Clin2
NCT02356653Possibly a fitRecruiting

Stem cell transplant help for children lacking a matching sibling

LeukemiaInborn Errors of MetabolismBone Marrow Failure SyndromesImmunodeficienciesImmunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome

Part of Blood & lymphatic, Cancer, Genetic & congenital, Hormones & metabolism, Immune system & allergy clinical trials.

This expanded-access study offers a specific type of stem cell transplant preparation that removes certain immune cells (CD3 and CD19) from donor stem cells. It is for children who need a transplant but do not have a fully matched sibling donor and whose team cannot use the hospital’s usual matching device protocol.

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

Phase
Early Phase 1
Enrollment
100 people
Ages
Up to 30 years
Study type
Interventional

Who can take part

  • You need a stem cell transplant for a serious blood, immune, or metabolic condition your team recommends
  • You do NOT have a fully HLA-matched sibling donor (a close genetic match)
  • Your child’s care team says the usual CD3/CD19 depletion method at this hospital is not suitable for you
  • A living donor is available who can donate stem cells (related or matched unrelated donor)
  • Your child can join the transplant plan safely: generally good strength/activity (Lansky/Karnofsky 60+), and organ and infection checks meet study/hospital rules
  • If you (or your child) could get pregnant, a pregnancy test must be negative

View the official record on ClinicalTrials.gov

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