Condition

Hemoglobinopathies clinical trials and research

• 10Clinical trials
• 6NIH research projects
• 19Countries

Newest Hemoglobinopathies clinical trials

All recruiting trials (10)

• HOPE Biobank: connecting blood disease patients through stem cell transplant and gene therapy samples

  This study will collect blood and other biospecimens along with clinical data from people undergoing stem cell transplant or gene therapy for non-cancer blood diseases to build a large biobank for future research.

  Recruiting at 14 sites Medical College of Wisconsin · Duarte, California and 13 other locations · NCT07227155

• Donor transplant for high risk blood disorders

  This study is testing if a stem cell transplant from a matched sibling can help people with high-risk blood disorders like sickle cell disease and thalassemia feel better.

  Recruiting Phase 2 Masonic Cancer Center, University of Minnesota · Minneapolis, Minnesota · NCT06872333

• Using CD45RA Depleted Stem Cells to Prevent Infections After Transplant

  This study is testing whether using special stem cells can help patients with high-risk leukemia recover their immune system faster and prevent infections after a stem cell transplant.

  Recruiting Phase 1/2 Children's Hospital of Philadelphia · Philadelphia, Pennsylvania · NCT06839456

• Investigating genetic factors in blood disorders

  This study is trying to find out how genes affect blood disorders like sickle cell disease and thalassemia by looking at the DNA of many people with these conditions.

  Recruiting at 17 sites Cyprus Institute of Neurology and Genetics · Boston, Massachusetts and 25 other locations · NCT05799118

• Evaluating a single dose of CTX001 for treating transfusion-dependent beta-thalassemia and severe sickle cell disease

  This study is testing a single dose of a new treatment called CTX001 to see if it can help people with transfusion-dependent beta-thalassemia or severe sickle cell disease feel better.

  Recruiting at 6 sites Phase 3 Vertex Pharmaceuticals Incorporated · New York, New York and 5 other locations · NCT05477563

• Using discarded bone marrow to improve treatments for blood disorders

  This study is trying to see if using leftover bone marrow from patients with blood disorders can help develop better gene therapies and drug treatments for conditions like sickle cell disease and β-thalassemia.

  Recruiting St. Jude Children's Research Hospital · Memphis, Tennessee · NCT04671212

• Cord blood transplant for children and young adults with blood cancers and disorders

  This study is testing a new treatment using chemotherapy followed by a cord blood transplant to see if it can help children and young adults with serious blood cancers and disorders who don't have a matched donor.

  Recruiting Phase 2 Memorial Sloan Kettering Cancer Center · New York, New York · NCT04644016

• Bone marrow transplant for young patients with non-malignant disorders

  This study is testing if a special type of bone marrow transplant is safe and effective for young people with serious non-cancerous blood disorders like sickle cell disease.

  Recruiting at 4 sites Phase 1/2 Washington University School of Medicine · New Haven, Connecticut and 3 other locations · NCT03128996

• Second or greater stem cell transplant using reduced intensity conditioning

  This study is testing a gentler approach to give a second or more stem cell transplant to patients with blood disorders who didn't get enough benefit from their first transplant to see if it helps them recover better.

  Recruiting Masonic Cancer Center, University of Minnesota · Minneapolis, Minnesota · NCT01666080

• Transplant conditioning for non-malignant diseases using immune suppression

  This study is testing a new way to prepare children with non-cancer diseases for a stem cell transplant to see if it helps their bodies accept the donor cells while causing fewer side effects.

  Recruiting Phase 1/2 Washington University School of Medicine · Phoenix, Arizona and 27 other locations · NCT00920972

NIH-funded research (6)

Early-stage science funded by the US National Institutes of Health. Most of these projects don't enroll patients, but they show what is being worked on.

• In utero gene editing for sickle cell disease and alpha-thalassemia using lipid nanoparticles

  A prenatal therapy using specially designed lipid nanoparticles to edit genes in fetal blood-forming cells could prevent sickle cell disease and alpha-thalassemia before symptoms start.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11422868

• Targeting blood stem cells with tiny fat bubbles to treat inherited blood diseases

  A new approach uses targeted lipid nanoparticles to temporarily reduce certain blood-forming stem cells, paving the way for safer stem cell transplants for conditions like sickle cell and thalassemia.

  NIH research Children's Hosp of Philadelphia · Philadelphia, United States · NIH-11422866

• In vivo gene therapy for blood diseases using multiplexed virus-like particles

  A new approach aims to repair patients’ own blood stem cells inside their bodies to treat genetic blood diseases.

  NIH research Fred Hutchinson Cancer Center · Seattle, United States · NIH-11364773

• Oral protein-targeting drugs to raise fetal hemoglobin for sickle cell disease

  Testing new oral medicines that remove proteins blocking fetal hemoglobin to help adults with sickle cell disease reduce red blood cell sickling.

  NIH research Yale University · New Haven, United States · NIH-11252565

• Developing new treatments to increase fetal hemoglobin for sickle cell disease

  This study is working on new medications that can help increase a type of hemoglobin in your blood to better manage Sickle Cell Disease, aiming to provide a more reliable and safer treatment option than what’s currently available.

  NIH research Yale University · New Haven, United States · NIH-11019948

• Using nurses to provide genetic counseling for sickle cell disease in primary care.

  This study is looking at how training nurses to provide genetic counseling and carrier screening for sickle cell disease can help families in Sierra Leone make informed choices about having children, especially since there aren't many genetic counselors available in the area.

  NIH research University of South Florida · Tampa, United States · NIH-10666605

Related topics

Quick answers

How many Hemoglobinopathies clinical trials are recruiting right now?

10, as of November 12, 2026. We update this list each time we refresh our data from ClinicalTrials.gov.

Where are these trials running?

In 19 countries. The most sites are in United States (10 trials), Italy (2 trials), Angola (1 trial), Argentina (1 trial), Belgium (1 trial). Each study page lists its sites and which ones are recruiting.

What phases are they in?

Phase 1/2: 3, Phase 2: 2, Phase 3: 1. Trials with no phase listed are usually device, behavioural or observational studies.

How do I join one?

Open a study, check the "Is this trial a fit for you?" section, then contact the study team listed on the page. They will ask a few screening questions. Asking is free and does not commit you to anything. Talk it over with your doctor first.

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