Plain-English translation of NCT01590628 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Thalassemia research guide →Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial studied ®, a treatment made from stem cells collected from umbilical cord blood that were grown and multiplied in the laboratory. Researchers tested whether this expanded cell product could be safely transplanted into patients with severe sickle cell disease or thalassemia major to help their bodies produce healthy blood cells and improve their symptoms.
Umbilical cord blood contains valuable stem cells that can treat life-threatening blood disorders, but there often isn't enough of these cells in a single donation to work well. This medication was designed to solve that problem by multiplying the cells in the laboratory, potentially making cord blood transplants more effective and available to more patients.
Participants received chemotherapy to prepare their body for the transplant, then received an infusion of the ® stem cells (with or without a second umbilical cord blood unit, depending on which part of the trial they were in). They were followed closely by the research team for approximately 180 days after the transplant to monitor how well the new cells were working and to watch for any side effects or complications.
AI-generated summary from trial data · Oct 12, 2026 · Not medical advice
United States