Clinical trials: Hereditary Red Blood Cell Disorder (Disorder), active
Sickle Cell Disease, Thalassemia
RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)
Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, Anemia Due to Membrane Defect
- Trial name
- RADeep (NCT06213402)
- Testing
- Data collection from EHR.
- Where
- Spain
- Volunteers
- 32,564 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Bone Marrow Failure Syndrome, Severe Aplastic Anemia
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
This study's goal is to determine the frequency and severity of acute graft versus host disease, to evaluate incidence of primary and secondary graft rejection, to assess event free survival and overall survival, to determine the…
- Conditions
- Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia
- Trial name
- NCT01966367
- Testing
- CD34 Stem Cell Selection Therapy
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Diane George
Red Cell Alloantibodies, Blood Transfusion-Dependent
Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients
Transfusion-dependent patients, particularly those with β-thalassemia major, require lifelong regular red blood cell (RBC) transfusions to maintain adequate hemoglobin levels and prevent severe anemia.
- Conditions
- Red Cell Alloantibodies, Blood Transfusion-Dependent, Beta Thalassemia
- Trial name
- NCT07480980
- Volunteers
- 100 people
- Sponsor
- Assiut University
Sickle Cell Disease (SCD)
Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients
The purpose of this study is to compare the efficacy and safety of 2 doses of crizanlizumab (5.0 mg/kg and 7.5 mg/kg) versus placebo in adolescent and adult sickle cell disease (SCD) patients with history of vaso-occlusive crisis…
- Trial name
- STAND (NCT03814746)
- Testing
- Crizanlizumab (SEG101)
- Where
- 58 sites in United States, Belgium and 19 more
- Volunteers
- 253 people
- Sponsor
- Novartis Pharmaceuticals
Neonatal Sepsis, Sepsis
CBC Indices and Serum Lactate in Neonatal Sepsis
Neonatal sepsis is a leading cause of illness and death in Neonatal Intensive Care Units (NICUs). Diagnosing it quickly is challenging because the early signs often overlap with other common newborn health issues.
- Trial name
- NCT07663084
- Volunteers
- 80 people
- Sponsor
- Assiut University
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC
This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- PRO-RIC (NCT04528355)
- Testing
- data collection
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Paul Szabolcs
Sickle Cell Disease
Evaluation of the Impact of Red Blood Cell Exchange on Thrombo-inflammation in Sickle Cell Disease
Sickle cell disease is the most common inherited blood disorder worldwide. It is a hemoglobinopathy characterized by chronic hemolysis, endotheliopathy, coagulation activation, and chronic inflammation.
- Trial name
- STIREX (NCT06769139)
- Testing
- Red blood cell exchange
- Where
- France
- Volunteers
- 20 people
- Sponsor
- University Hospital, Toulouse
Sickle Cell Disease
A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)
A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult…
- Trial name
- SPARKLE (NCT06439082)
- Testing
- Crizanlizumab
- Where
- 36 sites in United States, Brazil and 3 more
- Volunteers
- 354 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Study to Understand the Genetic Risk of Developing an Immune Response After Blood Transfusions Among Individuals With Sickle Cell Disease
The purpose of this research study is to look at genes and determine how they interact with each other to find changes that could explain why some people's immune systems may respond to blood transfusions.
- Trial name
- NCT06944067
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Beta-thalassemia
A Study to Evaluate Luspatercept (ACE-536) in Chinese Participants Who Require Regular Red Blood Cell Transfusions Due to Beta (β)-Thalassemia.
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of luspatercept plus best supportive care (BSC) versus placebo plus BSC in participants who require regular red blood cell transfusions due to…
- Trial name
- NCT05567458
- Testing
- Luspatercept
- Where
- 10 sites in China
- Volunteers
- 94 people
- Sponsor
- Bristol-Myers Squibb
Beta-Thalassemia
Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.
- Trial name
- NCT04143724
- Testing
- ACE-536
- Where
- 26 sites in United States, China and 7 more
- Volunteers
- 99 people
- Sponsor
- Celgene
Anemia, Blood Disease
Diamond Blackfan Anemia Registry (DBAR)
The purpose of this study is to maintain a comprehensive registry of patients with the rare inherited bone marrow failure syndrome Diamond Blackfan anemia (DBA).
- Trial name
- DBAR (NCT00106015)
- Where
- United States
- Volunteers
- 900 people
- Sponsor
- Northwell Health
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
Anemia
A Study to Evaluate the Safety and Effectiveness of Luspatercept for the Treatment of Transfusion-dependent (TD) Anemia Associated With Myelodysplastic Syndromes (MDS) & Beta-thalassemia (β-Thal) in India
The purpose of this study is to evaluate the safety and effectiveness of luspatercept in participants who require regular blood cell transfusions due to b-thalassemia and myelodysplastic syndromes in India
- Trial name
- NCT05891249
- Testing
- Luspatercept
- Where
- 9 sites in India
- Volunteers
- 85 people
- Sponsor
- Bristol-Myers Squibb
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Glanzmann Thrombasthenia
The Genetics and Functional Basis of Inherited Platelet, White Blood Cell, Red Blood Cell, and Blood Clotting Disorders.
Blood contains red blood cells, white blood cells, and platelets, as well as a fluid portion termed plasma. We primarily study blood platelets, but sometimes we also analyze the blood of patients with red blood cell disorders…
- Trial name
- NCT00230165
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Rockefeller University
Hereditary Red Blood Cell Disorder (Disorder)
Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies
This is a prospective exploratory multicentre pilot study designed to study the safety and efficacy of mitapivat in RBC membranopathies and CDAII
- Trial name
- SATISFY (NCT05935202)
- Testing
- Mitapivat sulfate
- Where
- 2 sites in Denmark, Netherlands
- Volunteers
- 25 people
- Sponsor
- EuroBloodNet Association
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
Retinopathy of Prematurity (ROP), Bronchopulmonary Dysplasia (BPD)
The Correlation Between Red Cell Transfusion and Complications of Prematurity
The aim of this clinical trial is to learn if there is a correlation between the erythrocyte transfusion in the early neonatal period in premature infants and early and late complications of prematurity.
- Conditions
- Retinopathy of Prematurity (ROP), Bronchopulmonary Dysplasia (BPD), Enterocolitis, Necrotizing, Intraventricular Hemorrhage Neonatal
- Trial name
- NCT07123948
- Testing
- Wechsler Intelligence Test, performed by educated psychologists
- Where
- Croatia
- Volunteers
- 128 people
- Sponsor
- Clinical Hospital Center Rijeka
Sickle Cell Disease, Stem Cell Transplant Complications
Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor
The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a…
- Conditions
- Sickle Cell Disease, Stem Cell Transplant Complications, Red Blood Cell Disorder, Pure Red Cell Aplasia
- Trial name
- Sickle-AID (NCT03214354)
- Testing
- Alemtuzumab, Total Body Irradiation, Sirolimus
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- University of Calgary