Clinical trials: Sickle Cell Disorder
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
Sickle Cell Disease
PASS of Xromi Comparing Safety and Effectiveness in Children Under 2 Years With Sickle Cell Disease [PRECISE PASS]
This post-authorisation safety and efficacy study (PRECISE PASS) evaluates the use of Xromi® (hydroxycarbamide 100 mg/mL oral solution) in children aged 9 months to under 2 years with sickle cell disease (SCD).
- Trial name
- PRECISE (NCT06923111)
- Testing
- Xromi
- Where
- 12 sites in Germany, United Kingdom
- Volunteers
- 180 people
- Sponsor
- Nova Laboratories Limited
Sickle Cell Disease
Long-term Endurance Training in Sickle Cell Disease Patients: Impact on Clinical Profile, Physical Fitness, and Quality of Life.
Sickle cell disease (SCD), the most common genetic disease worldwide and in France, is an inherited haemoglobinopathy characterised by chronic haemolytic anaemia, vaso-occlusive crisis (VOC), acute pain, and multi-organ damage.
- Trial name
- EXDRE2 (NCT06823219)
- Testing
- endurance training program
- Where
- France
- Volunteers
- 120 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Study to Assess the Effect of Long-term Treatment With Voxelotor in Participants Who Have Completed Treatment in Study GBT440-031
Open Label Extension Study of Voxelotor Clinical Trial Participants with Sickle Cell Disease Who Participated in Voxelotor Clinical Trials
- Trial name
- 034OLE (NCT03573882)
- Testing
- Voxelotor
- Where
- 60 sites in United States, Canada and 9 more
- Volunteers
- 179 people
- Sponsor
- Pfizer
Moya Moya Disease
Moya Moya Syndrome With or Withtout Sickle Cell Disease
Moya Moya disease or syndrome ar characterized by a progressive or occlusion of the intracranial carotid arteries and their mainproximal branches, followed by the development of fragile neovessels at the base of the skull, leding…
- Trial name
- BMM-ScD (NCT05050344)
- Where
- France
- Volunteers
- 50 people
- Sponsor
- University Hospital, Montpellier
Sickle Cell Anemia
SACRED A Prospective Research Study to Reduce Stroke in Children With Sickle Cell Anemia
Prospective screening and treatment study for children with Sickle Cell Anemia and increased stroke risk living in the Dominican Republic.
- Trial name
- SACRED (NCT02769845)
- Testing
- Hydroxyurea, TCD examination
- Where
- Dominican Republic
- Volunteers
- 283 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Sickle Cell Disease
Propanolol and Red Cell Adhesion Non-asthmatic Children Sickle Cell Disease
Propanolol is a beta blocker which has been found to inhibit the ability of epinephrine to upregulate sickle red cell adhesion to laminin and endothelial cells in vitro.
- Trial name
- NCT02012777
- Testing
- propranolol
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Miami
Iron Overload, Excessive Body Iron Burden
Massive Iron Deposit Assessment
Iron overload is a severe complication of multiple blood transfusions. As the body has no physiologic mechanism for clearing iron, repeated transfusions cause iron accumulation in organs and lead to iron toxicity.
- Trial name
- NCT01572922
- Testing
- R2*-UTE, R2*-GRE, Liver biopsy
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- St. Jude Children's Research Hospital
Acute Chest Syndrome, Sickle Cell Disease
Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death.
- Trial name
- INNOSTAPED (NCT01089439)
- Testing
- Nitric oxide by inhalation INOMAX
- Where
- France
- Volunteers
- 21 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease, Thalassemia Major
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD)…
- Trial name
- NCT04523376
- Testing
- CliniMACS
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Timothy Olson
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