Clinical trials: Anemia, Aplastic
Fanconi Anemia
Hematopoietic Stem Cell Transplantation in High Risk Patients With Fanconi Anemia
RATIONALE: A bone marrow or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy.
- Trial name
- NCT00258427
- Testing
- anti-thymocyte globulin, filgrastim, busulfan
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Purpura, Schoenlein-Henoch, Graft Versus Host Disease
Autologous Peripheral Blood Stem Cell Transplantation in Patients With Life Threatening Autoimmune Diseases
OBJECTIVES: I. Determine whether there is prompt engraftment after autologous peripheral blood stem cell transplantation using filgrastim (G-CSF) mobilization in patients with life threatening autoimmune diseases. II.
- Conditions
- Purpura, Schoenlein-Henoch, Graft Versus Host Disease, Anemia, Hemolytic, Autoimmune, Rheumatoid Arthritis
- Trial name
- NCT00006055
- Testing
- anti-thymocyte globulin, cyclophosphamide, cyclosporine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Fairview University Medical Center
Sickle Cell Anemia, Severe Aplastic Anemia
Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes
RATIONALE: Although used primarily to treat malignant disorders of the blood, allogeneic stem cell transplantation can also cure a variety of non-cancerous, inherited or acquired disorders of the blood.
- Conditions
- Sickle Cell Anemia, Severe Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria (PNH), Pure Red Cell Aplasia
- Trial name
- NCT00004143
- Testing
- Campath, Chemo and/or TBI Allo SCT
- Where
- 2 sites in United States
- Volunteers
- 2 people
- Sponsor
- David Rizzieri, MD
Anemia, Diamond-Blackfan
Treatment of Refractory Diamond-Blackfan Anemia With Eltrombopag
Background: Diamond-Blackfan anemia (DBA) is treated with steroids. But some people cannot take steroids, or steroids don t work.
- Trial name
- NCT04269889
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Failure Syndromes
Fludarabine Based RIC for Bone Marrow Failure Syndromes
This is a pilot study to determine whether fludarabine-based reduced intensity conditioning (RIC) regimens facilitate successful donor engraftment of patients with acquired aplastic anemia (AA) and Inherited bone marrow failure…
- Trial name
- NCT02928991
- Testing
- MRD-BMT with Fludarabine-based RIC for Acquired AA, MRD-BMT with Fludarabine-based RIC for iBMF with trilineage aplasia, MRD-BMT with Fludarabine-based RIC for iBMF without trilineage aplasia
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital of Philadelphia
Aplastic Anemia
Comparison of Cy-Atg vs Flu-Atg for the Conditioning Therapy in Allo-HCT for Adult Aplastic Anemia
The purpose of this study is to reduce the regimen related toxicities and transplantation related mortality after allogeneic stem cell transplantation in adult acquired aplastic anemia (AA), the trials of reduced dose of Cy along…
- Trial name
- CyATG-FluATG (NCT01145976)
- Testing
- Cy-ATG, Flu-ATG
- Where
- South Korea
- Volunteers
- 98 people
- Sponsor
- Cooperative Study Group A for Hematology
Bone Marrow Failure Syndrome, Aplastic Anemia
The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders.
This is a prospective hybrid implementation-effectiveness study of a model of care for patients with bone marrow failure syndromes and inherited predisposition to haematological malignancy that includes comprehensive diagnostic…
- Trial name
- EMBRACE (NCT04819607)
- Testing
- model of care
- Where
- Australia
- Volunteers
- 100 people
- Sponsor
- Peter MacCallum Cancer Centre, Australia
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, Healthy
Collection of Blood and Bone Marrow From Patients With Aplastic Anemia for Analysis of Adhesion Molecules, Chemokines and Their Receptors
This study is designed to collect bone marrow and/or peripheral blood cells and plasma, for the analysis of adhesion molecules, chemokines and their receptors from newly-diagnosed aplastic anemia patients, and patients treated…
- Trial name
- NCT01680055
- Where
- China
- Volunteers
- healthy volunteers welcome
- Sponsor
- Shandong University
Fanconi Anemia, Head and Neck Cancer
Safety and Tolerability of Metformin and Naproxen for Cancer Prevention in Fanconi Anemia
Finding effective ways to slow down or prevent the development of cancer in people with Fanconi anemia (FA) is a critical medical priority.
- Conditions
- Fanconi Anemia, Head and Neck Cancer, Squamous Cell Carcinoma, Squamous Cell Cancer of Head and Neck (SCCHN)
- Trial name
- NCT07901959
- Testing
- Metformin, Naproxen Sodium
- Where
- 2 sites in United States
- Volunteers
- 80 people
- Sponsor
- Rockefeller University
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
Bone Marrow Failure Disorders, Aplastic Anemia
TPO-Mimetic Use in Children for Hematopoietic Failure
This is an open label, prospective Pilot interventional study will investigate the safety and efficacy of Romiplostim, thrombopoietin (TPO) mimetic, in children (ages: 0 to 21 years) with broad scope of bone marrow failure…
- Conditions
- Bone Marrow Failure Disorders, Aplastic Anemia, Thrombocytopenia, Refractory Cytopenia of Childhood
- Trial name
- NCT04478227
- Testing
- Romiplostim
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Anjali Sharathkumar
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
Aplastic Anemia
A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment
This research study is testing a medicine called avatrombopag in adults with aplastic anemia, a rare condition where the bone marrow does not produce enough blood cells.
- Trial name
- NCT07786922
- Testing
- Avatrombopag 20 mg Oral Tablet
- Volunteers
- 26 people
- Sponsor
- Swedish Orphan Biovitrum
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
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Aplastic Anemia
A Study of the Safety and Effectiveness of Eltrombopag in Combination With Immunosuppressive Therapy (IST) in Korean Pediatric Patients With Treatment Naïve Severe Aplastic Anemia
The aim of this study was to investigate the safety, tolerability, and effectiveness of eltrombopag in combination with immunosuppressive therapy (IST) in real world practice in pediatric patients with severe aplastic anemia…
- Trial name
- NCT07415083
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
Aplastic Anemias
Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study
Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly.
- Trial name
- NCT06650553
- Testing
- Clinically diagnosed AA patients are divided into HLA-matched HSCT group and umbilical cord blood-supported haplo-HSCT group.
- Where
- China
- Volunteers
- 110 people
- Sponsor
- Shanxi Bethune Hospital
Aplastic Anemia
Safety and Efficacy of Exjade in the Treatment of Transfusion-dependent Iron Overload in Aplastic Anemia Patients
Evaluated Exjade efficacy and safety in patients with aplastic anemia and transfusion-dependent iron overload, undergoing treatment programs of immunosuppressive treatment (Cyclosporine A) , in comparison with a group of patients…
- Trial name
- NCT01818726
- Testing
- ICL670, Chelation, No chelation
- Where
- Russia
- Volunteers
- 15 people
- Sponsor
- Novartis Pharmaceuticals
Severe Aplastic Anemia (SAA)
Efficacy of Romiplostim in Treatment of SAA in Adults Previously Untreated With or Refractory to Immunosuppressive Therapy
Romiplostim has been used in clinical trials for the treatment of severe and very severe aplastic anemia (SAA/vSAA) in Asian participants who are either previously untreated with immunosuppressive therapy (IST) or refractory to…
- Trial name
- NCT05323617
- Testing
- Romiplostim, Antithymocyte Globulin, Cyclosporine A
- Sponsor
- Amgen