Phase 3 N=30 Stem Cell Transplantation for Sickle Cell Anemia
Sickle Cell Disease
Primary: Number of Participants With Graft Failure — 3 Participants
Clinical Trial Search
Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.
Phase 2 N=100 A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)
Sickle Cell Disease
Primary: Number Of Participants With Treatment-emergent Adverse Events (TEAEs) And Serious Adverse Events (SAEs) — 12; 24; 18; 23 Participants
N/A N=112 Sickle Cell Anemia WE CARE
Sickle Cell Disease
Primary: Number of Emergency Department (ED)/Acute Care Visits — 0; 1 number of visits
Phase 2 N=7 A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
Beta-Thalassemia Major · Sickle Cell Disease
Primary: Number of Treated Participants With Successful Neutrophil and Platelet Engraftment — 3; 4; 3; 4 Participants
Phase 2 N=29 HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity
Sickle Cell Disease · Beta Thalassemia-Major
Primary: Number of Participants With Sustained Cell Engraftment of Donor Cells — 29 Participants
Phase 2 N=30 Evaluating the Safety and Effectiveness of Bone Marrow Transplants in Children With Sickle Cell Disease (BMT CTN 0601)
Sickle Cell Disease
Primary: Percentage of Participants With Event-Free Survival (EFS) — 76; 69 percentage of participants
N/A N=145 Evaluation of Low-cost Techniques for Detecting Sickle Cell Disease and β-thalassemia in Nepal and Canada
Sickle Cell Disease · Sickle Cell Trait · Beta-Thalassemia
Primary: Sensitivity, Specificity, Positive Predictive Value and Negative Predictive Value — 96.6; 100; 0; 91.3 percentage
Phase 2 N=95 Haploidentical Bone Marrow Transplantation in Sickle Cell Patients (BMTCTN1507)
Sickle Cell Disease
Primary: Percentage of Participants With Two-Year Post-Transplant Event Free Survival (EFS) — 88.0; 79.3 Percentage of Participants
N/A N=163 Long-Term Effects of Hydroxyurea in Children With Sickle Cell Anemia (The BABY HUG Follow-up Study)
Anemia, Sickle Cell
Primary: Change in Qualitative Spleen Function From Randomized Control Trial Baseline Measurement - Compared Between Children Randomized to Hydroxyurea vs Placebo — 16; 15; 44…
Phase 2 N=10 Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease
Sickle Cell Disease
Primary: Fetal Hemoglobin Level — 8.3 Percentage
Phase 2 N=6 Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
Phase 3 N=22 Stem Cell Transplant for Hemoglobinopathy
Sickle Cell Disease · Thalassemia · Severe Congenital Neutropenia
Primary: Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity — 0; 0; 2 Participants
Phase 2 N=70 L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia
Sickle Cell Anemia · Thalassemia
Primary: Number of Occurrences of Painful Sickle Cell Crises — 4.5; 10.8 Crisis — p=0.076
N/A N=8 Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
Sickle Cell Disease · Thalassemia Major
Primary: Rate of Graft Failure — 2; 0 participants
Phase 1 N=11 Aspirin Prophylaxis in Sickle Cell Disease
Sickle Cell Disease
Primary: Number of Serious Adverse Events — 6; 9; 0; 0 events
Phase 3 N=24 A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
Beta-Thalassemia
Primary: Percentage of Participants Who Meet the Definition of Transfusion Independence (TI) — 91.3 Percentage of participants
Phase 2 N=22 Bone Marrow Transplantation in Young Adults With Severe Sickle Cell Disease
Sickle Cell Disease
Primary: Event -Free Survival Rate — 19 Participants
Phase 3 N=19 A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia
Beta-Thalassemia
Primary: Percentage of Participants Who Have Achieved Transfusion Independence (TI) — 88.9 Percentage of participants
N/A N=1,906 European Sickle Cell Disease Cohort - Hydroxyurea
Sickle Cell Disease
Primary: % of Patient-years With Malignancies — 0.001 % patient-years
Phase 2 N=16 Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Sickle Cell Disease & Thalassemia
Primary: Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity. — 0; 0; 3; 0 participants
Early Phase 1 N=22 Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease
Sickle Cell Disease · Sickle Cell Anemia
Primary: Mean Red Blood Cells Lifespan in Participants — 64.1; 113.4; 126.0; 123.7 day — p=<0.001
Phase 2 N=10 Transplantation for Patients With Sickle Cell Disease From Mismatched Family Donors of Bone Marrow
Sickle Cell Disease
Primary: Event-free Survival (EFS) Rate — 5 Participants
Phase 2 N=19 A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
β-thalassemia Major
Primary: Percentage of Participants With Sustained Production of >=2.0 Grams Per Deciliter (g/dL) of Hemoglobin A (HbA) Containing βA-T87Q-globin (HbAT87Q) for the Six Months…
Phase 3 N=230 A Phase III Safety and Efficacy Study of L-Glutamine to Treat Sickle Cell Disease or Sickle βo-thalassemia
Sickle Cell Anemia · Sickle ß0-Thalassemia
Primary: The Number of Occurrences of Sickle Cell Crises — 3; 4 Number of crises — p=0.0052
Phase 2 N=123 Bone Marrow Transplantation vs Standard of Care in Patients With Severe Sickle Cell Disease (BMT CTN 1503)
Sickle Cell Disease
Primary: Percentage of Participants With Overall Survival (OS) at 2 Years After Biologic Assignment — 89; 94 percentage of participants
Phase 3 N=208 Novel Use Of Hydroxyurea in an African Region With Malaria
Sickle Cell Anemia · Sickle Cell Disease · Malaria
Primary: Number of Malaria Episodes — 5; 7 malaria episodes
Phase 1 N=15 Peripheral Blood Stem Cell Collection for Sickle Cell Disease (SCD) Patients
Sickle Cell Disease
Primary: Number of Participants With Sufficient Collection of Hemopoietic Stem Cells (HSCs) Without Serious Adverse Events — 14 Participants
Phase 2 N=10 Decitabine for High-Risk Sickle Cell Disease
Neutropenia · Sickle Cell Disease
Primary: The Percentage Change in HbF Level From Baseline to the Average Over the Final 1 Month of Study. — 1; 1; 6 Participants
Early Phase 1 N=17 A Dose-Finding Study of AG-348 in Sickle Cell Disease
Sickle Cell Disease
Primary: Number Participants With Most Common Reported Drug Related Adverse Events — 6; 3; 3 Participants
N/A N=212 Examining Cognitive Function and Brain Abnormalities in Adults With Sickle Cell Disease
Anemia, Sickle Cell
Primary: Wechsler Adult Intelligence Scale (WAIS)-III Performance IQ — 90.6; 95.9 Points on a scale — p=0.008