Sloan Kettering Cancer Center · Updated on 2026-04-08
24
Participants Needed
6
Research Sites
208 weeks
Total Duration
On this page
AI-Summary
What this Trial 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. 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.
CONDITIONS
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 2 to 50 years
- Suitable haploidentical donor
- Performance score 70% or higher by Karnofsky (age > 16) or Lansky Play-Performance Scale 70% or higher (age ≤ 16)
- Adequate organ function, including kidney, liver, heart, and lung function within defined limits
- For sickle cell patients: specific complications like acute chest syndrome, vaso-occlusive episodes, priapism, osteomyelitis/osteonecrosis, cerebrovascular disease, pulmonary hypertension, or red blood cell alloimmunization
- For thalassemia patients: transfusion dependence starting in first 3 years of life, high transfusion history, low pre-transfusion hemoglobin, and hepatosplenomegaly
- Ability to provide written informed consent or assent with legal representative consent
- Agreement to use contraception if sexually active and of childbearing potential
You will not qualify if you...
- Prior myeloablative allogeneic hematopoietic cell transplant
- Stroke or CNS surgery within 6 months before enrollment
- Liver cirrhosis or high hepatic iron content
- Active hepatitis B or C or other uncontrolled infections
- Current cancer diagnosis unless in remission for at least 2 years (some exceptions apply)
- Positive pregnancy test in women of childbearing potential
- Inability to follow medical therapy or attend follow-up
- Known allergy to stem cell product components including DMSO
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
Memorial Sloan Kettering at Basking Ridge (Consent only)
Basking Ridge, New Jersey, United States, 07920
Actively Recruiting
2
Memorial Sloan Kettering Monmouth (Consent only)
Middletown, New Jersey, United States, 07748
Actively Recruiting
3
Memorial Sloan Kettering Bergen (Consent only)
Montvale, New Jersey, United States, 07645
Actively Recruiting
4
Memorial Sloan Kettering Suffolk - Commack (Consent only)
Commack, New York, United States, 11725
Actively Recruiting
5
Memorial Sloan Kettering Westchester (Consent only)
Harrison, New York, United States, 10604
Actively Recruiting
6
Memorial Sloan Kettering Nassau (All protocol activities)
Rockville Centre, New York, United States, 11553
Actively Recruiting
Research Team
M
Maria Cancio, MD
CONTACT
J
Jaap Jan Boelens, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here