Clinical trials: Fetal Hemoglobin
Beta Thalassemia
5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia
This study will evaluate the safety and effectiveness of 5-azacytidine and phenylbutyrate for treating thalassemia major. Patients with this disease have abnormal production of hemoglobin (the oxygen-carrying protein in red blood…
- Trial name
- NCT00005934
- Testing
- 5-Azacytidine
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Transfusion Dependent Beta-thalassemia
Effects of Hydroxyurea and Metformin in Transfusion Dependent Beta-Thalassemia
This clinical trial aims to evaluate and compare the effects and safety of hydroxyurea and metformin in transfusion-dependent beta-thalassemia major and correlate responders/non-responders with the single nucleotide polymorphism…
- Trial name
- NCT06606886
- Testing
- Tablet Metformin (Group A), Tablet Hydroxyurea (Group B)
- Where
- Pakistan
- Volunteers
- 96 people
- Sponsor
- Bahria University
Pregnancy
Néevo®/NéevoDHA® P.L.U.S. Program (Progress Through Learning, Understand & Support)
This is an observational study in which patients who have been prescribed Néevo®/NéevoDHA® are invited to participate in surveys about their pregnancy and experiences with Néevo®/NéevoDHA®.
- Trial name
- NCT01358552
- Testing
- Néevo®/ NéevoDHA®
- Where
- United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Pamlab, Inc.
Fetal Hemoglobin, Beta-Thalassemia
Long Term Beta Thalassemia Treatment: Findings From The Extension Period
The project, titled "Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus…
- Conditions
- Fetal Hemoglobin, Beta-Thalassemia, Hemoglobinopathies
- Trial name
- Thalidomide (NCT06490601)
- Testing
- thalidomide and hydroxyurea, Thalidomide
- Where
- Pakistan
- Volunteers
- 30 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Sickle Cell Anemia
Evaluation of Hydroxyurea Plus L-arginine or Sildenafil to Treat Sickle Cell Anemia
Patients with sickle cell disease have abnormal hemoglobin (the protein in red blood cells that carries oxygen to the body). This abnormality causes red blood cells to take on a sickle shape, producing disease symptoms.
- Trial name
- NCT00056433
- Testing
- Hydroxyurea, L-Arginine, Sildenafil
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Premature Infant Disease, Transfusion Related Complication
Cord Blood Transfusion In Preterm Neonates (CB-TrIP)
Repeated transfusions have been associated with very poor outcome of preterm infants. Fetal hemoglobin (HbF) and adult Hb (HbA) have different affinity for oxygen.
- Conditions
- Premature Infant Disease, Transfusion Related Complication, Fetal Hemoglobin
- Trial name
- CB-TrIP (NCT03764813)
- Where
- 2 sites in Italy
- Volunteers
- 25 people
- Sponsor
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Beta Thalassemia Intermedia
Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand
Beta thalassemia intermedia is an inherited blood disease caused by molecular mutations which reduce the beta globin protein chain of adult hemoglobin A, the protein in red blood cells which carries oxygen throughout the body.
- Trial name
- ST20-P2T (NCT01609595)
- Testing
- sodium 2,2 dimethylbutyrate
- Where
- Thailand
- Volunteers
- 10 people
- Sponsor
- Boston University
Fetal Hemoglobin
The Relevance Between the Level of Fetal Hemoglobin and Short-term Complications in Extremely Preterm Infants
A monocentric prospective study was conducted to find the relevance between the level of fetal hemoglobin and short-term complications in extremely preterm infants
- Trial name
- NCT05788562
- Where
- China
- Volunteers
- 92 people
- Sponsor
- The First Hospital of Jilin University
Mortality in Sickle Cell, Sickle Cell Cardiopulmonary Complications
Observational Study to Deeply Phenotype Major Organs in Sickle Cell Disease After Curative Therapies
Background: People with sickle cell disease (SCD) have problems with their heart, brain, kidneys, liver, and lungs as they age. These problems may improve after transplant. Researchers want to learn how and why this happens.
- Conditions
- Mortality in Sickle Cell, Sickle Cell Cardiopulmonary Complications, Sickle Cell Organ Damage, Sickle Cell Life Expectancy and Risk Factors for Early Death
- Trial name
- NCT05213572
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Anemia, Sickle Cell Disease
Fetal Hemoglobin Induction Treatment Metformin
The purpose of this study is to determine whether metformin is effective in the treatment for sickle cell anemia (SCA).
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Hemoglobin Disorder
- Trial name
- FITMet (NCT02981329)
- Testing
- Metformin, Questionnaires
- Where
- 2 sites in United States
- Volunteers
- 37 people
- Sponsor
- Baylor College of Medicine
Sickle Red Blood Cell, Fetal Hemoglobin
Effect of Broccoli Sprouts Homogenate on SS RBC
The overall purpose of this study is to obtain a better understanding of the biological response of red blood cells to sulforaphane contained in fresh broccoli sprouts that have been put through a blending process.
- Conditions
- Sickle Red Blood Cell, Fetal Hemoglobin, Oxidative Stress
- Trial name
- BSH (NCT01715480)
- Testing
- Broccosprouts® (Brassica Protection Products LLC) homogenate
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Duke University
Infant, Extremely Premature, Erythrocyte Transfusion
Red Blood Cells From Umbilical Cord for Transfusion of Preterm Infants
This study has been designed to demonstrate that red blood cell from umbilical cord blood (UCB-RBC) is a safe and available product for extremely preterm infants (EPI) transfusion and that transfusion of UCB-RBC is non-less…
- Conditions
- Infant, Extremely Premature, Erythrocyte Transfusion, Umbilical Cord Issue
- Trial name
- NCT05612919
- Testing
- Red blood cell from umbilical cord blood, Red blood cell from adult donor
- Where
- Spain
- Volunteers
- 41 people
- Sponsor
- Hospital Clinic of Barcelona
Beta Thalassemia
Association of Hb F Level With Clinical Severity of Beta Thalassemia
The production of Hb F after birth is an important factor in modifying the clinical severity of beta thalassemia because an increased gamma-globin level will bind the additional a-globin and form Hb F.
- Trial name
- NCT04917978
- Testing
- Hematological Phenotype Analysis
- Where
- China
- Volunteers
- 1,200 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Beta Thalassemia, Hemoglobinopathy
Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)
This 12-month study will evaluate the safety and effectiveness of hydroxyurea in treating beta-thalassemia, a type of anemia caused by defective hemoglobin (the oxygen-carrying pigment in blood).
- Trial name
- NCT00001958
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Sickle Cell Disease
A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own…
- Trial name
- GRASP (NCT05353647)
- Testing
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Where
- 9 sites in United States
- Volunteers
- 25 people
- Sponsor
- David Williams
Sickle Cell Disease
Gene Transfer for Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease.
- Trial name
- NCT03282656
- Testing
- single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- David Williams
Sickle Cell Anemia
Gum Arabic as Fetal Hemoglobin Agent in Sickle Cell Anemia
The purpose of this study is to determine whether Gum Arabic is effective as fetal hemoglobin inducing agent for sickle cell anemia patients.
- Trial name
- NCT02467257
- Testing
- Gum Arabic
- Where
- Sudan
- Volunteers
- 47 people
- Sponsor
- Al-Neelain University
Haemoglobin Barts Hydrops
In-utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Bart's Hydrops Fetalis Syndrome
This is a prospective observational pilot study on pregnant women who are diagnosed to have Bart's hydrops fetalis syndrome (BHFS) affected fetuses and opt for continuation of pregnancy will be invited to consider undergoing…
- Trial name
- NCT05797272
- Testing
- in-utero HSCT
- Where
- Hong Kong
- Volunteers
- 10 people
- Sponsor
- Chinese University of Hong Kong
Sickle Cell Disease
Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease
This clinical trial is a study to evaluate the pharmacokinetics of the tablet formulation Pociredir in fasted and fed state participants with Sickle Cell Disease (SCD).
- Trial name
- NCT07431398
- Testing
- Pociredir
- Where
- 6 sites in United States
- Volunteers
- 13 people
- Sponsor
- Fulcrum Therapeutics
Beta-Thalassemia
Testing SIROLIMUS in Beta-thalassemia Transfusion Dependent Patients
Beta-thalassemias are hereditary blood disorders caused by reduced or absent synthesis of hemoglobin beta chains, with variable outcomes ranging from severe anemia to clinically asymptomatic individuals.
- Trial name
- SIRTHALACLIN (NCT03877809)
- Testing
- Sirolimus 0.5 mg
- Where
- 2 sites in Italy
- Volunteers
- 26 people
- Sponsor
- Rare Partners srl Impresa Sociale