Sickle cell-hemoglobin c disease syndrome
MONDO:0016669A rare, genetic hemoglobinopathy characterized by anemia, reticulocytosis and erythrocyte abnormalities including target cells, irreversibly sickled cells and crystal-containing cells. Clinical course is similar to sickle cell disease, but less severe and with less complications. Signs and symptoms may include acute episodes of pain, splenic infarction and splenic sequestration crisis, acute chest syndrome, focal segmental glomerulosclerosis, ischemic brain injury, peripheral retinopathy, and osteonecrosis.
Also known as: HbSC disease, sickle cell - haemoglobin C disease, sickle cell - hemoglobin C disease
19 clinical trials for this condition and its sub-types.
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Sickle cell gene therapy trial pulled before it even started
Disease control TerminatedThis study was designed to test a single dose of exa-cel, a gene-edited cell therapy, in people with severe sickle cell disease who have the HbSC genotype. Participants would have received their own modified stem cells after chemotherapy. However, the trial was withdrawn before a…
Phase: PHASE3 • Sponsor: Vertex Pharmaceuticals Incorporated • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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New cord blood matching method could expand transplant access for blood cancer patients
Disease control OngoingThis study tests a new approach to stem cell transplants for people with blood cancers like leukemia and lymphoma. Patients receive a combination of umbilical cord blood and stem cells from a family member, with the cord blood chosen based on specific genetic markers rather than …
Phase: PHASE2 • Sponsor: Weill Medical College of Cornell University • Aim: Disease control
Last updated Jun 26, 2026 13:02 UTC
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Tiny study probes Mitapivat's inner workings in sickle cell
Knowledge-focused OngoingThis observational study will examine how the drug mitapivat changes red blood cells in 6 people with sickle cell disease. Researchers will measure oxygen binding and cell survival markers from blood samples and muscle oxygen tests. The goal is to understand the drug's effects, n…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Aug 15, 2026 00:00 UTC