Clinical trials: Aplasia
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
Fanconi Anemia
TBI Dose De-escalation for Fanconi Anemia
This is a single arm, total body irradiation (TBI) trial. All patients will be prescribed TBI 300 cGy with the goal of evaluating secondary endpoints.
- Trial name
- NCT00352976
- Testing
- Cyclophosphamide, Fludarabine, Total Body Irradiation
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Diamond Blackfan Anemia
Safety and Efficacy Study of Sotatercept in Adults With Transfusion Dependent Diamond Blackfan Anemia
The purpose of this study is to determine the safety and dosing of drug Sotatercept, as a subcutaneous injection, to stimulate production of red blood cell production. To be given every 28 days for up to four doses.
- Trial name
- ACE-011-DBA (NCT01464164)
- Testing
- Sotatercept, Sotatercept with prednisone boost
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- Northwell Health
Aplastic Anemia
A Phase 2 Study to Evaluate the Efficacy and Safety of AMG531 in Aplastic Anemia
The present study will be conducted to evaluate the efficacy and safety of AMG531 and to determine the recommended initial dose of AMG531 on the basis of its efficacy and safety when it is administered subcutaneously (SC) to the…
- Trial name
- NCT02094417
- Testing
- AMG531
- Where
- South Korea
- Volunteers
- 35 people
- Sponsor
- Kyowa Kirin Korea Co., Ltd.
Blood Stem Cell Transplant Failure, Leukemia
Mismatched Transplantation Using High-dose Post-transplant Cyclophosphamide
The goal of this clinical research study is to learn about the safety of giving a stem cell transplant from a tissue-mismatched donor, followed by cyclophosphamide, to patients with certain types of blood disorders or blood…
- Conditions
- Blood Stem Cell Transplant Failure, Leukemia, Hematologic Malignancies
- Trial name
- NCT01010217
- Testing
- Cyclophosphamide, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 176 people
- Sponsor
- M.D. Anderson Cancer Center
Acute Myelogenous Leukemia, Lymphoid Leukemia
T-Cell Depleted Allogeneic Stem Cell Transplantation for Patients With Hematologic Malignancies
Objectives: 1. To evaluate disease free survival after Campath 1H-based in vivo T-cell depletion and non-myelo-ablative ablative stem cell transplantation in patients with hematologic malignancies. 2.
- Conditions
- Acute Myelogenous Leukemia, Lymphoid Leukemia, Chronic Myelogenous Leukemia, Malignant Lymphoma
- Trial name
- NCT00683046
- Testing
- Fludarabine, Melphalan, Stem cells
- Where
- United States
- Volunteers
- 204 people
- Sponsor
- University of Chicago
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
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
Growth Hormone Deficiency, Septo-Optic Dysplasia
Endocrine Dysfunction and Growth Hormone Deficiency in Children With Optic Nerve Hypoplasia
Hypotheses: 1. The prevalence of endocrinopathies, and growth hormone (GH) deficiency in particular, among young children diagnosed with optic nerve hypoplasia (ONH) is higher than is commonly thought. 2.
- Conditions
- Growth Hormone Deficiency, Septo-Optic Dysplasia, Hypopituitarism
- Trial name
- NCT00140413
- Testing
- Nutropin AQ
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Children's Hospital Los Angeles
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
Profound Bilateral Deafness Due to, Bilateral Cochlear Aplasia
A Safety Study of the Auditory Brainstem Implant for Pediatric Profoundly Deaf Patients
Current treatment options for bilateral profoundly deaf children, diagnosed with inner ear anatomical abnormalities, are limited and, in the case of absent cochleas, non-existent.
- Conditions
- Profound Bilateral Deafness Due to, Bilateral Cochlear Aplasia, Bilateral Cochlear Nerve Deficiency, Bilateral Cochlear Ossification Secondary to Meningitis
- Trial name
- NCT02102256
- Testing
- Auditory Brainstem Implant
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- Laurie Eisenberg
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
DiGeorge Syndrome, Complete Typical DiGeorge Anomaly
Thymus Transplantation in DiGeorge Syndrome #668
The study purpose is to determine whether cultured thymus tissue implantation (CTTI) is effective in treating typical complete DiGeorge syndrome.
- Trial name
- NCT00576407
- Testing
- Cultured Thymus Tissue for Implantation (CTTI)
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH