Clinical trials: Bone Marrow Aplasia
Aplastic Anemia
Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia
The objective of this study is to evaluate the efficacy of romiplostim administered once weekly to Aplastic Anemia (AA) patients with thrombocytopenia refractory to or ineligible for immunosuppressive therapy in Japan and Korea.
- Trial name
- NCT02773290
- Testing
- Romiplostim
- Where
- 3 sites in Japan, South Korea
- Volunteers
- 46 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Childhood Cancer, Aplastic Anemia
Trial of Two Central Venous Catheter (CVC) Flushing Schemes in Pediatric Hematology and Oncology Patients
The purpose of this study is to determine whether flushing Central Venous Catheters (CVCs) with Normal saline once per week is not inferior to flushing with 10U/ml heparin 3 times per week, in preventing CVC occlusions.
- Conditions
- Childhood Cancer, Aplastic Anemia, Metabolic Disorders
- Trial name
- NCT01343680
- Testing
- Heparin, Normal saline
- Where
- 2 sites in Canada
- Volunteers
- 2 people
- Sponsor
- Alberta Children's Hospital
Aplastic Anemia
Study of CM313(SC) Injection in Subjects With Relapsed/Refractory Aplastic Anemia
This is a randomized, open-label, phase IB/II clinical study to evaluate the safety, tolerability and preliminary efficacy of CM313(SC) injection in patients with relapsed/refractory aplastic anemia
- Trial name
- NCT06791824
- Testing
- CM313(SC) injection
- Where
- China
- Volunteers
- 125 people
- Sponsor
- Keymed Biosciences Co.Ltd
Hematologic Malignancies, Nonmalignant Diseases
Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Thiotepa is a chemotherapy drug used extensively in bone marrow transplantation. Thiotepa is a prodrug that undergoes metabolic conversion in the liver by CYP2B6 and CYP3A4 to its primary active metabolite, triethylene…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies
- Trial name
- NCT03609840
- Testing
- Thiotepa
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
Primary Immune Deficiency Disorder, Immune Deficiency Disease
Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune…
- Conditions
- Primary Immune Deficiency Disorder, Immune Deficiency Disease, Bone Marrow Failure, Short Telomere Length
- Trial name
- NCT04232085
- Testing
- Alemtuzumab, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Chronic Myeloproliferative Disorders, Leukemia
NMA Allogeneic Hematopoietic Cell Transplant in Hematologic Cancer/Disorders
RATIONALE: Giving low doses of chemotherapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells. It also stops the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00053989
- Testing
- anti-thymocyte globulin, graft-versus-tumor induction therapy, sargramostim
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Roswell Park Cancer Institute
Acquired Aplastic Anaemia
Clinico-Epidemiological Profile Of Acquired Aplastic Anemia Among Children At Assiut Governorate .
1. Assessment the clinical presentation and epidemiological profile of children with acquired aplastic anemia. 2. Determining the possible risk factors and associated conditions contributing to the development of acquired…
- Trial name
- NCT07259863
- Testing
- observational case series study no intervention
- Volunteers
- 35 people
- Sponsor
- Assiut University
Cytopaenia
A Study to Assess the Safety and Efficacy of Eltrombopag in Japanese Subjects With Refractory, Moderate or More Severe Aplastic Anemia
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Japanese moderate or more severe aplastic anemia (AA) subjects with a platelet count <30,000/microliter who were refractory…
- Trial name
- NCT02148133
- Testing
- Eltrombopag 12.5 mg, Eltrombopag 25 mg
- Where
- 12 sites in Japan
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
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
Diamond Blackfan Anemia
Mobilization of CD34+ Peripheral Blood Stem Cells in Patients With Diamond Blackfan Anemia Syndrome (DBAS)
Gene therapy is a new possible treatment for the anemia of DBAS. Gene therapy will soon be available for patients with RPS19-mutated DBAS.
- Trial name
- NCT07186179
- Testing
- Mobilization Regimen
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Northwell Health
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
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.
Diamond Blackfan Anemia, Dyskeratosis Congenita
Cancer in Inherited Bone Marrow Failure Syndromes
Background: A prospective cohort of Inherited Bone Marrow Failure Syndrome (IBMFS) will provide new information regarding cancer rates and types in these disorders.
- Conditions
- Diamond Blackfan Anemia, Dyskeratosis Congenita, Fanconi Anemia, Shwachman Diamond Syndrome
- Trial name
- NCT00027274
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Anemia, Aplastic
Allogeneic Stem Cell Transplantation for Patients With Severe Aplastic Anemia
For patients with severe aplastic anemia (SAA) who have failed to respond to immunosuppressive therapy and lack an HLA identical family member, our objectives are to make an initial assessment of the safety and efficacy of…
- Trial name
- NCT00578266
- Testing
- Cyclophosphamide,Campath IH and TBI
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Mayo Clinic
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
Fanconi's Anemia, Myelodysplastic Syndromes
Study of Allogeneic Bone Marrow Transplantation Following Cyclophosphamide and Radiotherapy in Patients With Myelodysplastic Syndrome and Acute Leukemia Related to Fanconi's Anemia
OBJECTIVES: I. Determine the effectiveness of moderate dose cyclophosphamide and radiotherapy in terms of improving survival and reducing the morbidity following allogeneic bone marrow transplantation in patients with…
- Conditions
- Fanconi's Anemia, Myelodysplastic Syndromes, Leukemia, Nonlymphocytic, Acute, Leukemia, Lymphocytic, Acute
- Trial name
- NCT00005892
- Testing
- cyclophosphamide, Allogeneic Bone Marrow Transplantation
- Where
- United States
- Sponsor
- Fairview University Medical Center
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
Poor Graft Function, Aplastic Anemia Idiopathic
REscuing Bone Marrow Function in Patients with AplaStic AnaEmia and Bone Marrow FaiLure Post AllogEneiC Transplantation 2
Allogeneic stem cell transplantation involves the transplanting of donor blood stem cells into a recipient, this is performed mainly for the treatment of blood cancers.
- Trial name
- RESELECT2 (NCT06607367)
- Testing
- Ruxolitinib (JAKAVI®), Eltrombopag (Revolade®)
- Where
- Australia
- Volunteers
- 20 people
- Sponsor
- Melbourne Health
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 4 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Leukemia, Non Hodgkin's Lymphoma
Blood Transplantation for Patients With Hematologic Malignancies or Bone Marrow Failure States
The purpose of this study is to assess early treatment failure within 100 days and to assess the effect of this regimen on engraftment, GVHD, immune recovery, relapse of malignancy and survival.
- Conditions
- Leukemia, Non Hodgkin's Lymphoma, Aplastic Anemia
- Trial name
- NCT00038779
- Testing
- Megadose T-cell Depleted HLA-nonidentical Blood Progenitor Cell Transplantation
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- M.D. Anderson Cancer Center