Plain-English translation of NCT02140554 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle cell disease 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 tested a new gene therapy called lovo-cel for people with severe sickle cell disease. The treatment involved collecting stem cells from a patient's bone marrow, modifying them in the laboratory to produce healthy hemoglobin, and then infusing the modified cells back into the patient. Researchers studied whether this medication could reduce the severe pain crises and other complications that people with sickle cell disease experience.
Sickle cell disease causes severe pain crises and organ damage that are difficult to manage with current medications. This treatment was designed to address the root cause of the disease by fixing the genetic defect that causes red blood cells to become sickle-shaped, potentially offering a long-lasting solution for people who don't respond well to or cannot tolerate existing therapies.
Participants had their bone marrow harvested to collect stem cells, which were then modified in the laboratory and infused back into their body. After the infusion, participants were monitored closely for 2 years post-transplant, with follow-up visits to assess safety and how well the treatment worked. Those who completed the initial study were invited to participate in a long-term follow-up program that continued for up to 15 years total to track lasting effects of the treatment.
AI-generated summary from trial data · Sep 24, 2026 · Not medical advice
United States