Clinical trials: Severe Anemia
Aplastic Anemia
Study of Fludarabine + Cyclophosphamide + TBI Conditioning Regimen for Double Units Cord Blood Transplantation(CBT)in Severe Aplastic Anemia(SAA)
Severe aplastic anemia is a fatal disease and patients without HLA matched siblings need alternative treatment option. Cord blood transplantation (CBT) has become an alternative treatment means in various diseases, but it has not…
- Trial name
- NCT00881933
- Testing
- Cyclophosphosphamide, TBI
- Where
- South Korea
- Volunteers
- 5 people
- Sponsor
- The Korean Society of Pediatric Hematology Oncology
Transfusion Dependent Beta-thalassemia
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age.
- Trial name
- NCT03432364
- Testing
- ST-400 Investigational product
- Where
- 6 sites in United States
- Volunteers
- 5 people
- Sponsor
- Sangamo Therapeutics
Transfusion-dependent Non-severe Aplastic Anemia
Enarodustat + CsA vs CsA in Newly Diagnosed TD-NSAA
This study aimed to compare the efficacy and safety of enarodustat combined with cyclosporine versus cyclosporine monotherapy in the treatment of newly-diagnosed tansfusion-dependent non-severe aplastic anemia (TD-NSAA).
- Trial name
- NCT07548671
- Testing
- Enarodustat, Cyclosporine (CsA)
- Volunteers
- 90 people
- Sponsor
- Peking Union Medical College Hospital
Sickle Cell Disease
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features.
- Trial name
- STRIDE (NCT01565616)
- Testing
- Conditioning Regimen with Bone Marrow Transplant
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Emory University
Thrombocytopenia
Managed Access Programs for ETB115, Eltrombopag
The purpose of this registration is to list Managed Access Programs (MAPs) related to ETB115, Eltrombopag
- Trial name
- NCT04837703
- Testing
- Eltrombopag
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia
UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia
This Phase II open-label interventional clinical trial aims to evaluate the efficacy of romiplostim, in patients with severe aplastic anemia (SAA), both treatment naïve and relapsed/refractory, in inducing trilineage…
- Trial name
- NCT07001254
- Testing
- Romiplostim, Immunosuppressive therapy (IST)
- Where
- United States
- Sponsor
- Anjali Sharathkumar
Severe Aplastic Anemia
A Study of PEG-rhG-CSF and rhG-CSF Used for Aplastic Anemia Granulocyte Deficiency
This study was a single-center,open-label,randomized,dose-exploring prospective study.Patients with granulocytotic aplastic anemia who received cytokine treatment with PEG-rhG-CSF or rhG-CSF were enrolled.Clinical demographic…
- Trial name
- NCT05531279
- Testing
- PEG-rhG-CSF
- Where
- China
- Volunteers
- 45 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Tacrolimus and Mycophenolate Mofetil in Preventing Acute Graft-Versus-Host Disease in Patients With Advanced Hematologic Cancer Who Are Undergoing Donor Stem Cell Transplantation
RATIONALE: Tacrolimus and mycophenolate mofetil may be an effective treatment for graft-versus-host disease caused by donor stem cell transplantation.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00096096
- Testing
- mycophenolate mofetil, tacrolimus
- Where
- United States
- Sponsor
- Fred Hutchinson Cancer Center
Sickle Cell Disease
HLA-Identical Sibling Donor Bone Marrow Transplantation for Individuals With Severe Sickle Cell Disease Using a Reduced Intensity Conditioning Regimen
Sickle cell disease (SCD) is the most common inherited blood disorder in Saudi Arabia . Its clinical severity is widely heterogeneous among patients who share the same genetic mutation .
- Trial name
- NCT02776202
- Testing
- Fludarabine monophosphate
- Where
- Saudi Arabia
- Volunteers
- 15 people
- Sponsor
- National Guard Health Affairs
Beta-thalassemia Major, Hemosiderosis
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available.
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Trial name
- ESCALATOR (NCT00171301)
- Testing
- Deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 233 people
- Sponsor
- Novartis Pharmaceuticals
Thalassemia Major (TM), Thalassemia Intermedia (TI)
Beta-thalassemia and Microparticles
The results will allow us to evaluate the role of MP in the thrombo-embolic risk observed in thalassemic patients and to underline a possible difference between TM and TI.
- Conditions
- Thalassemia Major (TM), Thalassemia Intermedia (TI), Microparticles (MP)Originating From Platelets, Endothelial Cells and Monocytes
- Trial name
- NCT01284738
- Testing
- Physiopathology
- Where
- France
- Volunteers
- 33 people
- Sponsor
- Assistance Publique Hopitaux De Marseille
Sickle Cell Disease, Hematological Diseases
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participants with severe sickle cell disease (SCD).
- Conditions
- Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
- Trial name
- NCT03745287
- Testing
- Exa-cel
- Where
- 17 sites in United States, Belgium and 5 more
- Volunteers
- 63 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sever Aplastic Aneia, Children
A Randomized, Controlled Clinical Study on the Efficacy and Safety of Umbilical Cord Mesenchymal Stem Cells Combined With Standard Immunosuppressants in the Treatment of Children With Severe Aplastic Anemia
Study on the efficacy and safety of umbilical cord mesenchymal stem cells combined with standard immunosuppressive agents in the treatment of children with acquired severe aplastic anemia
- Conditions
- Sever Aplastic Aneia, Children, Umbilical Cord Mesenchymal Stem Cells
- Trial name
- SAA MSC (NCT07903649)
- Testing
- Umbilical Cord Mesenchymal Stem Cells with ATG/ALG, ATG/ALG
- Where
- China
- Volunteers
- 64 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Sickle Cell Disease
Stem Cell Transplant in Patients With Severe Sickle Cell Disease
This is a prospective pilot study of matched-related donor allogeneic stem cell transplantation in adults with severe sickle cell disease using a matched-sibling PBSC graft with a non-myeloablative conditioning regimen…
- Trial name
- NCT03421756
- Testing
- Alemtuzumab, Total Body Irradiation, Sirolimus
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Kathleen Dorritie
Sickle Cell Disease
Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease
This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease.
- Trial name
- Restore (NCT04819841)
- Testing
- nula-cel Drug Product
- Where
- 6 sites in United States
- Volunteers
- 15 people
- Sponsor
- Kamau Therapeutics
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
This is a study to collect the outcomes of stem cell transplantation for patients with hematologic diseases other than cancer.
- Conditions
- Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT02179359
- Testing
- Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Bloodstream Infection, Salmonella Bacteremia
Clinical Decision Support in Non-typhoidal Salmonella Bloodstream Infections in Children
In sub-Saharan Africa, non-typhoidal Salmonella (NTS) are a frequent cause of bloodstream infection, display high levels of antibiotic resistance and have a high case fatality rate (15%).
- Conditions
- Bloodstream Infection, Salmonella Bacteremia, Malaria,Falciparum, Severe Malaria
- Trial name
- DeNTS (NCT04473768)
- Where
- Belgium
- Volunteers
- 1,880 people
- Sponsor
- Institute of Tropical Medicine, Belgium
Lymphoma, Leukemia
Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation
RATIONALE: Following stem cell transplantation, a major risk is graft-versus-host disease (GVHD). This occurs when donor immune cells that have been infused recognise the host's cells as 'foreign' and attack these cells.
- Conditions
- Lymphoma, Leukemia, Myeloma, Myelodysplastic Syndromes
- Trial name
- ToTem (NCT03836690)
- Testing
- CD62L- Tem
- Where
- United Kingdom
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- University College, London
Beta-Thalassemia
Evaluating the Safety of G-CSF Mobilization in Individuals With Beta Thalassemia Major
Beta thalassemia major is a serious genetic disease of the blood. Treatments are limited, and although a bone marrow transplant from a compatible donor can be curative, only a limited percentage of individuals with this disease…
- Trial name
- NCT00336362
- Testing
- Hydroxyurea
- Where
- Greece
- Volunteers
- 26 people
- Sponsor
- University of Washington
Anaemia
Preoperative Intravenous Iron to Treat Anaemia in Major Surgery
Anaemia is a common problem in patients undergoing surgery. About half of patients undergoing a major operation have anaemia, often a consequence of the disease requiring surgery. Anaemia causes patients to feel tired and unwell.
- Trial name
- PREVENTT (NCT01692418)
- Testing
- Ferric carboxymaltose, Normal saline
- Where
- 28 sites in United Kingdom
- Volunteers
- 487 people
- Sponsor
- University College, London