Clinical trials: Bone Marrow Failure Syndrome
Acute Leukemias, Burkitt's Lymphoma
Reduced Intensity Conditioning and Haploidentical Related Bone Marrow for Patients With Hematologic Diseases
This is a treatment guideline for HLA-Haploidentical hematopoietic stem cell transplant (HSCT) using a reduced intensity conditioning (RIC) regimen.
- Conditions
- Acute Leukemias, Burkitt's Lymphoma, Chronic Myelogenous Leukemia
- Trial name
- NCT02145039
- Testing
- Fludarabine, Cyclophosphamide, Total Body Irradiation
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Hematologic Neoplasms, Multiple Myeloma
Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States
The purpose of this study is to look at whether the combination of lower-dose chemotherapy with two chemotherapy (anti-cancer) drugs, called busulfan and melphalan, and an antibody medication called alemtuzumab (Campath®), can…
- Conditions
- Hematologic Neoplasms, Multiple Myeloma, Anemia, Aplastic, Hemoglobinuria, Paroxysmal
- Trial name
- ALBUM (NCT00997386)
- Testing
- busulfan, and melphalan, and alemtuzumab
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Arizona
Immunodeficiency Syndrome, Severe Aplastic Anemia
Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders
This phase I/II trial studies the side effects of fludarabine phosphate, cyclophosphamide and total-body irradiation followed by donor bone marrow transplant and cyclophosphamide, mycophenolate mofetil, tacrolimus, and sirolimus…
- Conditions
- Immunodeficiency Syndrome, Severe Aplastic Anemia, Genetic Disorder
- Trial name
- NCT00358657
- Testing
- Allogeneic Bone Marrow Transplantation, Cyclophosphamide, Fludarabine Phosphate
- Where
- 3 sites in United States
- Volunteers
- 14 people
- Sponsor
- Fred Hutchinson Cancer Center
Diamond Blackfan Anemia, Blackfan Diamond Syndrome
Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions.
- Conditions
- Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia
- Trial name
- LeucineDBA (NCT01362595)
- Testing
- leucine
- Where
- 11 sites in United States
- Volunteers
- 55 people
- Sponsor
- Northwell Health
Fanconi Anemia, Dyskeratosis Congenita
Safety and Efficacy Trial of Danazol in Patients With Fanconi Anemia or Dyskeratosis Congenita
Fanconi anemia (FA) and Dyskeratosis congenita (DC) are inherited bone marrow failure syndromes. The current androgen treatments (e.g., oxymetholone) used to treat FA and DC can cause unwanted masculinizing side effects…
- Trial name
- NCT01001598
- Testing
- danazol
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Boston Children's Hospital
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Severe Aplastic Anemia
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
Background: Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells.
- Trial name
- NCT01623167
- Testing
- Cohort 1: hATG, CsA, EPAG Day 14 to Month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6
- Where
- United States
- Volunteers
- 207 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
Fanconi Anemia
Cytoxan, Fludara, and Antithymocyte Globulin Conditioning Followed By Stem Cell Transplant in Treating Fanconi Anemia
RATIONALE: Giving chemotherapy, such as cyclophosphamide and fludarabine, before a donor stem cell transplant helps to remove the patient's cells to allow for the transplant cells to take and grow.
- Trial name
- NCT00630253
- Testing
- Anti-Thymocyte Globulin, Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sars-CoV2, Acute Respiratory Distress Syndrome
Mesenchymal Stromal Cells for the Treatment of SARS-CoV-2 Induced Acute Respiratory Failure (COVID-19 Disease)
*At this time, we are only enrolling at Houston Methodist Hospital (HMH)/Baylor College of Medicine (BCM) and are not shipping cells outside of BCM/HMH.* This is a study for patients who have respiratory infection caused by…
- Conditions
- Sars-CoV2, Acute Respiratory Distress Syndrome, COVID-19
- Trial name
- NCT04345601
- Testing
- Mesenchymal Stromal Cells, Supportive Care
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Baylor College of Medicine
Aplastic Anemia
Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia
To assess the tolerability and effectiveness of rabbit antithymocyte globulin (ATG, Thymoglobuline) with ciclosporin in the first line treatment of patients with acquired severe aplastic anaemia, and patients with non-severe…
- Trial name
- RATGAA07 (NCT00471848)
- Testing
- rabbit antithymocyte globulin
- Where
- 14 sites in France, Germany and 4 more
- Volunteers
- 35 people
- Sponsor
- European Society for Blood and Marrow Transplantation
Severe Aplastic Anemia
Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
The purpose of this study was to evaluate the efficacy and safety of eltrombopag in combination with cyclosporine alone as first-line therapy on overall hematologic response
- Trial name
- SOAR (NCT02998645)
- Testing
- eltrombopag, Cyclosporine
- Where
- 20 sites in Brazil, Hong Kong and 7 more
- Volunteers
- 54 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder
RATIONALE: Cyclosporine eye drops may prevent graft-versus-host disease of the eye in patients who have undergone donor stem cell transplant for hematologic cancer or bone marrow failure disorder.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00755040
- Testing
- cyclosporine ophthalmic emulsion
- Where
- 5 sites in United States
- Volunteers
- 164 people
- Sponsor
- Vanderbilt-Ingram Cancer Center