Clinical trials: Aplastic Anaemia
Severe Aplastic Anemia, Telomere Biology Disorders
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Background: Bone marrow failure diseases are rare. Much is known about the diseases at the time of diagnosis, but long-term data about the effects of the diseases and treatments are lacking.
- Conditions
- Severe Aplastic Anemia, Telomere Biology Disorders, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT05012111
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia, Bone Marrow Failure Syndrome
Haploidentical HCT for Severe Aplastic Anemia
This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation.
- Trial name
- NCT04558736
- Testing
- Anti-Thymocyte Globulin (Rabbit), Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- St. Jude Children's Research Hospital
Aplastic Anemia, Hypoplastic Myelodysplastic Syndrome
Phase IIA Open Label Study to Evaluate Efficacy and Safety of BL-8040 Followed by (hATG), Cyclosporine and Methyprednisolone in Adult Subjects With Aplastic Anemia or Hypoplastic Myelodysplastic Syndrome
An open label single arm study to assess efficacy and safety of BL-8040 on top of standard immunotherapy regimen of hATG, cyclosporine and steroids in patients with Hypoplastic MDS and AA over the course of a six month (180 day)…
- Trial name
- NCT02462252
- Testing
- BL-8040, horse anti-thymocyte globulin (hATG), Methylprednisolone
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- BioLineRx, Ltd.
Fanconi Anemia
Mobilization and Collection of Peripheral Blood Stem Cells in Patients With Fanconi Anemia Using G-CSF and Plerixafor
The purpose of this study is to assess the feasibility of Plerixafor used in combination with G-CSF (Granulocyte Colony Stimulating Factor) in 5 Fanconi anemia patients to mobilize and collect a sufficient number of peripheral…
- Trial name
- FancoMob (NCT02678533)
- Testing
- G-CSF, Plerixafor
- Where
- France
- Volunteers
- 4 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Acquired Aplastic Anemia
Haploidentical Stem Cell Transplantation for Children With Acquired Severe Aplastic Anemia
Rationale: Fludarabine, cyclophosphamide, anti-thymocyte globulin and low-dose total body irradiation (LD-TBI) may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic…
- Trial name
- NCT01759732
- Testing
- Fludarabine, Cyclophosphamide, anti-thymocyte globulin
- Where
- South Korea
- Volunteers
- 10 people
- Sponsor
- Asan Medical Center
Cancer, Anemia
Efficacy and Safety of Heated Chinese Herb Medicine Patch Therapy on Chemotherapy-Induced Aplastic Anemia
The objective of this clinical trial is to establish a treatment for chemotherapy-induced pancytopenia symptoms using heated traditional Chinese medicine (TCM) plasters through a randomized, single-blind clinical trial.
- Trial name
- NCT07790692
- Testing
- The experimental group (heated TCM plaster group), Heat source without TCM plaster group, No heat source with TCM plaster group
- Where
- Taiwan
- Volunteers
- 40 people
- Sponsor
- Hung-Rong Yen
Aplastic Anemia
Prophylactic NAC to Improve Platelet Engraftment After Haploidentical Transplantation in Severe Aplastic Anemia Patients
This study aims to evaluate the efficacy and safety of prophylactic oral N-acetylcysteine (NAC) for facilitating platelet engraftment in patients with severe aplastic anemia (SAA) receiving haploidentical hematopoietic stem cell…
- Trial name
- NCT07737262
- Testing
- N-acetylcysteine
- Volunteers
- 142 people
- Sponsor
- Peking University People's Hospital
Hematologic Malignancy
Conditioning Regimen for Allogeneic Hematopoietic Stem Cell Transplantation of Patients With Hematological Diseases
Participants in this study have a hematologic malignancy (a disorder in the bone marrow that affects the body's ability to create blood) that might benefit from receiving an allogeneic stem cell transplant (meaning the cells come…
- Trial name
- NCT00056966
- Testing
- ANTI-CD45, CAMPATH-1H, FK506
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- Baylor College of Medicine
Anemia, Aplastic
Conditioning Regimens for Patients With Severe Aplastic Anemia Transplanted With Marrow From an Unrelated Donor
A single arm dose optimization study in which all patients are given a fixed dose of Cytoxan (4 x 50 mg/kg) plus ATG (3 x 30 mg/kg) and the TBI dose starting at 3 x 200 cGy is escalated or de-escalated dependent upon engraftment…
- Trial name
- NCT00144729
- Testing
- Bone marrow transplant
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Fred Hutchinson Cancer Center
The Prophylactic Value of Ruxolitinib for aGVHD in HSCT Recipients of AA
Ruxolitinib as GVHD Prophylaxis in AA Patients
Our study aimed to determine the prophylactic value of ruxolitinib for aGVHD in HSCT recipients of AA.Ruxolitinib was initiated at the beginning of conditioning regimen at 5 twice daily until 3 months post-transplantation.
- Trial name
- NCT05914714
- Testing
- Ruxolitinib 5 MG BID Oral Tablet
- Volunteers
- 46 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Fanconi Anemia
Quercetin in Children With Fanconi Anemia; a Pilot Study
Fanconi anemia (FA) is an autosomal recessive disease characterized by progressive bone marrow failure (BMF), congenital abnormalities and a predisposition to malignancy.
- Trial name
- NCT01720147
- Testing
- Quercetin (dietary supplement)
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Severe Aplastic Anemia
Bone Marrow Transplant Trial for Patients With Refractory Severe Aplastic Anemia
Patients with severe, refractory aplastic anemia have a severe, life threatening disease in their bone marrow. Refractory disease means that disease has come back or not responded after receiving one or more immunosuppressive…
- Trial name
- NCT01383434
- Testing
- Bone Marrow Transplant
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Myeloid Diseases
T Cell Depleted Allogeneic Hematopoietic Stem Cell Transplantation Conditioned With a Reduced Intensity Regimen in Patients With Hematologic Malignancies and Aplastic Anemia
The main purpose of this study is to learn if a new combination of chemotherapy, in combination with low-dose radiation, will be safe for the patient, and at the same time provide the best opportunity to cure the bone marrow…
- Trial name
- NCT03531736
- Testing
- Antithymocyte globulin (Rabbit), fludarabine, total body irradiation
- Where
- 2 sites in United States
- Volunteers
- 17 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Fanconi Anemia
Prognostic Significance of Mutation Type and Chromosome Fragility in Fanconi Anemia
The goal of this observational study is to analyze the data included in the Spanish Registry of Patients with Fanconi anemia to better understand the natural history of the disease, identify genetic risk and prognostic factors…
- Trial name
- NCT06490510
- Where
- Spain
- Volunteers
- 227 people
- Sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau
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.
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.
Aplastic Anemia
Human Leukocyte Antigen (HLA)-Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Aplastic Anemia
Rationale: Chemotherapy with fludarabine, cyclophosphamide and anti-thymocyte globulin may induce the engraftment cross the immunologic barrier in the setting of HLA-haploidentical allogeneic hematopoietic cell transplantation.
- Trial name
- NCT01105273
- Testing
- anti-thymocyte globulin, filgrastim, Fludarabine
- Where
- South Korea
- Volunteers
- 12 people
- Sponsor
- Asan Medical Center
Severe Aplastic Anemia
Improving Immunosuppressive Treatment for Patients With Severe Aplastic Anemia
Severe aplastic anemia (SAA) is a life-threatening bone marrow failure disorder characterized by pancytopenia and a hypocellular bone marrow.
- Trial name
- NCT00061360
- Testing
- ATG+Rapamune+cyclosporine, ATG+cyclosporine
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia
MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.
A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal…
- Conditions
- Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT06412497
- Testing
- Rituximab, Rabbit ATG, Cyclophosphamide
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Severe Aplastic Anemia
Peripheral Blood Allogenic Stem Cell Transplantation Using Non-anti Thymocyte Globulin Regimens in Severe Aplastic Anemia Patients
Aplastic anemia is a syndrome of bone marrow failure characterized by peripheral pancytopenia and marrow hypoplasia.The theoretical basis for marrow failure includes primary defects in or damage to the stem cell or the marrow…
- Trial name
- NCT03295058
- Testing
- Non ATG Conditioning regimen, GVHD Prophylaxis, Allogenic Stem Cell Transplantation
- Volunteers
- 50 people
- Sponsor
- Assiut University