Clinical trials: Severe Anemia
Beta Thalassemia Major, Beta Thalassemia Intermedia
Study to Determine the Safety and Tolerability of Sotatercept (ACE-011) in Adults With Beta( β)- Thalassemia.
Dose finding study to determine the safety and tolerability of Sotatercept (ACE-011) in adults with Beta (β)-Thalassemia
- Trial name
- NCT01571635
- Testing
- SOTATERCEPT (ACE-011)
- Where
- 18 sites in France, Greece and 2 more
- Volunteers
- 46 people
- Sponsor
- Celgene
Leukemia, Lymphoma
PS-341 in Treating Patients With Hematologic Cancer
RATIONALE: PS-341 may stop the growth of tumor cells by blocking the enzymes necessary for tumor cell growth. PURPOSE: Phase I trial to study the effectiveness of PS-341 in treating patients who have hematologic cancer.
- Conditions
- Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes
- Trial name
- NCT00006098
- Testing
- bortezomib
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickling Disorder Due to Hemoglobin S
131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)
The purpose of this study is to find the smallest amount of the 131 I-apamistamab needed for preparing patients with severe sickle cell disease (SCD) for a bone marrow transplant.
- Trial name
- NCT07015684
- Testing
- 131I-apamistmab, Sirolimus, Campath
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Columbia University
Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Deferasirox in Treating Iron Overload Caused By Blood Transfusions in Patients With Hematologic Malignancies
RATIONALE: Deferasirox may remove excess iron from the body caused by blood transfusions. PURPOSE: This clinical trial studies deferasirox in treating iron overload caused by blood transfusions in patients with hematologic…
- Conditions
- Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT01273766
- Testing
- deferasirox, laboratory biomarker analysis, enzyme-linked immunosorbent assay
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Wake Forest University Health Sciences
Thalassemia, Iron Overload
Amlodipine in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major
This study aims to investigate the use of amlodipine, a drug that blocks the uptake of calcium into cells, in the prevention and treatment of iron overload in patients with thalassemia major.
- Trial name
- AmloThal (NCT01395199)
- Testing
- Amlodipine
- Where
- Brazil
- Volunteers
- 62 people
- Sponsor
- University of Campinas, Brazil
Hemolytic Uremic Syndrome, Hemorrhagic Colitis
Study of Repetitive Intestinal Lavage in Patients With EHEC Associated Hemorrhagic Colitis
The investigators examined the outcome of patients with severe Enterohaemorrhagic E. Coli (EHEC) O104:H4 infection suffering from bloody diarrhoea that were at risk to develop hemolytic uremic syndrome and underwent repetitive…
- Conditions
- Hemolytic Uremic Syndrome, Hemorrhagic Colitis, Intestinal Infectious Disease, Intestinal Infection Due to E. Coli
- Trial name
- EHEC-PEG (NCT01561248)
- Testing
- polyethylene glycol solution for daily bowel lavage.
- Where
- Germany
- Volunteers
- 33 people
- Sponsor
- Universitätsklinikum Hamburg-Eppendorf
Sickle Cell Disease
Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years…
- Trial name
- NCT04774536
- Testing
- CRISPR_SCD001
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Mark Walters, MD
Beta Thalassemia Major Anemia
Thrombin Generation in Beta-thalassemia Major
This study aims to evaluate the persistence of a hypercoagulable state in chronically-transfused patients with beta thalassemia major, by using the thrombin generation test (performed in whole blood and plasma).
- Trial name
- TG-THAL (NCT04962984)
- Testing
- blood sampling
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Central Hospital, Nancy, France
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia
Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Due to an overall and disease free survival of 85% to 100%, allogeneic blood or bone marrow stem cell transplantation using an HLA matched sibling donor is the therapy of choice for patients with severe aplastic anemia (SAA).
- Conditions
- Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
- Trial name
- NCT00244010
- Testing
- Allogeneic stem cell transplant
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Jude Children's Research Hospital
Aplastic Anemia
A Prospective Study on the Treatment of Recurrent/Refractory/Intolerable NSAA With Lusutrombopag
In a prospective, single-arm study, the efficacy and safety of Lusutrombopag in the treatment of relapsed/refractory/intolerable non-severe aplastic anemia (NSAA) were explored.
- Trial name
- NCT06426043
- Testing
- Lusutrombopag
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Peking Union Medical College Hospital
Transfusion Dependent Beta-thalassemia
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism…
- Trial name
- NCT06606886
- Testing
- Tablet Metformin (Group A), Tablet Hydroxyurea (Group B)
- Where
- Pakistan
- Volunteers
- 96 people
- Sponsor
- Bahria University
Severe Aplastic Anemia
Different Doses of Anti-thymocyte Globin to Treat Child Severe Aplastic Anemia
Severe acquired aplastic anaemia (SAA) is a life-threatening disease characterized by pancytopenia and hypoplastic bone marrow. Immunosuppressive treatment with antithymocyte globulin (ATG)and cyclosporine remain the standard…
- Trial name
- NCT01997372
- Testing
- ATG
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Chinese Academy of Medical Sciences
Thalassemia Major
To Evaluate Long- Term Safety and Efficacy of Luspatercept
An observational study to evaluate long-term safety and efficacy of luspatercept in subjects with transfusion dependent who received the first dose of luspatercept after its introduction in the clinical practice
- Trial name
- LUSPAREAL001 (NCT06875219)
- Testing
- Luspatercept (ACE-536)
- Where
- Italy
- Volunteers
- 350 people
- Sponsor
- Fondazione per la Ricerca sulle Anemie ed Emoglobinopatie in Italia
Sickle Cell Anemia, Hemoglobinopathy
Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies
The major goal of this study is to determine the risks and benefits of bone marrow transplants in patients with severe thalassemia or sickle cell disease. Participation in this project will be for two years.
- Conditions
- Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
- Trial name
- NCT00040469
- Testing
- Campath -1H, Dilantin, Busulfan
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- Baylor College of Medicine
Aplastic Anemia
Phase I/II Study of Anti-CD38 Monoclonal Antibody in Refractory Severe Aplastic Anemia
This is a phase I/II clinical study in adult patients with refractory severe aplastic anemia (SAA). Eligible patients must meet the criteria for refractory SAA and have a platelet count (PLT) <30 × 10^9/L and/or hemoglobin (HGB)…
- Trial name
- NCT07714512
- Testing
- Isatuximab
- Where
- China
- Volunteers
- 32 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Thalassemia Major
Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major
The purpose of this study is to collect peripheral blood and bone marrow aspirate samples from thalassemia patients in Tehran, in a collaborative effort to develop an erythroid lineage specific chimerism assay applicable to…
- Trial name
- NCT00480506
- Where
- 2 sites in United States, Iran
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Dana-Farber Cancer Institute
Hemolytic Disease of the Fetus and Newborn
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (HDFN)
The purpose of this study is to evaluate the safety in mother and neonate/infant of M281 administered to pregnant women who are at high risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN).
- Trial name
- NCT03842189
- Testing
- M281
- Where
- 19 sites in United States, Australia and 7 more
- Volunteers
- 25 people
- Sponsor
- Janssen Research & Development, LLC
Beta Thalassemia Major
Gene Therapy for Beta-Thalassemia Major Using Autologous Hematopoietic Stem Cell Genetically Modified
This is a single group, open label study in 10 subjects who are 8 years of age or older with beta-thalassemia major. The objective of this study is to evaluate the safety and efficacy of autologous hematopoietic stem cell…
- Trial name
- NCT03276455
- Testing
- Autologous CD34+ cells genetically modified
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
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.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
Severe Aplastic Anemia, Relapse
CD7 CAR T Cells (RD13-02) in the Treatment of Relapsed/Refractory Severe Aplastic Anemia
This trial is exploratory research aimed at evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD13-02, a universal CD7 CAR T therapy, in subjects with relapsed/refractory severe aplastic…
- Conditions
- Severe Aplastic Anemia, Relapse, Refractory, CAR T-cell Therapy
- Trial name
- NCT06622694
- Testing
- RD13-02
- Where
- China
- Volunteers
- 15 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China