Clin2
NCT03587272Possibly a fitRecruiting

Sibling bone marrow transplant to reduce treatment side effects

Sickle Cell Disease

Part of Blood & lymphatic, Genetic & congenital clinical trials.

This study tests a way to do a bone marrow (stem cell) transplant from an HLA-matched sibling in children with sickle cell disease, aiming to lower the risk of serious side effects. You may be considered if you have certain past sickle complications and your body can safely handle the transplant plan.

Summary written for real people, not researchers, by Clin2.

Phase
Phase 2
Enrollment
100 people
Ages
2 years to 25 years
Study type
Interventional

Who can take part

  • You have sickle cell disease with hemoglobin SS or Sβ0 thalassemia, or another listed genotype (SC or Sβ+ thalassemia) plus specific complications
  • You have a history of specific serious problems such as stroke or abnormal brain blood-flow tests, frequent acute chest syndrome, severe pain requiring strong IV/opioid medicines, or related hospitalizations
  • You have had significant transfusion needs (for example, many red blood cell transfusions in the last year) or serious splenic complications despite prior care
  • Your infectious diseases are under control (no uncontrolled infection in the month before transplant; HIV and active hepatitis B/C are excluded)
  • Your heart, lungs, liver, and kidneys work well enough for transplant (specific safety thresholds apply)
  • You don’t have certain red blood cell compatibility or immune issues (donor-related antibodies or major blood type mismatch)

View the official record on ClinicalTrials.gov

Quick eligibility check

Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.

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