Clinical trials: Transfusions, open to volunteers
α-thalassemia
the Safety and Efficacy Evaluation of HGI-002 Injection in Patients With Transfusion-Dependent α-Thalassemia
This is an open label study to evaluate the safety and efficacy of α-globin Restored Autologous Hematopoietic Stem Cells in α-Thalassemia Major Patients
- Trial name
- NCT05851105
- Testing
- α-globin restored autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Shenzhen Hemogen
Sickle Cell Disease
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.
- Trial name
- NCT07656415
- Testing
- Mitapivat
- Where
- 5 sites in United States
- Volunteers
- 159 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Thalassemia
A Study of SNH-119014 in Adult Participants With Non-transfusion-dependent Thalassemia (NTDT)
This study is a multicenter study to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of treatment with SNH-119014 in adult participants with non-transfusion-dependent thalassemia.
- Trial name
- NCT07579949
- Testing
- SNH-119014 dose 1, SNH-119014 dose 2
- Where
- 8 sites in China
- Volunteers
- 30 people
- Sponsor
- ScinnoHub Pharmaceutical Co., Ltd.
β-thalassemia
A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector…
- Trial name
- NCT06465550
- Testing
- BD211
- Where
- 3 sites in China
- Volunteers
- 9 people
- Sponsor
- Shanghai BDgene Co., Ltd.
Thalassemia, Beta
Long-term Follow-up Study of BHC001 for TDT
Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.
- Trial name
- NCT06314529
- Testing
- Assessments
- Where
- 2 sites in China
- Volunteers
- 6 people
- Sponsor
- Bioray Laboratories
Sickle Cell Anaemia, Sickle Cell Anemia Crisis
Hypoxic Red Blood Cells in Sickle Cell Anemia
The overall objective of this study is to evaluate the effectiveness and safety of transfusing hypoxic red blood cells manufactured with the Hemanext ONE system in patients with sickle cell anemia.
- Conditions
- Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
- Trial name
- NCT06743113
- Testing
- Hemanext ONE System, Conventional RBCs
- Where
- 5 sites in United States
- Volunteers
- 48 people
- Sponsor
- Hemanext
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
Myelodysplastic Syndromes, Anemia
A Study to Evaluate Ofirnoflast in Adults With Very Low- to Intermediate-risk Myelodysplastic Syndromes Requiring Red Blood Cell Transfusions
The primary objective of this study is to two part. First, dose selection for dose optimization based on safety of ofirnoflast. Second, to evaluate the efficacy of ofirnoflast administered in the study population.
- Trial name
- NCT07738510
- Testing
- Ofirnoflast
- Volunteers
- 70 people
- Sponsor
- Halia Therapeutics, Inc.
Bone Marrow Failure Syndromes, Aplastic Anemias
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
This is a multicenter, open-label, Phase 1/2 basket study to evaluate the safety and efficacy of Dibotatug (DR-01) in adults with Bone Marrow Failure syndromes.
- Conditions
- Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse
- Trial name
- BMF (NCT07712562)
- Testing
- Dibotatug (DR-01)
- Where
- 10 sites in United States
- Volunteers
- 60 people
- Sponsor
- Dren Bio
Transfusion Reaction, Hemolytic
Challenge Transfusion of INTERCEPT Pathogen Reduced Red Blood Cells (RBCs) in Subjects With or Without Pre-existing Antibodies to INTERCEPT RBCs
This study aims to determine whether transfusion of INTERCEPT RBCs into patients with pre-existing antibodies to INTERCEPT RBCs will result in increased antibody titer indicative of a secondary immune response, and whether these…
- Trial name
- Challenge (NCT07152379)
- Testing
- 30-40 mL of INTERCEPT RBCs, ~30 mL of INTERCEPT RBCs plus plus 10 mL biotinylated INTERCEPT RBCs
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Cerus Corporation
Aplastic Anemia
CK0801 for Treatment of Aplastic Anemia
This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care…
- Trial name
- NCT07499102
- Testing
- CK0801
- Volunteers
- 12 people
- Sponsor
- Cellenkos, Inc.
Primary Myelofibrosis, Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
The purpose of this Phase 2 study is to evaluate the efficacy and safety of momelotinib (MMB) in combination with luspatercept (LUSPA) in participants with transfusion dependence (TD) primary myelofibrosis (PMF) or…
- Trial name
- ODYSSEY (NCT06517875)
- Testing
- Momelotinib, Luspatercept
- Where
- 33 sites in United States, Canada and 4 more
- Volunteers
- 68 people
- Sponsor
- GlaxoSmithKline
Beta-Thalassemia, Thalassemia
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD).
- Conditions
- Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn
- Trial name
- NCT05477563
- Testing
- CTX001
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 26 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia
A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301
The purpose of this study is to evaluate the long-term safety and efficacy of EDIT-301 in participants with severe sickle cell disease (SCD) or transfusion-dependent β-thalassemia (TDT) who have received EDIT-301.
- Conditions
- Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
- Trial name
- NCT06363760
- Testing
- Safety and efficacy assessments
- Where
- 18 sites in United States, Canada
- Volunteers
- 54 people
- Sponsor
- Editas Medicine, Inc.
β -Thalassemia
A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia
This is a phase II, randomized, double-blinded, placebo-controlled study to treat patients with transfusion-dependent and non-transfusion dependent β -thalassemia with AND017 and optimal supportive care, including blood…
- Trial name
- NCT06302491
- Testing
- AND017 capsules
- Where
- 5 sites in China
- Volunteers
- 64 people
- Sponsor
- Kind Pharmaceuticals LLC
Myelodysplastic Syndrome, Anemia
A Study to Compare Elritercept With Epoetin Alfa to Treat Anemia in Adults With Lower Risk Myelodysplastic Syndromes (LR-MDS) Who Need Regular Blood Transfusions
The main aim of this study is to assess how much elritercept reduces the need for RBC (red blood cell) transfusions and improves hemoglobin blood values in addition to how safe elritercept is when compared with epoetin alfa.
- Trial name
- ELRiSE MDS (NCT07422480)
- Testing
- Elritercept, Epoetin Alfa
- Where
- 162 sites in United States, Argentina and 26 more
- Volunteers
- 300 people
- Sponsor
- Takeda
Platelet Transfusion Refractoriness
Study of CM313(SC) Injection in Subjects With Platelet Transfusion Refractoriness
This is a randomized, open-label, phase IB/II clinical study to evaluate the safety and preliminary efficacy of CM313(SC) injection in patients with Platelet Transfusion Refractoriness.
- Trial name
- NCT06792019
- Testing
- CM313(SC) injection-low dose, CM313(SC) injection-high dose
- Where
- China
- Volunteers
- 110 people
- Sponsor
- Keymed Biosciences Co.Ltd
Myelodysplastic Syndromes, Cytopenia
A Study of Elritercept to Treat Anemia in Adults With Very Low, Low, or Intermediate Risk Myelodysplastic Syndromes (MDS)
The main aim of this study is to learn how safe elritercept is and how well adults with anemia associated with lower-risk MDS tolerate treatment with different doses of elritercept.
- Trial name
- NCT04419649
- Testing
- Elritercept
- Where
- 53 sites in United States, Australia and 6 more
- Volunteers
- 210 people
- Sponsor
- Takeda
β-thalassemia
the Safety and Efficacy Evaluation of HGI-001 Injection in Patients With Transfusion-Dependent β-Thalassemia
This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients
- Trial name
- NCT05864170
- Testing
- β-globin restored autologous hematopoietic stem cells
- Where
- China
- Volunteers
- 3 people
- Sponsor
- Shenzhen Hemogen
Thromboembolic Event, FDP
Safety And Field Evaluation of Plasma Lyophilized for Acute and Surgical Management Applications
The purpose of this study is to compare the safety of freeze-dried plasma (FDP), known as EZPLAZ, with conventional plasma in adults who need a plasma transfusion.
- Trial name
- SAFE-PLASMA (NCT07840352)
- Testing
- Freeze-Dried Plasma, Conventional Plasma
- Volunteers
- 867 people
- Sponsor
- Teleflex