Clinical trials: Hereditary Red Blood Cell Disorder (Disorder)
Sickle Cell Disease, Thalassemia
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients
Red Blood Cell - IMProving trAnsfusions for Chronically Transfused recipients (RBC-IMPACT) is an observational cohort study to assess donor, component, and recipient factors that contribute to RBC efficacy in chronically and…
- Conditions
- Sickle Cell Disease, Thalassemia, Pediatric Cancer
- Trial name
- RBC-IMPACT (NCT05255445)
- Testing
- Red Blood Cell (RBC) Transfusion
- Where
- 14 sites in United States, Brazil
- Volunteers
- 157 people
- Sponsor
- Westat
Hemoglobinopathies, Sickle Cell Disease
ERN-EuroBloodNet Registry on Patients With Rare Red Blood Cell Defects and COVID-19
Patients with red blood cell disorders (RBCDs), such as Sickle cell disease (SCD) and Thalassemia, are chronic, life-threatening conditions that can become multi-organ complications over time, and are likely at an increased risk…
- Conditions
- Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia, Sickle Beta Thalassemia
- Trial name
- NCT06831799
- Testing
- Non applicable, is a patient registry
- Where
- Spain
- Volunteers
- 684 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Sickle Cell Disease, Thalassemia
Stem Cell Transplant for Hemoglobinopathy
This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies.
- Conditions
- Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia
- Trial name
- NCT00176852
- Testing
- Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
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
Sickle Cell Disease
Biotin-RBC Transfusion in SCD
This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion…
- Trial name
- NCT04426591
- Testing
- Biotin Labeled Red Blood Cells, Pathogen-reduced Biotin Labeled Red Blood Cells
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Marianne Yee
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
Iron Overload Due to Repeated Red Blood Cell Transfusions, Thalassemia Majors (Beta-Thalassemia Major)
Evaluate the Efficacy and Safety of Irochel for the Management of Iron Overload in Thalassemia Patients in Comparison With Deferoxamine
This research study was an experimental, randomized, controlled clinical trial to evaluate the safety and efficacy of coded Unani formulation Irochel for the management of IOL due to repetitive blood transfusion in comparison…
- Trial name
- NCT07782931
- Testing
- Irochel, Deferoxamine
- Where
- Pakistan
- Volunteers
- 132 people, healthy volunteers welcome
- Sponsor
- Hamdard University
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
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
Retinopathy of Prematurity
umBilical Or Adult Donor Red Blood Cells in Extremely Low Gestational Age Neonates and Retinopathy of Prematurity (BORN)
Extremely low gestational age neonates (ELGAN, i.e., born before 28 gestation weeks) are among the most heavily transfused pediatric patients.
- Trial name
- BORN (NCT05100212)
- Testing
- cord blood-RBC concentrates, adult donor RBC concentrates
- Where
- 10 sites in Italy
- Volunteers
- 146 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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
Hereditary Red Blood Cell Disorder (Disorder)
Luspatercept in Patients Affected With Rare Inherited Anemias
This is a prospective multicenter phase II basket trial evaluating Luspatercept in patients affected with rare inherited anemias
- Trial name
- LUSPARA (NCT07331818)
- Testing
- Reblozyl
- Where
- 6 sites in France, Italy
- Volunteers
- 45 people
- Sponsor
- EuroBloodNet Association
Stomatocytosis
National Exhaustive Cohort of Hereditary Stomatocytoses and Other Channelopathies Affecting the Red Blood Cell
Hereditary stomatocytosis is a heterogeneous group of rare constitutional diseases of dominant transmission in the vast majority of cases.
- Trial name
- COHSTO (NCT04778657)
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Neutropenia, Sickle Cell Disease
Decitabine for High-Risk Sickle Cell Disease
Background: In sickle cell disease (SCD), the proteins in the red blood cells that carry oxygen do not behave normally. In parts of the body where there are low levels of oxygen or where oxygen is used more, the sickle hemoglobin…
- Trial name
- NCT01375608
- Testing
- Decitabine
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Thalassemia
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
This is a clinical trial of bone marrow transplantation for patients with the diagnosis of a genetic disease of blood cells that do not have an HLA-matched sibling donor.
- Conditions
- Sickle Cell Disease, Thalassemia, Anemia, Granuloma
- Trial name
- NCT00730314
- Testing
- Hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Children's Hospital Los Angeles
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
Folate Deficiency, Neural Tube Defects
Folic Acid Salt Study (FISFA Zambia)
Question: How effective is fortified iodized salt with folic acid (FISFA) in increasing serum and red blood cell folate in non-lactating, non-pregnant women of reproductive age in the country of Zambia who do not have active food…
- Trial name
- NCT06734611
- Testing
- Fortified Iodized Salt with Folic Acid (FISFA)
- Where
- 2 sites in United States, Zambia
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
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
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
Sickle Cell Disease
A Study to Compare if the Uptake of Ticagrelor in the Body Differs When Different Tablets Are Administered
To evaluate the relative bioavailability of ticagrelor for the different formulations. A randomized cross-over design has been chosen to minimize the effects of between-subject variability and any period effects on the overall…
- Trial name
- NCT03126695
- Testing
- Ticagrelor granule, Ticagrelor pediatric tablets, Ticagrelor pediatric tablets suspended in water
- Where
- Germany
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- AstraZeneca