Sickle cell-hemoglobin E disease syndrome
MONDO:0016671A rare, genetic hemoglobinopathy usually characterized by mild microcytic hemolysis and, very rarely, vaso-occlusive complications. Severe manifestations have been reported, including hematuria, splenic infarction, acute chest syndrome, acute episodes of pain and reversible bone marrow necrosis. The genotype is characterized by an HbS allele in combination with an HbE variant (beta26glu>lys); symptoms are due to the low allelic expression of HbE leading to HbS predominance (65+/-5%).
Also known as: HbSE disease
13 clinical trials for this condition and its sub-types, 0 tagged with Sickle cell-hemoglobin E disease syndrome itself.
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Milder chemo may make stem cell transplants safer for Non-Cancer patients
Disease control Recruiting nowThis phase 2 trial tests a combination of three chemotherapy drugs (treosulfan, fludarabine, thiotepa) plus an immune-suppressing antibody before a donor stem cell transplant in up to 40 people under 50 with non-cancerous blood disorders. The goal is to see if this milder conditi…
Phase 2 • Sponsor: Fred Hutchinson Cancer Center • Aim: Disease control
Last updated Sep 18, 2026 00:00 UTC
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New hope for kids: drug may prevent transplant complications
Disease control Recruiting nowThis study tests whether adding the drug vorinostat to standard care can prevent graft-versus-host disease (GVHD) in children and young adults (ages 1-26) with non-cancerous blood disorders who are getting a bone marrow transplant. GVHD is a serious complication where donor cells…
Phase 2 • Sponsor: Sung Won Choi • Aim: Disease control
Last updated Sep 02, 2026 00:00 UTC
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Second chance: milder prep for stem cell transplants may help when first fails
Disease control Recruiting nowThis trial tests a second stem cell transplant using a gentler conditioning regimen of busulfan, fludarabine, and low-dose total body irradiation. It is for people with blood disorders, hemoglobin diseases, or immune deficiencies whose first transplant did not achieve enough dono…
Sponsor: Masonic Cancer Center, University of Minnesota • Aim: Disease control
Last updated Jul 08, 2026 00:00 UTC
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New bone marrow transplant trial offers hope for kids with severe blood disorders
Disease control Recruiting nowThis study tests a bone marrow transplant from a family donor for children under 21 with severe non-cancer blood disorders like sickle cell disease, bone marrow failure, or immune problems. The goal is to see if the transplant can replace the diseased cells with healthy donor cel…
Phase 1/2 • Sponsor: Washington University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 13:00 UTC
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New stem cell transplant option for patients without a perfect donor match
Disease control Recruiting nowThis expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic disea…
Early phase 1 • Sponsor: Children's Hospital of Philadelphia • Aim: Disease control
Last updated Jun 27, 2026 09:07 UTC
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Tracking the long road after gene therapy and transplant for blood disorders
Knowledge-focused Recruiting nowResearchers at St. Jude Children's Research Hospital are following people with hemoglobin disorders who have received a stem cell transplant or gene therapy. The study enrolls up to 200 participants who had or plan to have one of these treatments within 15 years. It collects rout…
Sponsor: St. Jude Children's Research Hospital • Aim: Knowledge-focused
Last updated Sep 17, 2026 00:00 UTC
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NIH launches Long-Term study to track stem cell transplant survivors
Knowledge-focused Recruiting nowThis study provides ongoing check-ups for people who received a donor stem cell transplant at the NIH at least three years ago. Researchers will monitor for late side effects, disease return, and overall health. Participants will have yearly visits that may include blood tests, b…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Sep 03, 2026 00:00 UTC
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How long do red blood cells live? new study uses biotin to find out
Knowledge-focused Recruiting nowThis study aims to measure how long red blood cells survive in people with sickle cell disease, thalassemia, and other inherited blood disorders. Researchers will take a blood sample, label the red cells with biotin (a vitamin), and infuse them back into the participant. Over up …
Early phase 1 • Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Sep 02, 2026 00:00 UTC