Clinical trials: Hereditary Anemias
Anemia, Sickle Cell
STRIDE Biorepository
The STRIDE Biorepository is an optional substudy available to participants in "Bone Marrow Transplantation vs Standard of Care in Patients with Severe Sickle Cell Disease (BMT CTN 1503) (STRIDE)".
- Trial name
- NCT02843347
- Testing
- Blood draw
- Where
- 40 sites in United States
- Volunteers
- 200 people
- Sponsor
- Emory University
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Busulfan, Antithymocyte Globulin, and Fludarabine Followed By a Donor Stem Cell Transplant in Treating Young Patients With Blood Disorders, Bone Marrow Disorders, Chronic Myelogenous Leukemia in First Chronic Phase, or Acute Myeloid Leukemia in First Remission
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Fanconi Anemia, Leukemia
- Trial name
- NCT00305708
- Testing
- anti-thymocyte globulin, busulfan, fludarabine phosphate
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of California, San Francisco
Sickle Cell Anemia, Sickle Cell β+ or β0 Thalassemia
Sickle Cell Anemia - A Comparative Study Between Three Ethnical Communities, a Multicenter Study
The purpose of this study is to take advance of the presence of two different cohorts of SCA patients in one country, the first group included SCA patients from Bedouin Arab origin that lives in Israel for more than one century…
- Trial name
- NCT01905787
- Where
- 4 sites in United States, Israel
- Volunteers
- 300 people
- Sponsor
- HaEmek Medical Center, Israel
Renal Impairment
Study of GBT021601 in Participants With Renal Impairment
Renal Impairment study of GBT021601.
- Trial name
- NCT05878704
- Testing
- GBT021601
- Where
- 4 sites in United States
- Volunteers
- 8 people
- Sponsor
- Pfizer
Anemia, Sickle Cell
Study of SANGUINATE™ In the Treatment of Sickle Cell Disease Patients With Vaso-Occlusive Crisis
Safety and effect of SANGUINATE on Sickle Cell Disease patients experiencing a vaso-occlusive crisis
- Trial name
- NCT02411708
- Testing
- SANGUINATE
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Prolong Pharmaceuticals
Sickle Cell Disease, Sickle Cell Anemia
Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease
Sickle Cell Disease (SCD) is a hereditary anemia that causes the red blood cells to change their shape from a round and doughnut-like shape to a half-moon/crescent, or sickled shape.
- Trial name
- NCT01000155
- Testing
- vorinostat
- Where
- 3 sites in United States
- Volunteers
- 5 people
- Sponsor
- Dana-Farber Cancer Institute
POEMS Syndrome, Amyloidosis
A Clinical Study of CD19/BCMA CAR-T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
A Clinical Study on the Safety and Effectiveness of CD19/BCMA Chimeric Antigen Receptor T Cells in the Treatment of Refractory POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, and Vasculitis
- Conditions
- POEMS Syndrome, Amyloidosis, Autoimmune Hemolytic Anemia, Vasculitis
- Trial name
- NCT05263817
- Testing
- CD19/BCMA CAR T-cells
- Where
- China
- Volunteers
- 75 people
- Sponsor
- Zhejiang University
Sickle Cell Anemia
Carbon Monoxide Measurement to Screen for Sickle Cell Disease
Modify the design of the CoSense device (Model C20112, currently cleared by the FDA for ETCO (end-tidal carbon monoxide) monitoring to improve accuracy and consistency under temperature conditions encountered in countries with…
- Trial name
- SCDCO (NCT02530242)
- Testing
- End-tidal Carbon Monoxide Subjects, End-tidal Carbon Monoxide Controls
- Volunteers
- 32 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Dyskeratosis Congenita, Fanconi Anemia
Assessing Immune Function in Young Patients With Cytopenia That Did Not Respond to Treatment
RATIONALE: Studying biopsy, bone marrow, and blood samples from patients with cytopenia that did not respond to treatment may help doctors learn more about the disease and plan the best treatment.
- Conditions
- Dyskeratosis Congenita, Fanconi Anemia, Myelodysplastic Syndromes, Pearson Marrow-pancreas Syndrome
- Trial name
- NCT00499070
- Testing
- polymerase chain reaction, flow cytometry, immunologic technique
- Where
- 10 sites in Austria, Belgium and 8 more
- Volunteers
- 119 people
- Sponsor
- University Hospital Freiburg
Sickle-Cell Disease with Crisis
Analgesic Effect of Parecoxib Versus Morphine in SCD Patients Presenting to the Emergency Department
Introduction: This study focuses on the treatment of painful crises in Sickle Cell Disease (SCD) patients using Paracoxib, a non-opioid, compared to Morphine.
- Trial name
- PASC (NCT06315920)
- Testing
- Parecoxib, Morphine
- Volunteers
- 226 people, healthy volunteers welcome
- Sponsor
- Oman Medical Speciality Board
Bone Marrow Failure Syndromes, Erythrocyte Disorder
Investigation of the Genetics of Hematologic Diseases
The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases.
- Conditions
- Bone Marrow Failure Syndromes, Erythrocyte Disorder, Leukocyte Disorder, Hemostasis
- Trial name
- NCT02720679
- Where
- United States
- Volunteers
- 1,716 people
- Sponsor
- St. Jude Children's Research Hospital
Sickle Cell Disorders
GenoMed4ALL: Improving SCD Classification and Prognosis by AI
GenoMed4All 'Genomics and Personalized Medicine for all though Artificial Intelligence in Haematological Diseases' aims to advance on individual SCD patients' disease characterisation and to improve the monitoring of patients'…
- Trial name
- GenoMed4ALL (NCT06019208)
- Where
- 5 sites in France, Italy and 2 more
- Volunteers
- 1,000 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Fanconi's Anemia
Phase I Pilot Study of CD34 Enriched, Fanconi's Anemia Complementation Group C Gene Transduced Autologous Peripheral Blood Stem Cell Transplantation in Patients With Fanconi's Anemia
OBJECTIVES: I. Determine the safety of transferring the Fanconi anemia complementation group C (FACC) gene to hematopoietic progenitors by retroviral mediated gene transfer in patients with Fanconi's anemia, complementation group…
- Trial name
- NCT00005896
- Testing
- filgrastim, Autologous stem cells transduced with FACC retroviral vector, Autologous Stem Cell Transplantation
- Volunteers
- 6 people
- Sponsor
- University of Minnesota
Sickle Cell Anemia, Malaria
Sulfadoxine- Pyrimethamine Versus Weekly Chloroquine for Malaria Prevention in Children With Sickle Cell Anemia
Malaria is fatal and increases the risk of death among children with sickle cell anemia. Chemoprophylaxis significantly improves quality of life in these children.
- Trial name
- NCT00399074
- Testing
- sulfadoxine pyrimethamine
- Where
- Uganda
- Volunteers
- 220 people
- Sponsor
- Makerere University
Sickle Cell Disease, Vaso-occlusive Crisis
Study of Erythrocyte Parameters and Hypercoagulability in Sickle Cell Disease (SCD-TGA)
Sickle cell disease (SCD) is an inherited haemoglobinopathy disorder caused by mutations in HBB gene with amino-acid substitution on β globin chain.
- Trial name
- SCD-TGA (NCT05376046)
- Testing
- Erythrocytic parameters and thrombin generation assay measurement
- Where
- France
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- BILLOIR
Leukemia, Lymphoma
Dexamethasone, Intravenous Injection of Human Immunoglobulin, and Increased Infusion of Mononuclear Cells to Reduce Donor Specific Antibodies in Haploidentical Hematopoietic Stem Cell Transplantation
This study tests whether a combination of three treatments - dexamethasone (a steroid), intravenous immunoglobulin (IVIG, a protein that helps the immune system), and an extra dose of donor mononuclear cells - can safely lower…
- Conditions
- Leukemia, Lymphoma, Thalassemia, Aplastic Anemia
- Trial name
- NCT07698080
- Testing
- Intravenous Immunoglobulin, Dexamethasone, Mononuclear Cells
- Volunteers
- 60 people
- Sponsor
- Hematology department of the 920th hospital
Sickle Cell Disease
BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease
This is an open-label, single-arm, multicenter, Phase 1/2 study evaluating the safety and efficacy of the administration of autologous base edited CD34+ HSPCs (BEAM-101) in patients with severe SCD
- Trial name
- BEACON (NCT05456880)
- Testing
- BEAM-101
- Where
- 19 sites in United States
- Volunteers
- 15 people
- Sponsor
- Beam Therapeutics Inc.
Sickle Cell Disease
Sleep Study in Adult Patients With Major Sickle Cell Disease With Paroxysmal Nocturnal Events
Hypothesis is that the occurrence of nocturnal Vaso-Occlusive Crisis (VOC) and priapism in adults might be related to episodes of nocturnal desaturation secondary to a sleep apnea syndrome.
- Trial name
- DREPASOM (NCT02539771)
- Testing
- Polysomnography and blood sample
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hematologic Malignancies
A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders
To investigate the safety of partially matched related human placental-derived stem cells (HPDSC) administered in conjunction with umbilical cord blood (UCB) stem cells from the same donor in subjects with various malignant or…
- Trial name
- HPDSC (NCT00596999)
- Testing
- UCB and HPDSC
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Celgene Corporation
Pulmonary Hypertension, Sickle Cell Disease
Phosphodiesterase Type-5 Inhibitor Therapy in Sickle Cell People With Pulmonary Hypertension
Background: Sickle cell disease (SCD) is a common inherited blood disorder. Many people with SCD are at risk to get pulmonary hypertension (PH).
- Trial name
- NCT03572036
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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