Clinical trials: Aplastic Anemias
Aplastic Anemia, Cytopenia
A Study of Emapalumab for Pediatric Aplastic Anemia
The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. Funding Source- FDA OOPD
- Conditions
- Aplastic Anemia, Cytopenia, Hypocellular Marrow
- Trial name
- NCT06430788
- Testing
- Emapalumab
- Where
- 6 sites in United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- Memorial Sloan Kettering Cancer Center
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
Aplastic Anemia, Paroxysmal Hemoglobinuria, Nocturnal
Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
OBJECTIVES: I. Confirm the efficacy demonstrated in a pilot study using high dose cyclophosphamide in patients with severe aplastic anemia. II.
- Trial name
- NCT00004464
- Testing
- cyclophosphamide, filgrastim
- Where
- United States
- Volunteers
- 69 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Aplastic Anemia
Alemtuzumab and Rituximab in Aplastic Anemia
The purpose of the study is to evaluate the efficacy of combination of alemtuzumab and rituximab as first line therapy in aplastic anemia.
- Trial name
- NCT01408342
- Testing
- Alemtuzumab and Rituximab
- Where
- Mexico
- Sponsor
- Hospital Universitario Dr. Jose E. Gonzalez
Anemia, Aplastic Anemia
Patient-Driven Transfusion Thresholds in Hematological Disorders: A Pilot Study
This pilot study evaluates safety of administration of red blood cell transfusions requested by patients based on their symptoms instead of levels of hemoglobin for the treatment of chronic anemia in patients with blood disorders.
- Conditions
- Anemia, Aplastic Anemia, Bone Marrow Failure, Hematopoietic and Lymphoid Cell Neoplasm
- Trial name
- NCT03086252
- Testing
- Red Blood Cell Transfusions (RBCT)
- Where
- United States
- Sponsor
- Emory University
Refractory Idiopathic Aplastic Anemia, Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia or in Relapse After Immunosuppression
Outcomes for patients with severe aplastic anemia (SAA) who are refractory to first-line immunosuppressive therapy (IST) and who lack a matched unrelated donor (MUD) remain poor.
- Trial name
- HAPLO-EMPTY (NCT05126849)
- Testing
- Allogenic transplantation
- Where
- 35 sites in France
- Volunteers
- 31 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Diamond-Blackfan Anemia, Kostmann's Neutropenia
Stem Cell Transplant for Bone Marrow Failure Syndromes
The researchers hypothesize that it will be possible to perform unrelated bone marrow or cord blood transplants in a safer manner by using less intensive therapy yet still achieve an acceptable level of donor cell engraftment for…
- Conditions
- Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
- Trial name
- NCT00176878
- Testing
- Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Severe Aplastic Anemia (SAA), Transfusion-dependent Non-severe Aplastic Anemia
CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA
This study aimed to explore the efficacy and safety of cyclosporine (CsA) combined with eltrombopag (EPAG)/hetrombopag (HPAG) and romiplostim N01 in the treatment of newly-diagnosed transfusion-dependent aplastic anemia (TD-NSAA)…
- Trial name
- NCT07523009
- Testing
- Cyclosporine (CsA), Thrombopoietin Receptor Agonist, Romiplostim N01
- Sponsor
- Peking Union Medical College Hospital
Chronic Myeloproliferative Disorders, Leukemia
Flu Vaccine in Preventing Influenza Infection in Healthy Volunteers and in Patients Who Have Undergone Stem Cell Transplant
RATIONALE: Studying immune response to flu vaccine in patients who have undergone a stem cell transplant may help doctors plan the best treatment.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00964821
- Testing
- trivalent influenza vaccine, Immunoenzyme technique, Laboratory biomarker analysis
- Where
- 2 sites in United States
- Volunteers
- 459 people, healthy volunteers welcome
- Sponsor
- City of Hope Medical Center
Aplastic Anemia Idiopathic
Outcomes in Bone Marrow Aplasia.
Bone marrow aplasia, also known as aplastic anemia (AA) is a potentially fatal bone marrow failure syndrome characterized by a paucity of hematopoietic stem cells (HSCs) and progenitor cells with varying degrees of cytopenia and…
- Trial name
- NCT06493981
- Testing
- Eltrombopag
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Assiut University
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
Childhood Langerhans Cell Histiocytosis, Fanconi Anemia
Umbilical Cord Blood for Stem Cell Transplantation in Treating Young Patients With Malignant or Nonmalignant Diseases
RATIONALE: Umbilical cord blood transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Fanconi Anemia, Leukemia, Lymphoma
- Trial name
- NCT00084695
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Milton S. Hershey Medical Center
Hereditary Diseases, Acquired Aplastic Anemia
Cord Blood Transplantation in Severe Aplastic Anemia
This is a Prospective Phase II Study to evaluate Cord Blood Transplantation in Inherited or Acquired Severe Aplastic Anemia Refractory or in Relapse after Immunosuppressive Therapy in the absence of an HLA identical donor;
- Conditions
- Hereditary Diseases, Acquired Aplastic Anemia, Relapse, Absence of an HLA Identical Donor
- Trial name
- APCORD (NCT01343953)
- Testing
- Cord Blood Transplantation
- Where
- France
- Volunteers
- 26 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome
Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders
This clinical trial is studying how well giving fludarabine phosphate and melphalan together with total-body irradiation followed by donor stem cell transplant works in treating patients with hematologic cancer or bone marrow…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00856388
- Testing
- fludarabine phosphate, melphalan, total-body irradiation
- Where
- United States
- Volunteers
- 62 people
- Sponsor
- Roswell Park Cancer Institute
Fanconi Anemia
Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Fanconi Anemia. The biospecimens will be used to create a biorepository that can be used to identify disease associated…
- Trial name
- NCT05910853
- Testing
- Specimen Donation
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sanguine Biosciences
Severe Aplastic Anemia
Cyclophosphamide Added to Standard Immunosuppressive Therapy With Herombopag as Front-line Therapy in Patients With Severe Aplastic Anemia
This is a prospective, single-center, single-arm, phase 2 study. This study aims to evaluate the efficacy and safety of Anti-lymphocyte globulin plus herombopag in combination with moderate-dose cyclophosphamide for severe…
- Trial name
- NCT05975996
- Testing
- Cyclophosphamide added to standard immunosuppressive therapy with herombopag
- Where
- China
- Volunteers
- 43 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
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