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 Phase 2 Interventional Study
Sickle Cell Disease Thalassemia, Beta Thalassemia
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
2 – 50
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
24 (estimated)
Sponsor
Memorial Sloan Kettering Cancer Center · Other
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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 …
Trial Locations
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Eligibility Criteria
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 pa…
Contacts

Maria Cancio, MD

212-639-2446

canciom@mskcc.org

CONTACT

Jaap Jan Boelens, MD, PhD

212-639-3643

boelensj@mskcc.org

CONTACT