Clinical trials: Severe Anaemia
Thalassemia
Endocrine and Nutritional Assessment in B Thalassemia Major
B Thalassemia patients developed short stature and low weight in spite no evident endocrine abnormalities. One hypothesis is that they developed some degree of malnutrition,then the purpose of this study is to assess the…
- Trial name
- NCT00456690
- Testing
- Nutritional recommendations
- Where
- Israel
- Volunteers
- 133 people
- Sponsor
- HaEmek Medical Center, Israel
Anemia
RBC Transfusion in Severe Anemia With Lactic Acidosis
The hypothesis of this study is that prolonged-storage RBCs are not inferior to short-storage RBCs for the time required to clear elevated blood lactate levels in children with severe anemia.
- Trial name
- TOTAL (NCT01586923)
- Testing
- RBC transfusion
- Where
- Uganda
- Volunteers
- 290 people
- Sponsor
- Massachusetts General Hospital
Severe Aplastic Anemia, Hypo-Plastic MDS
Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
Background: Severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) are bone marrow diseases. People with these diseases usually need a bone marrow transplant.
- Conditions
- Severe Aplastic Anemia, Hypo-Plastic MDS, Myelodysplastic Syndrome (MDS)
- Trial name
- NCT03173937
- Testing
- Omidubicel (former CordIn), Miltenyi CliniMACS(R) CD34 Reagent System
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
GVHD Prophylaxis by Addition of CD20 Monoclonal Antibody to the Conditioning Regimen in SAA With Treatment of Allo-HSCT
Objectives 2.1 Primary objectives 1) To observe and compare incidence and severity of aGVHD and cGVHD between the two arms within 2 years after transplantation. 2) To observe and compare the engraftment rate between the two arms.
- Trial name
- NCT05510505
- Testing
- CD20 monoclonal antibody, ATG
- Where
- China
- Volunteers
- 100 people
- Sponsor
- The First Affiliated Hospital of Soochow University
Severe Aplastic Anemia (SAA)
Extended Dosing With Eltrombopag for Severe Aplastic Anemia
Background: Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients.
- Trial name
- NCT01891994
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Beta Thalassemia Major
Effect of Spirulina Compared to Amlodipine on Cardiac Iron Overload in Children With Beta Thalassemia
the aim of this study is to evaluate the effect of Spirulina compared to Amlodipine on cardiac iron overload and cardiac functions in multi-transfused children with beta thalassemia major
- Trial name
- NCT02671695
- Testing
- Spirulina, Amlodipine
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Tanta University
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Acute Leukemia, Myelodysplastic Syndromes
Sirolimus+Ruxolitinib+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to CNI
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled.
- Conditions
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia (SAA)
- Trial name
- NCT07796620
- Testing
- Sirolimus, Ruxolitinib, MMF
- Volunteers
- 40 people
- Sponsor
- Peking University People's Hospital
Fetal Hemoglobin, Thalassemia Major
Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia
The project "Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia" investigates the safety and efficacy of low-dose thalidomide in managing beta thalassemia, a genetic disorder causing…
- Conditions
- Fetal Hemoglobin, Thalassemia Major, Genetic Disease
- Trial name
- BTM (NCT06490627)
- Testing
- Thalidomide
- Where
- Pakistan
- Volunteers
- 54 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Immune Abnormalities
Immunomodulatory Effects of Silymarin in Patients With Beta-Thalassemia Major
A wide spectrum of immune abnormalities has been described by numerous studies involving β-thalassemic patients with multiple transfusions.
- Trial name
- NCT01752153
- Testing
- Desferrioxamine, Legalon® (Silymarin), Silymarin (Legalon)
- Where
- Iran
- Volunteers
- 25 people
- Sponsor
- Shiraz University of Medical Sciences
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia.
- Trial name
- NCT01174108
- Testing
- Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC
- Where
- 2 sites in United States
- Volunteers
- 120 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Alpha-Thalassemia, Alpha Thalassemia Major
International Registry of Patients With Alpha Thalassemia
This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of…
- Conditions
- Alpha-Thalassemia, Alpha Thalassemia Major, Alpha Thalassemia Minor
- Trial name
- ATM Registry (NCT04872179)
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- University of California, San Francisco
Acute Leukemia, Myelodysplastic Syndromes
Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors
Graft-versus-host disease (GVHD) is an important complication after transplantation, with an incidence of 40-60%, which can increase non-relapse mortality if poorly controlled.
- Conditions
- Acute Leukemia, Myelodysplastic Syndromes, Severe Aplastic Anemia
- Trial name
- NCT06279494
- Testing
- Sirolimus, Abatacept, MMF
- Volunteers
- 6 people
- Sponsor
- Peking University People's Hospital
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson
Hepatitis-associated Aplastic Anemia, Severe Aplastic Anemia(SAA)
Clinical Study of UC-MSC Combined With Immunosuppressive Agents in the Treatment of Pediatric HAAA
The goal of this clinical trial is to evaluate the effectiveness of umbilical cord mesenchymal stem cells combined with standard immunosuppressive therapy in children with HAAA, and to provide a new therapeutic idea for children…
- Conditions
- Hepatitis-associated Aplastic Anemia, Severe Aplastic Anemia(SAA), Umbilical Cord Blood, Mesenchymal Stem Cells
- Trial name
- NCT07850362
- Testing
- UC-MSC
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
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
Compare Blood Age for Transfusion
Effect of Blood Storage Age on the Resolution of Lactic Acidosis in Children With Severe Malarial Anemia at Mulago Hospital
In resolving lactic acidosis among children with severe malarial anemia, there is no difference between those transfused with blood of longer storage compared to shorter storage age
- Trial name
- NCT01580111
- Testing
- Blood transfusion
- Where
- Uganda
- Volunteers
- 74 people
- Sponsor
- Makerere University
Sickle Cell Crisis, Acute Pain
Low-dose Ketamine vs Morphine for Vaso-occlusive Crisis in Sicklers
This clinical trial will inform of the role of Low dose ketamine in the acute treatment of severe painful sickle cell crisis in children in a day-case sickle cell centre.
- Trial name
- KEM-VOC (NCT02434939)
- Testing
- Low dose ketamine, Morphine
- Where
- Uganda
- Volunteers
- 240 people
- Sponsor
- Makerere University
Aplastic Anaemia
Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF)
The purpose of this study is to examine the effect of G-CSF on disease free survival and overall survival in aplastic anaemia patients who also receive ATG and Cyclosporin A.
- Trial name
- SAA-G-CSF (NCT01163942)
- Testing
- G-CSF, Early retreatment with ATG
- Where
- 71 sites in Czechia, France and 7 more
- Volunteers
- 205 people
- Sponsor
- European Society for Blood and Marrow Transplantation