Clinical trials: Hemoglobinopathy (Disorder)
Diabetes Mellitus, Heart Disease
Deferoxamine for the Treatment of Hemochromatosis
When patients receive repeated blood transfusions the level of iron in the patient s blood can rise. When iron is processed in the body a protein known as hemosiderin can begin collecting in the organs.
- Conditions
- Diabetes Mellitus, Heart Disease, Hemochromatosis, Thalassemia
- Trial name
- NCT00001203
- Where
- United States
- Volunteers
- 151 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Anemia, Sickle Cell
Establishing a Repository of Blood and DNA Samples From People With Sickle Cell Disease (Comprehensive Sickle Cell Centers Collaborative Genotype-Phenotype Database and Sample Repository)
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited blood disease that can cause intense pain episodes.
- Trial name
- Gen/Phen (NCT00528203)
- Where
- 13 sites in United States
- Volunteers
- 3,640 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease
Interest of Famotidine in Children With Sickle Cell Disease
The purpose of this study is to determine whether oral famotidine, a histamine type 2 receptor antagonist already widely used with very few side effects in other indications in children, is effective in reducing endothelial…
- Trial name
- FAMODREP (NCT05084521)
- Testing
- Famotidine 400 mg/50 mL
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies
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.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
- Trial name
- NCT06647979
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Daniel Bauer
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
Sickle Cell Anemia, Sickle Cell Thalassemia
Clinical and Laboratory Characteristics of Sickle Cell Anemia Patients Admitted With Fever
This study will summarized the clinical and laboratory data and the outcome of all the patients suffering from Sickle Cell Anemia (Including Sickle cell thalassemia) admitted to the pediatric ward.
- Trial name
- NCT00736060
- Testing
- Observational
- Where
- Israel
- Volunteers
- 60 people
- Sponsor
- HaEmek Medical Center, Israel
Thalassemia, Sickle Cell Anemia
Blood Transfusions in Thalassemia Patients, Complications and Adverse Effects
Patients suffering from Thalassemia or another hemoglobinopathies required regular blood transfusions. The complications and adverse effects of blood transfusions can be classified as immediate and late.
- Trial name
- NCT00971880
- Testing
- Clinical medical record summary
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- HaEmek Medical Center, Israel
Bone Marrow Transplantation, Sickle Cell Disease
Sickle Cell Disease Conditioning for Bone Marrow Transplant
Most bone marrow transplants for children with sickle cell disease are performed using high doses of two chemotherapy agents: busulfan and cyclophosphamide for the pre-transplant conditioning.
- Trial name
- NCT00968162
- Testing
- fludarabine
- Where
- 10 sites in United States
- Volunteers
- 8 people
- Sponsor
- Emory University
Sickle Cell Disease, Thalassemia
Effects of Transfusion of Older Stored Red Cells
The purpose of this study is to determine the effects of transfusion of fresh and stored blood on patients. The investigators hope to test: whether a similar effect (older stored blood is associated with worse outcomes) is seen…
- Trial name
- NCT01534676
- Testing
- Transfusion, Blood, Chelation therapy
- Where
- United States
- Volunteers
- 3 people, healthy volunteers welcome
- Sponsor
- Columbia University
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Hematopoietic Stem Cell BCL11A Enhancer Gene Editing 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 deoxyribonucleic acid (DNA)) from the patient to treat his…
- Trial name
- NCT07708350
- Testing
- autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Daniel Bauer
Sickle Cell Anemia, Sickle Cell Disease
Novel Use Of Hydroxyurea in an African Region With Malaria
Multiple studies have shown that hydroxyurea has clinical efficacy in preventing acute painful episodes and reducing the need for blood transfusions in children with sickle cell anemia (SCA), but no study has been conducted in…
- Conditions
- Sickle Cell Anemia, Sickle Cell Disease, Malaria
- Trial name
- NOHARM (NCT01976416)
- Testing
- Hydroxyurea
- Where
- Uganda
- Volunteers
- 208 people
- Sponsor
- Indiana University
Iron Overload, Oxidative Stress
Hydroxy Urea, Omega 3, Nigella Sativa,Honey on Oxidative Stress and Iron Chelation in Pediatric Major Thalassemia
The aim of the present study is evaluating the strength of combination therapy of hydroxy urea, omega 3, nigella sativa and honey on antioxidant-oxidant status (OXIDATIVE STRESS) in response to reactive oxygen species production…
- Conditions
- Iron Overload, Oxidative Stress, Thalassemia Major
- Trial name
- NCT04292314
- Testing
- Omega 3, Nigella Sativa Oil, Hydroxyurea
- Where
- 4 sites in Egypt, Saudi Arabia
- Volunteers
- 350 people
- Sponsor
- Beni-Suef University
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML)
CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant
The purpose of this study is to learn more about the effects of (classification determinant) CD34+ stem cell selection on graft versus host disease (GVHD) in children, adolescents, and young adults.
- Conditions
- Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML)
- Trial name
- NCT02061800
- Testing
- CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Diane George
Sickle Cell Disease, Sickle Beta Zero Thalassemia
An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease
The primary objective of this study is to evaluate a potential behavioral intervention (MED-Go app). To meet this objective, the researchers will conduct a pilot randomized controlled trial to test the feasibility and…
- Conditions
- Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
- Trial name
- NCT04688411
- Testing
- MED-Go App
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Thalassemia Major
Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital
Thalassemia is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha \& Beta thalassemia.
- Trial name
- NCT03161899
- Where
- Egypt
- Volunteers
- 246 people
- Sponsor
- Assiut University
Thalassemia, Iron Overload
Efficacy and Safety of Desferal Versus Osveral in Transfusional Iron Overload
Thalassemic patients often suffer from iron overload due to frequent blood transfusion. Oral iron chelators reduce iron overload in transfusion dependent patients.
- Conditions
- Thalassemia, Iron Overload, Transfusion Related Complications
- Trial name
- NCT01369719
- Testing
- osveral, Desferal
- Where
- Iran
- Volunteers
- 138 people
- Sponsor
- Hormozgan University of Medical Sciences
Sickle Cell Disease, Sickle Cell Hemoglobin C
Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study
The overarching goal of this proposal is to identify modifiable behavioral strategies based on patient-reported outcomes (PROs) and health-related quality of life (HRQOL) that will improve hydroxyurea (HU) adherence among…
- Conditions
- Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
- Trial name
- NCT04678037
- Testing
- PROs assessment using sickle cell disease mobile app (SCD-app)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Sickle Cell Disease
Patient and Public Involvement and Engagement in Research With Children and Young People With Sickle Cell Disorder and Their Families
Aim: To co-produce resources for inclusive and equitable Patient and Public Involvement and Engagement in research on life-limiting conditions, with children and young people with sickle cell disorder and their families.
- Trial name
- NCT06293222
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- King's College London
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
Sickle Cell Disease
Exploring Adherence Monitoring in Sickle Cell Disease
Despite the well-documented benefits of hydroxyurea (HU) therapy in decreasing morbidity and mortality in youth with Sickle cell disease (SCD), pediatric HU adherence rates range as low as 49% and lead to discontinuation of HU…
- Trial name
- NCT03708731
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital