30 closest matches · ranked by relevance
Sickle Cell Disease & Thalassemia
Primary: Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity. — 0; 0; 3; 0 participants
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
Sickle Cell Disease · Thalassemia · Severe Congenital Neutropenia
Primary: Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity — 0; 0; 2 Participants
Hemoglobinopathies · Sickle Cell Disease · Thalassemia
Primary: Stable Engraftment With Donor Stem Cells in Patients With Severe Hemoglobinopathy. — 2 participants
Sickle Cell Disease
Primary: Number of Emergency Department (ED)/Acute Care Visits — 0; 1 number of visits
Sickle Cell Disease
Primary: Number of Participants With Graft Failure — 3 Participants
Beta-Thalassemia Major · Sickle Cell Disease
Primary: Number of Treated Participants With Successful Neutrophil and Platelet Engraftment — 3; 4; 3; 4 Participants
Sickle Cell Disease · Beta Thalassemia-Major
Primary: Number of Participants With Sustained Cell Engraftment of Donor Cells — 29 Participants
Sickle Cell Trait
Primary: Acceptability — 11; 8; 11; 8 Participants
Sickle Cell Disease · Thalassemia Major
Primary: Rate of Graft Failure — 2; 0 participants
Sickle Cell Anemia · Severe Aplastic Anemia · Paroxysmal Nocturnal Hemoglobinuria (PNH)
Primary: Number of Patients With Neutrophil Engraftment — 2 participants
Sickle Cell Disease
Primary: % of Patient-years With Malignancies — 0.001 % patient-years
Sickle Cell Disease · Thalassemia · Hemoglobinopathies
Primary: Development of GVHD Within 1 Year of BMT — 2 participants
Sickle Cell Disease
Primary: Number Participants With Most Common Reported Drug Related Adverse Events — 6; 3; 3 Participants
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…
Beta-Thalassemia
Primary: Percentage of Participants Who Have Achieved Transfusion Independence (TI) — 88.9 Percentage of participants
Sickle Cell Disease
Primary: SCD-specific Knowledge — 32.0; 32.0; 33.5; 33 score on a scale
Beta-Thalassemia
Primary: Percentage of Participants Who Meet the Definition of Transfusion Independence (TI) — 91.3 Percentage of participants
Sickle Cell Disease
Primary: Number of Participants With Increase in Hb >1 g/dL From Baseline to Week 24 — 30; 46; 6 Participants
G6PD Deficiency
Primary: Sensitivity of SD Biosensor STANDARD G6PD Test for Identifying G6PD Deficient Individuals — 100.0; 100.0 percentage of participants
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
Premature Infant Disease · Transfusion Related Complication · Fetal Hemoglobin
Primary: Median Percentage of HbF at 32 Weeks of Post Menstrual Age — 93.2; 90.8; 58.0; 69.0 percentage of total Hb — p=<0.0001
Sickle Cell Disease
Primary: Number Of Participants With Treatment-emergent Adverse Events (TEAEs) And Serious Adverse Events (SAEs) — 12; 24; 18; 23 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…
Sickle Cell Disease
Primary: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) — 1; 3; 1; 0 Participants
Thalassemia
Primary: Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL — 2 participants
Sickle Cell Disease · Anti-D Antibodies
Primary: Anti-D Recurrence — 0; 5 Participants
Sickle Cell Disease
Primary: MRI Parameter - LVEDVi, mL/cm2 (Measured Using Method of Disks, Controls Serve as Normal Ranges) — 124.0; 78.7 mL/cm2 — p=0.0039
Thalassemia · Hypertension, Pulmonary
Primary: Change in Six-minute Walk Test (6MWT) Distance From Baseline to Week 12 Among Sildenafil Group — -1.38 meters — p=0.97
Sickle Cell Anemia · Thalassemia
Primary: Number of Occurrences of Painful Sickle Cell Crises — 4.5; 10.8 Crisis — p=0.076