Sickle Cell Disease Clinical Trials in Boston, Massachusetts

9 studies are recruiting for sickle cell disease in Boston, Massachusetts right now. Each one is listed on ClinicalTrials.gov, the public registry the research teams themselves file with. TrialBoost is not a doctor, a research site, or a study sponsor: the research team decides who takes part.

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Studies now recruiting

1. A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

   Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking i…

   Stage

   Phase 3

   Ages

   2 and older

   Site

   Boston Medical Center, Boston, Massachusetts

   Locations

   107 in total

   Run by

   Novo Nordisk A/S

   See if you can join NCT06609226 on ClinicalTrials.gov

2. A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

   This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can red…

   Stage

   Phase 3

   Ages

   12 and older

   Site

   Boston Children's Hospital, Boston, Massachusetts

   Locations

   175 in total

   Run by

   Novo Nordisk A/S

   See if you can join NCT06612268 on ClinicalTrials.gov

3. A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

   The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants …

   Stage

   Phase 1

   Ages

   12 to 55

   Site

   Boston Childrens Hospital, Boston, Massachusetts

   Locations

   10 in total

   Run by

   Novartis Pharmaceuticals

   See if you can join NCT06546670 on ClinicalTrials.gov

4. ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

   In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly. Some of the recently FDA-approved therapies for congenital and acquired hematologic conditions have not yet demonstrated long-term safety and effectiveness beyond…

   Stage

   Not phased

   Ages

   Any age

   Site

   Massachusetts General Hospital Comprehensive Hemophilia and Thrombosis Treatment Center, Boston, Massachusetts

   Locations

   76 in total

   Run by

   American Thrombosis and Hemostasis Network

   See if you can join NCT04398628 on ClinicalTrials.gov

5. Promoting Resilience Among Adolescents and Young Adults With Sickle Cell Disease

   Adolescents and young adults with sickle cell disease (SCD) face challenges managing their illness and maintaining their well-being. This study proposes to test the feasibility and acceptability of a resilience-promoting intervention through a Collaborative Care Model. The primary goal is to determ…

   Stage

   Not phased

   Ages

   8 to 25

   Site

   Boston Children's Hospital, Boston, Massachusetts

   Run by

   Boston Children's Hospital

   See if you can join NCT06555939 on ClinicalTrials.gov

6. Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

   A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order …

   Stage

   Phase 1

   Ages

   13 to 40

   Site

   Boston Children's Hospital, Boston, Massachusetts

   Run by

   Daniel Bauer

   See if you can join NCT06647979 on ClinicalTrials.gov

7. A Long-term Follow-up Study in Patients Who Received BEAM-101

   This is a Long-term Follow-up (LTFU) study in patients who received BEAM-101 in the parent study (Study BTX-AUT-001). Eligible patients who received BEAM-101 will be asked to participate in this LTFU study prior to completing Study BTX-AUT-001. Patients who enter into this study will be followed fo…

   Stage

   Not phased

   Ages

   14 to 37

   Site

   Boston Children's Hospital, Boston, Massachusetts

   Locations

   17 in total

   Run by

   Beam Therapeutics Inc.

   See if you can join NCT07373639 on ClinicalTrials.gov

8. A Pilot Study on Neuroimaging in SCD: Part of The Boston Consortium to Cure Sickle Cell Disease

   Sickle Cell Disease (SCD) impairs oxygen transport to tissue and causes endothelial injury. Thus, therapeutic interventions aim to improve both, but there is an unmet need for biomarkers to determine when intervention is necessary and evaluate the effectiveness of the chosen intervention in individ…

   Stage

   Not phased

   Ages

   8 to 18

   Site

   Boston Children's Hospital, Boston, Massachusetts

   Run by

   Boston Children's Hospital

   See if you can join NCT04166526 on ClinicalTrials.gov

9. Study of the Role of Genetic Modifiers in Hemoglobinopathies

   This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).

   Stage

   Not phased

   Ages

   2 and older

   Site

   Boston Children's Hospital, Boston, Massachusetts

   Locations

   26 in total

   Run by

   Cyprus Institute of Neurology and Genetics

   See if you can join NCT05799118 on ClinicalTrials.gov