Clinical trials: Idiopathic Aplastic Anemia
Bone Marrow Failure Syndromes, Anemia, Aplastic
Identifying Characteristics of Bone Marrow Failure Syndromes
Bone marrow failure syndromes (BMFS) are rare disorders characterized by dysfunctional hematopoietic stem cells, which give rise to all red and white blood cells.
- Conditions
- Bone Marrow Failure Syndromes, Anemia, Aplastic, Myelodysplastic Syndromes, Hemoglobinuria, Paroxysmal
- Trial name
- NCT00315419
- Where
- 4 sites in United States
- Volunteers
- 450 people
- Sponsor
- Office of Rare Diseases (ORD)
Aplastic Anemia
CK0801 for Treatment of Aplastic Anemia
This Phase 2, multicenter, open-label study is evaluating CK0801 for the treatment of aplastic anemia in adults with persistent transfusion dependence after at least one prior line of therapy or intolerance to standard-of-care…
- Trial name
- NCT07499102
- Testing
- CK0801
- Volunteers
- 12 people
- Sponsor
- Cellenkos, Inc.
Anemia, Aplastic
Sirolimus and Cyclosporine for Treatment-Resistant Aplastic Anemia
Aplastic anemia is a rare autoimmune disorder in which the bone marrow production of blood cells is greatly decreased or absent.
- Trial name
- NCT00319878
- Testing
- Sirolimus, Cyclosporine
- Where
- 4 sites in United States
- Volunteers
- 52 people
- Sponsor
- Office of Rare Diseases (ORD)
Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission
Reduced Intensity Chemotherapy and Radiation Therapy Before Donor Stem Cell Transplant in Treating Patients With Hematologic Malignancies
This clinical trial studies the use of reduced intensity chemotherapy and radiation therapy before donor stem cell transplant in treating patients with hematologic malignancies.
- Conditions
- Acute Myeloid Leukemia, Acute Myeloid Leukemia in Remission, Aplastic Anemia, Chronic Myelomonocytic Leukemia
- Trial name
- NCT02566304
- Testing
- Fludarabine, Total-Body Irradiation, T Cell-Depleted Donor Lymphocyte Infusion
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
Aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) are interrelated and very rare diseases. Therefore, little data about clinical characteristics, especially the variety of symptoms in the course of the respective…
- Trial name
- ePRO-AA-PNH (NCT04128943)
- Testing
- symptom questionnaire, recording of vital signs, European Organisation for Research and Treatment of Cancer (EORTC) quality of life questionnaire (QLQ)-C30 questionnaire
- Where
- Switzerland
- Volunteers
- 9 people
- Sponsor
- University Hospital, Basel, Switzerland
Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Nonmyeloablative Allogeneic Transplant
Allogeneic transplant from a matched sibling for the treatment of a variety of illnesses including bone marrow failure states, leukemias, myelodysplastic or myeloproliferative syndromes, lymphoma, or myeloma using a…
- Conditions
- Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia
- Trial name
- Mini-allo (NCT01272817)
- Testing
- Nonmyeloablative Allogeneic Transplant
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Scripps Health
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
Severe Aplastic Anemia
RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial
After exiting the RACE trial (NCT02099747) patients will be invited to participate in this long term follow-up study
- Trial name
- RACE2 (NCT05049668)
- Testing
- ATGAM plus CsA with or without Eltrombopag
- Where
- France
- Volunteers
- 197 people
- Sponsor
- European Society for Blood and Marrow Transplantation
Severe Aplastic Anemia
Retrospective Study of Patients With Severe Aplastic Anemia Who Relapsed After Immunosuppressive Therapy
Background: Severe aplastic anemia (SAA) is a form of bone marrow failure. It usually results from a cytotoxic T cell attack on the marrow stem cell. Two treatments can be used for most people with SAA.
- Trial name
- NCT04436380
- Where
- United States
- Volunteers
- 173 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
Anti-thymocyte Globulin and Cyclosporine as First-Line Therapy in Treating Patients With Severe Aplastic Anemia
RATIONALE: Immunosuppressive therapies, such as anti-thymocyte globulin and cyclosporine, may improve bone marrow function and increase blood cell counts.
- Trial name
- NCT01231841
- Testing
- cyclosporine, anti-thymocyte globulin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- The Cleveland Clinic
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
Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia
The purpose of this study is to determine the feasibility of comparing outcomes of patients treated de novo with immunosuppressive therapy (IST) versus matched unrelated donor (MUD) hematopoietic stem cell transplant (HSCT) for…
- Trial name
- NCT02845596
- Testing
- cyclosporine, Matched Unrelated Donor Hematopoietic Stem Cell Transplant, horse anti-thymocyte globulin (ATG)
- Where
- 13 sites in United States
- Volunteers
- 40 people
- Sponsor
- Michael Pulsipher
Severe Aplastic Anemia
Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia
RATIONALE: It has been shown that about 30% of patients do not respond to immunosuppressive therapy or experience recurrence, and graft rejection and graft-versus-host-disease (GVHD) decrease event-free survival to 30% to 50% in…
- Trial name
- NCT02407470
- Testing
- Rabbit antithymoglobulin (ATG), Adipose derived mesenchymal stem cells ( AD-MSCs), AD-MSC transdifferentiated HSCs (AD-HSCs)
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Navy General Hospital, Beijing
Leukemia, Aplastic Anemia
Methadone as an Alternative Treatment for Children Underdoing HSCT
Mucositis is a normal side effect of stem cell transplant which happens as a result of chemotherapy being given prior to a new donor cell infusion (bone marrow transplant).
- Conditions
- Leukemia, Aplastic Anemia, Bone Marrow Failure Syndromes, SCID
- Trial name
- MATCH (NCT06940570)
- Testing
- PCA plus methadone (Experimental), PCA Alone (SOC)
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- University of Texas Southwestern Medical Center