Clinical trials: Bone Marrow Failure
Advanced Solid Tumors or Non-Hodgkin's Lymphoma (NHL)
A Phase 1/2 Trial of SRA737 in Subjects With Advanced Cancer
The purpose of this clinical study is to establish the safety profile, determine the maximum tolerated dose (MTD) and recommend a Phase 2 dose and schedule of SRA737; and to evaluate the efficacy of SRA737 in…
- Trial name
- NCT02797964
- Testing
- SRA737
- Where
- 15 sites in United Kingdom
- Volunteers
- 107 people
- Sponsor
- Sierra Oncology LLC - a GSK company
Severe Aplastic Anemia
Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia
This is a multicenter, randomized, placebo-control, phase III study to investigate hetrombopag in subjects with severe AA who are treated naive. 180 treated naive patients with SAA will be enrolled in the study.
- Trial name
- NCT03825744
- Testing
- Hetrombopag Olamine+Standard Therapy
- Where
- China
- Volunteers
- 240 people
- Sponsor
- Jiangsu HengRui Medicine Co., Ltd.
Thalassemia, Sickle Cell Disease
Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot…
- Conditions
- Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
- Trial name
- NCT00235391
- Testing
- Deferasirox
- Where
- 141 sites in United States, Belgium and 10 more
- Volunteers
- 1,683 people
- Sponsor
- Novartis Pharmaceuticals
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
Severe Aplastic Anemia (SAA)
Combination of Eltrombopag With Immunosuppressive Therapy in East-Asian Patients With Severe Aplastic Anemia
This study was designed to evaluate the efficacy and safety of eltrombopag when added to r-ATG and CsA in treatment naive East-Asian adult and pediatric patients with severe aplastic anemia (SAA).
- Trial name
- REACTS (NCT04328727)
- Testing
- eltrombopag, rabbit anti-thymocyte globulin (r-ATG), cyclosporine A (CsA)
- Where
- 12 sites in China, Japan and 2 more
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemia
A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the…
- Trial name
- NCT02094417
- Testing
- AMG531
- Where
- South Korea
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Korea Co., Ltd.
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
Aplastic Anemia
Expanded Access Use of Itacitinib to Treat a Single Patient With Aplastic Anemia
Expanded Access Use of Itacitinib to Treat a Single Patient With Aplastic Anemia
- Trial name
- NCT03906318
- Testing
- Iticitinib
- Sponsor
- Incyte Corporation
Infantile Malignant Osteopetrosis
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.
- Trial name
- NCT04525352
- Testing
- RP-L401
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Kidney Tubular Necrosis, Acute
Allogeneic Multipotent Stromal Cell Treatment for Acute Kidney Injury Following Cardiac Surgery
The purpose of this trial is to determine if the administration of allogeneic MSCs at defined doses is safe in patients who are at high risk of developing significant Acute Kidney Injury (AKI) after undergoing on-pump cardiac…
- Trial name
- NCT00733876
- Testing
- Multipotent Stromal Cells, Administration of MSC
- Where
- 2 sites in United States
- Volunteers
- 15 people
- Sponsor
- AlloCure Inc.
Fanconi Anemia Complementation Group A
Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A
This is an open-label Phase II clinical trial to evaluate the efficacy of a hematopoietic cell-based gene therapy for pediatric patients with Fanconi Anemia, subtype A (FA-A).
- Trial name
- NCT04069533
- Testing
- RP-L102
- Where
- 2 sites in Spain, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Aplastic Anemia
Study Assessed the Safety and Efficacy of Eltrombopag in Chinese Refractory or Relapsed Severe Aplastic Anemia (SAA) Subjects.
This was a non-randomized, open-label, phase II study to assess the efficacy and safety of eltrombopag in Chinese subjects with refractory or relapsed severe aplastic anemia (SAA).
- Trial name
- NCT03988608
- Testing
- Eltrombopag
- Where
- 5 sites in China
- Volunteers
- 20 people
- Sponsor
- Novartis Pharmaceuticals
Thrombocytopenia, Hematologic Malignancy
Thrombosomes® in Bleeding Thrombocytopenic Patients Study
This prospective, multicenter, randomized, open-label, Phase 2, parallel, dose ranging, multidose trial will enroll patients into 3 Thrombosomes dose groups and 1 control liquid stored platelets (LSP) group in order to evaluate…
- Conditions
- Thrombocytopenia, Hematologic Malignancy, Bone Marrow Aplasia, Myeloproliferative Disorders
- Trial name
- NCT04631211
- Testing
- Thrombosomes, Liquid Stored Platelets (LSP)
- Where
- 6 sites in United States, Israel and 1 more
- Volunteers
- 21 people
- Sponsor
- Cellphire Therapeutics, Inc.
Leukemia, Myeloid, Chronic, Anemia, Aplastic
Study of Parathyroid Hormone Following Sequential Cord Blood Transplantation From an Unrelated Donor
The purpose of this study is to determine whether the addition of parathyroid hormone after a sequential cord blood transplant will improve engraftment, which is the ability of the transplanted stem cells to grow and to…
- Conditions
- Leukemia, Myeloid, Chronic, Anemia, Aplastic, Myelofibrosis, Lymphoma
- Trial name
- NCT00393380
- Testing
- Parathyroid Hormone (teriparatide)
- Where
- 4 sites in United States
- Volunteers
- 13 people
- Sponsor
- The Emmes Company, LLC
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
Chronic Myeloid Leukemia
A Study of Full Treatment-free Remission in Patients With Chronic Myeloid Leukemia
This study is constituted of two stage: Treatment-Free Remission 1 (TFR1) stage and Treatment-Free Remission 2 (TFR2) stage. The purpose of the TFR1 stage is to assess the effect of nilotinib reduced to half the standard dose for…
- Trial name
- DANTE (NCT03874858)
- Testing
- Nilotinib, Asciminib
- Where
- 26 sites in Italy
- Volunteers
- 135 people
- Sponsor
- Novartis Pharmaceuticals
Heart Failure
Congestive Heart Failure Cardiopoietic Regenerative Therapy (CHART-2) Trial - THE CHART-2 TRIAL
This study is designed to evaluate the efficacy and safety of the C3BS-CQR-1 in the treatment of patients with chronic heart failure secondary to ischemic cardiomyopathy.
- Trial name
- CHART-2 (NCT02317458)
- Testing
- Experimental
- Volunteers
- 400 people
- Sponsor
- Celyad Oncology SA
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
Sickle Cell Disease
Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with Hemoglobinopathies to halt disease progression.
- Trial name
- NCT00777231
- Testing
- Enriched Hematopoetic Stem Cell Infusion
- Where
- 3 sites in United States
- Volunteers
- 9 people
- Sponsor
- Talaris Therapeutics Inc.
Congestive Heart Failure
Feasibility Study of BMAC Enhanced CABG
Patients undergoing CABG will have 60 mL of bone marrow drawn, concentrated in a SmartPRep2 centrifuge and have the concentrated nucleated cells injected into areas of ischemic myocardium
- Trial name
- NCT01074099
- Testing
- BMAC, CABG only
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Harvest Technologies