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.
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