Clinical trials: Sickle Cell Disorders
Sickle Cell Anemia, Cerebrovascular Accident
Allogeneic Genoidentical Stem Cell Transplantation in Children With Sickle-cell Anemia and Cerebral Vasculopathy
The aim of this study is to demonstrate that cerebral velocities assessed by transcranial doppler (TCD) are more significantly decreased by SCT than by long-term transfusion program A multicenter, national, non-randomized…
- Trial name
- DREPAGREFFE (NCT01340404)
- Testing
- Stem cell transplantation, Transfusion program
- Where
- France
- Volunteers
- 63 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
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
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Sickle Cell Disease
Sickle-cell Disease Registry of the GPOH
Sickle cell disease is one of the most common hereditary diseases. Most severe complications can be avoided if the disease is detected early and treated appropriately.
- Trial name
- SichReg (NCT03327428)
- Where
- Germany
- Volunteers
- 1,000 people
- Sponsor
- University Hospital Heidelberg
Hemoglobinopathies, Anemia, Sickle Cell
Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia
The purpose of this study is to find out if using a lower dose of chemotherapy before stem cell transplantation can cure patients of sickle cell anemia or thalassemia while causing fewer severe side effects than conventional high…
- Conditions
- Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia
- Trial name
- NCT00034528
- Testing
- Busulfan, Fludarabine, FK506
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Transfusional Iron Overload, Beta-thalassemia
Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload
The purpose of this research study is to evaluate the safety of two doses of FBS0701, a new oral iron chelator, and its effectiveness in clearing iron from the liver.
- Trial name
- NCT01186419
- Testing
- SPD602 (FBS0701, SSP-004184)
- Where
- 9 sites in United States, Italy and 3 more
- Volunteers
- 51 people
- Sponsor
- Shire
Sickle Cell Disease
HLA Haploidentical Bone Marrow Transplant in Patients With Severe Sickle Cell Disease
multicentric interventional biomedical research phase II, prospective, non-randomized evaluating a haploidentical marrow transplants after reduced-intensity conditioning and prevention of GvHD based on cyclophosphamide…
- Trial name
- DREPHAPLO (NCT03240731)
- Testing
- bone marrow transplant
- Where
- 7 sites in France
- Volunteers
- 25 people
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Healthy Volunteers, Sickle Cell Disease
A Study on the Effect of Etavopivat on Heart Rhythm in Healthy Participants
Novo Nordisk is developing a new study medicine, Etavopivat, to treat individuals with sickle cell disease (SCD). The purpose of the study is to determine the effect of Etavopivat on the electrical activity of the heart in…
- Conditions
- Healthy Volunteers, Sickle Cell Disease, Thalassemia
- Trial name
- NCT07023029
- Testing
- Etavopivat, Moxifloxacin
- Where
- United States
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease
Inflammatory Markers and Pain in Sickle Cell Disease
This observational cross-sectional study aims to assess the relationship between inflammatory markers and pain intensity in adult patients with sickle cell disease.
- Trial name
- NCT07865039
- Where
- Egypt
- Volunteers
- 75 people
- Sponsor
- Assiut 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
Graft Versus Host Disease
Acute GVHD Suppression Using Costimulation Blockade to Expand Non-malignant Transplant
This trial will see if extended abatacept administration (combined with a standard regimen of tacrolimus and mycophenolate mofetil) will prevent acute and chronic graft-versus-host disease (GVHD) in children and adolescents…
- Trial name
- ASCENT (NCT03924401)
- Testing
- Abatacept
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- Emory University
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
Study of Propranolol as Anti-Adhesive Therapy in Sickle Cell Disease (SCD)
An open label, prospective, randomized cross-over phase II study in up to 60 sickle cell patients who are either homozygous for Hb S or have HbSB0 thalassemia.
- Trial name
- NCT01077921
- Testing
- Propranolol
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Laura M. De Castro, MD
Sickle Cell Disease
School Readiness Intervention for Preschool Children With Sickle Cell Disease
The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease.
- Trial name
- NCT06367192
- Testing
- The adapted Kids in Transition to School Intervention, Standard school resources
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- St. Jude Children's Research Hospital
Healthy Volunteers, Anemia, Sickle Cell
A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease
The purpose of the study is to assess the safety and tolerability of AG-946 in healthy volunteers after oral administration of single ascending doses (SAD) and multiple ascending doses (MAD) of AG-946 over 14 or up to 28 days of…
- Trial name
- NCT04536792
- Testing
- AG-946
- Where
- 10 sites in United States, Denmark and 1 more
- Volunteers
- 122 people, healthy volunteers welcome
- Sponsor
- Agios Pharmaceuticals, Inc.
Sickle Cell Anemia in Children
Hydroxyurea Therapy for Neurological and Cognitive Protection in Pediatric Sickle Cell Anemia in Uganda ( BRAINSAFE-II )
Worldwide, an estimated 200,000 babies are born with Sickle Cell Anemia (SCA) annually. Affected children suffer chronic ill health with some having frequent hospitalization.
- Trial name
- BRAINSAFEII (NCT04750707)
- Testing
- Hydroxyurea
- Where
- Uganda
- Volunteers
- 270 people
- Sponsor
- Global Health Uganda LTD
Sickle Cell Disease
Severe Acute Respiratory Syndrome CoV 2 COVID-19 Survey and Vaccination Coverage in the Sickle Cell Population in Ile-De-France
The objective of this study is to determine the seroprevalence of severe acute respiratory syndrome-CoV-2 in unvaccinated sickle cell patients living in an area with high viral circulation and at risk of high viral transmission…
- Trial name
- COVIDO-DREP (NCT05153044)
- Testing
- severe acute respiratory syndrome CoV-2 serology
- Volunteers
- 880 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Sickle Cell Disease
Integrative Training Program for Pediatric Sickle Cell Pain
This research aims to answer the question: does a group training program specifically for teens with chronic sickle cell disease (SCD) pain that teaches skills to strengthen the mind and body help improve everyday functioning and…
- Trial name
- I-STRONG SCD (NCT06691867)
- Testing
- I-STRONG, Enhanced Usual Care (EUC)
- Where
- 4 sites in United States
- Volunteers
- 155 people
- Sponsor
- Emory University
Sickle Cell Disease, Sickle Cell Anemia
Risk Clinical Stratification of Sickle Cell Disease in Nigeria, Assessment of Efficacy/Safety of Hydroxyurea Treatment
The vast majority of births with sickle cell disease (SCD) occur in Africa and 90% are thought to die before the age of five. Hydroxyurea (HU) is the only drug approved by the FDA for the treatment of sickle cell anemia.
- Trial name
- NCT02149537
- Testing
- hydroxyurea
- Where
- Nigeria
- Volunteers
- 53 people
- Sponsor
- Loyola University
Anemia, Sickle Cell, Transfusion Hemosiderosis
Clinical Importance of Treating Iron Overload in Sickle Cell Disease
Hypothesis: The investigators suspect that significant degrees of iron overload in subjects with SCD result in decreased red cell survival, abnormal endothelial function and markedly dysregulated autonomic function.
- Trial name
- NCT00981370
- Testing
- deferasirox
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Children's Hospital Los Angeles
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