Clinical trials: Severe Anemia
Severe Aplastic Anemia (SAA)
Extended Dosing With Eltrombopag for Severe Aplastic Anemia
Background: Eltrombopag is a drug being tested for treating severe aplastic anemia. It can help improve blood counts in these patients.
- Trial name
- NCT01891994
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson
Sickle Cell Crisis, Acute Pain
Low-dose Ketamine vs Morphine for Vaso-occlusive Crisis in Sicklers
This clinical trial will inform of the role of Low dose ketamine in the acute treatment of severe painful sickle cell crisis in children in a day-case sickle cell centre.
- Trial name
- KEM-VOC (NCT02434939)
- Testing
- Low dose ketamine, Morphine
- Where
- Uganda
- Volunteers
- 240 people
- Sponsor
- Makerere University
Hirsutism, Androgenetic Alopecia
DUS on the Prescribing Indications for CPA/EE in 5 European Countries
This study was designed to compile the reasons and specific indications for the prescription of Cyproterone Acetate combined with Ethinyl Estradiol by Health Care Providers.
- Conditions
- Hirsutism, Androgenetic Alopecia, Severe Anemia, Seborrhea
- Trial name
- NCT02494297
- Where
- Germany
- Volunteers
- 1,597 people, healthy volunteers welcome
- Sponsor
- Center for Epidemiology and Health Research, Germany
Transfusion-dependent Hemachromatosis, Thalassemia Major
Pilot Study for Patients With Poor Response to Deferasirox
This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron…
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Trial name
- NCT00749515
- Testing
- Deferoxamine, Deferasirox, HIDA
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Myeloproliferative Disorders, Leukemia
Donor Umbilical Cord Blood Transplant By Injection Into the Bone Marrow in Treating Patients With Hematologic Cancer
Rationale: Giving chemotherapy and total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune system from rejecting the donor's…
- Conditions
- Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00295880
- Testing
- umbilical cord blood transplantation
- Volunteers
- 12 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Disease
A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease
Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell disease lack a matched family donor. This study aims for cure of sickle cell disease by performing unrelated donor (outside family)…
- Trial name
- NCT01279616
- Testing
- Fludarabine monophosphate, Rituximab, Busulfan
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Nationwide Children's Hospital
Severe Aplastic Anemia
Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)
This study is a prospective, multicenter phase II study with patients receiving haploidentical transplantation for Severe Aplastic Anemia (SAA).
- Trial name
- CHAMP (NCT02918292)
- Testing
- Antithymocyte Globulin (ATG), Fludarabine, Cyclophosphamide
- Where
- 28 sites in United States
- Volunteers
- 32 people
- Sponsor
- Medical College of Wisconsin
Chronic Myelogenous Leukemia (CML), Acute Myelogenous Leukemia (AML)
Unrelated Umbilical Cord Blood (UBC)Transplantation
Hematopoietic progenitor cell (HPC- primitive cells in the blood, bone marrow and umbilical cord that can restore the bone marrow) transplant can be a curative therapy for the treatment of hematologic malignancies (a disease of…
- Conditions
- Chronic Myelogenous Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome, Multiple Myeloma
- Trial name
- NCT01768845
- Testing
- umbilical cord blood (UCB)
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- West Virginia University
Chronic Iron Overload
Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients
Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate efficacy and safety of a 12-months treatment with deferiprone (DFP) at dose of 75-100 mg/kg/day versus deferasirox (DFX) at dose of 20-40…
- Trial name
- NCT01825512
- Testing
- Deferiprone, Deferasirox
- Where
- 22 sites in Albania, Cyprus and 5 more
- Volunteers
- 435 people
- Sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
Sickle Cell Disease
Haploidentical Hematopoietic Stem Cell Transplantation (HSCT) for Patients With Severe Sickle Cell Disease
The purpose of this study is to develop a safe and curative stem cell transplant approach to treating sickle cell disease by assessing the safety of haploidentical hematopoietic stem cell transplantation using αβ+ T-cell…
- Trial name
- NCT04207320
- Testing
- αβ+ T-cell depletion with Miltenyi CliniMACS system
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- University of Chicago
Sickle Cell Disease
Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
This is a Phase II, single arm, multi-center trial. It is designed to estimate the efficacy and toxicity of hematopoietic stem cell transplantation (HSCT) in patients with sickle cell disease (SCD) who have high risk features.
- Trial name
- STRIDE (NCT01565616)
- Testing
- Conditioning Regimen with Bone Marrow Transplant
- Where
- 8 sites in United States
- Volunteers
- 22 people
- Sponsor
- Emory University
Aplastic Anemia, Neutropenia
A Pilot Study of Fludarabine Plus Cyclophosphamide in Refractory Severe Aplastic Anemia
Background: Severe aplastic anemia (SAA) can lead to problems with bone marrow health and result in low blood cell counts, which require frequent transfusions.
- Conditions
- Aplastic Anemia, Neutropenia, Pancytopenia, Severe Aplastic Anemia
- Trial name
- NCT01187017
- Testing
- Cyclophosphamide, Fludarabine
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Donor Peripheral Stem Cell Transplant in Treating Patients With Advanced Hematologic Cancer or Other Disorders
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00544115
- Testing
- busulfan, cyclophosphamide, cyclosporine
- Volunteers
- 260 people
- Sponsor
- City of Hope Medical Center
Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Patients with medical conditions requiring allogeneic hematopoietic cell transplantation (allo-HCT) are at risk of developing a condition called graft versus host disease (GvHD) which carries a high morbidity and mortality.
- Conditions
- Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia in Remission, Myelodysplastic Syndromes, Chronic Myeloid Leukemia
- Trial name
- HAPLOTAB (NCT05236764)
- Testing
- CliniMACS
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- Baylor College of Medicine
Thalassemia, Sickle Cell Disease
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01319851
- Testing
- Alefacept
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
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
Severe Aplastic Anemia
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
Background: Severe aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells.
- Trial name
- NCT04304820
- Testing
- Eltrombopag, Cyclosporine, Horse-Anti-thymocyte-Globulin
- Where
- United States
- Volunteers
- 80 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Aplastic Anemia
A Phase II Dose-escalation Study Characterizing the PK of Eltrombopag in Pediatric Patients With Previously Untreated or Relapsed Severe Aplastic Anemia or Recurrent Aplastic Anemia
This is a phase II, open label, multi-center, intra-patient dose escalation study to characterize the pharmacokinetics (PK) after oral administration of eltrombopag in combination with immunosuppressive therapy in pediatric…
- Trial name
- NCT03025698
- Testing
- Eltrombopag, hATG, CsA
- Where
- 20 sites in United States, Hong Kong and 4 more
- Volunteers
- 51 people
- Sponsor
- Novartis Pharmaceuticals