Clinical trials: Sickle Cell Disorder
Sickle Cell Disease
Stem Cell Transplantation for Sickle Cell Anemia
This protocol will be investigating the use of stem cell transplantation, in related donors, to cure sickle cell disease. Sickle cell disease is a recessive disorder caused by a point mutation that results in the substitution of…
- Trial name
- NCT01877837
- Testing
- Alemtuzumab, Fludarabine, Melphalan
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Hackensack Meridian Health
Sickle Cell Disease
Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody
This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell…
- Trial name
- SUN-RAY (NCT06358638)
- Testing
- Daratumumab, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Children's National Research Institute
Sickle Cell Disease Without Crisis
The Impact of Oxidative Stress on Erythrocyte Biology
This study will address if red blood cells transfused to a sickle cell patient from a donor with a glucose-6-phosphate-dehydrogenase (G6PD) enzyme deficiency have a different lifespan as measured by the percentage of red blood…
- Trial name
- RBC Survival (NCT04028700)
- Testing
- G6PD Deficient Red Blood Cell Transfusion, Non-G6PD deficient Red Blood Cell Transfusion
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- University of North Carolina, Chapel Hill
Cancer, Sickle Cell Thalassemia
Autologous Testicular Tissue Transplantation
Freezing testicular tissue of prepubertal boys is a method for preserving spermatogonial stem cells in case of imminent gonadotoxic treatment during childhood.
- Conditions
- Cancer, Sickle Cell Thalassemia, Hematologic Diseases
- Trial name
- NCT05414045
- Testing
- Autologous testicular tissue transplantation of prepubertal frozen testicular tissue
- Where
- Belgium
- Volunteers
- 5 people
- Sponsor
- Universitair Ziekenhuis Brussel
Sickle Cell Disease
Early Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation
This study aims to enroll 58 pre-adolescent (<13 years) pediatric participants with sickle cell disease (SCD) who have a pre-adolescent sibling bone marrow donor.
- Trial name
- NCT04018937
- Testing
- Alemtuzumab, Fludarabine, Melphalan
- Where
- 15 sites in United States, Canada
- Volunteers
- 43 people
- Sponsor
- Emory University
Sickle Cell Disease, Acute Chest Syndrome
Efficacy and Safety of Tocilizumab for Acute Chest Syndrome Treatment in Patients With Sickle Cell Disease
The purpose of this study is to determine whether a single infusion of tocilizumab is effective in reducing the time to successful weaning from both supplemental oxygen and any respiratory support, in pediatric and adult patients…
- Trial name
- TOCIACS (NCT06818266)
- Testing
- Tocilizumab (RoActemra®, 20 mg/mL).
- Where
- France
- Volunteers
- 130 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Pulmonary Hypertension, Sickle Cell Disease
Macitentan in Pulmonary Hypertension of Sickle Cell Disease
This is a pilot study to assess the safety and efficacy of macitentan in patients with pulmonary hypertension of sickle cell disease. This study will enroll approximately 10 subjects.
- Trial name
- MENSCH (NCT02651272)
- Testing
- macitentan
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Boston University
Sickle Cell Disease
Transfusion Alternatives Pre-operatively in Sickle Cell Disease (TAPS)
TAPS is a sequential trial which aims to investigate whether the administration of a blood transfusion pre-operatively to patients with sickle cell disease (HB SS or Hb SB0 thal)having low or medium risk elective surgery…
- Trial name
- TAPS (NCT00512577)
- Testing
- Red blood cell transfusion
- Where
- United Kingdom
- Volunteers
- 70 people
- Sponsor
- NHS Blood and Transplant
Sickle Cell Disease
Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
The objective of this Expanded Access Protocol (EAP) is to provide voxelotor for the treatment of sickle cell disease (SCD): The study GBT440-041 is the EAP for pediatric patients aged 6 months to 11 years who have no alternative…
- Trial name
- NCT04724421
- Testing
- Voxelotor
- Where
- 37 sites in United States, Brazil
- Sponsor
- Pfizer
Phase 1 Sickle Cell
Safety, Tolerability, Pharmacokinetics, And Pharmacodynamics Study Of PF-04447943, Co-Administered With And Without Hydroxyurea, In Subjects With Stable Sickle Cell Disease
This study is being conducted to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of an investigational drug, PF-04447943, in subjects with stable sickle cell disease with and without co-administration with…
- Trial name
- NCT02114203
- Testing
- PDE9i
- Where
- 23 sites in United States, Belgium and 3 more
- Volunteers
- 30 people
- Sponsor
- Pfizer
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
Sickle Cell Disease
Safety, Pharmacokinetic, and Pharmacodynamic Study of NKTT120 in Adult Patients With Stable Sickle Cell Disease (SCD)
The purpose of this study is to determine the safety, pharmacokinetics, and pharmacodynamics of NKTT120 in adult patients with stable sickle cell disease.
- Trial name
- NCT01783691
- Testing
- NKTT120
- Where
- 6 sites in United States
- Volunteers
- 21 people
- Sponsor
- NKT Therapeutics
Healthy, Sickle Cell Disease
Study of Methodologies to Measure Blood Flow and Oxygenation in Adults With Sickle Cell Disease
The purpose of the study is to determine whether imaging techniques, such as magnetic resonance imaging (MRI), near infrared spectroscopy (NIRS), laser speckle contrast imaging (LSCI), and optical imaging (OI), can detect…
- Trial name
- NCT02447627
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Bioverativ Therapeutics Inc.
Brain Development Abnormality, Sickle Cell Disease
Brain Structure and Neurocognitive Development in Sickle Cell Disease; a Longitudinal Cohort Study (BRICK Study)
Sickle cell disease (SCD) is an autosomal recessive red blood cell blood disorder. One especially vital organ affected in SCD is the brain.
- Trial name
- BRICK (NCT05564845)
- Testing
- MR scan of the brain, Blood work, Neurological examination
- Where
- 2 sites in Netherlands
- Volunteers
- 84 people
- Sponsor
- Dr. Marjon H. Cnossen MD PhD
Anemia, Sickle Cell, Hematologic Diseases
Pediatric Hydroxyurea in Sickle Cell Anemia (PED HUG)
To determine whether hydroxyurea prevents the onset of chronic end organ damage in young children with sickle cell anemia.
- Conditions
- Anemia, Sickle Cell, Hematologic Diseases, Hemoglobinopathies
- Trial name
- NCT00000602
- Testing
- hydroxyurea
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sickle Cell Disease, Pregnancy Related
Prophylactic Transfusion In Pregnant in Women With Sickle Cell Disease
The goal of this study is to determine if there is a positive effect of prophylactic red blood cell (RBC) transfusion of leukoreduced, ABO, Rh (D/Cc/Ee) and Kell matched blood compared to standard of care on the number of…
- Trial name
- ProTIP (NCT06979492)
- Testing
- Prophylactic Transfusion Intervention group: Transfusion, Control group
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- Emory University
Sickle Cell Disease
Pharmacokinetics, Pharmacodynamics and Safety of Epeleuton in Patients With Sickle Cell Disease
To assess the pharmacokinetics, pharmacodynamics and safety of Epeleuton capsules in adult SCD patients who are aged ≥18 years.
- Trial name
- NCT05861453
- Testing
- Epeleuton
- Where
- 18 sites in United States, Canada
- Volunteers
- 35 people
- Sponsor
- Afimmune
Sickle Cell Disease, Sickle Cell Anemia
Alendronate for Osteonecrosis in Adults With Sickle Cell Disease
A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Osteonecrosis, Ischemic Necrosis
- Trial name
- NCT06016634
- Testing
- Alendronate Sodium
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- University of California, Davis
Sickle Cell Disease
Acupuncture for Adults With Sickle Cell Disease (SCD): A Feasibility Study
The investigators long-term goal is to demonstrate the effectiveness of acupuncture for the treatment of adults with chronic pain due to sickle cell disease (SCD), a debilitating pain syndrome characterized by acute and chronic…
- Trial name
- NCT04156399
- Testing
- Acupuncture
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Illinois Chicago
Anemia, Sickle Cell, Cerebral Embolism and Thrombosis
Stroke Prevention in Sickle Cell Anemia (STOP 1)
To reduce episodes of first time stroke by 75 percent in children with sickle cell anemia by the administration of prophylactic transfusion therapy.
- Conditions
- Anemia, Sickle Cell, Cerebral Embolism and Thrombosis, Cerebrovascular Disorders, Hematologic Diseases
- Trial name
- NCT00000592
- Testing
- blood transfusion
- Sponsor
- Augusta University
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