30 closest matches · ranked by relevance
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…
Beta-Thalassemia
Primary: Percentage of Participants Who Have Achieved Transfusion Independence (TI) — 88.9 Percentage of participants
Thalassemia
Primary: Prevalence of Pain — 75; 7; 11 participants — p=0.45
Beta-Thalassemia
Primary: Percentage of Participants Who Meet the Definition of Transfusion Independence (TI) — 91.3 Percentage of participants
Thalassemia
Primary: Report of Pain by Age Group — 23; 61; 18; 51 percentage of participant-cycles — p=<.001
Thalassemia
Primary: Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL — 2 participants
Sickle Cell Disease · Beta Thalassemia-Major
Primary: Number of Participants With Sustained Cell Engraftment of Donor Cells — 29 Participants
Beta-Thalassemia · Non-transfusion-dependent Thalassemia
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 6; 7; 3 Participants
Transfusion Dependent Beta-thalassemia
Primary: Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 156 Weeks After the ST-400 Infusion — 5; 2 participants
Beta-thalassemia · Iron Overload
Primary: Magnetic Resonance Imaging (MRI) T2* and Absolute Change From Baseline in MRI T2* — 9.92; 11.73; 1.77; 11.93 milliseconds (msec)
Beta-Thalassemia Major · Sickle Cell Disease
Primary: Number of Treated Participants With Successful Neutrophil and Platelet Engraftment — 3; 4; 3; 4 Participants
Severe Thalassemia
Primary: Primary Objective: Event-free Survival at 1 Year. — 17 Participants
Thalassemia
Primary: Percentage of Participants Achieving Erythroid Response (Week 13 to Week 24) — 77.1; 0.0 Percent of Participants
Beta Thalassemia Major · Congenital Anemias
Primary: Number of Participants With no Serious Adverse Events — 5 participants
Erythrocyte Transfusion · Beta-Thalassemia
Primary: Percentage of Participants Who Achieved Erythroid Response - Week 13 to Week 24 — 21.0; 4.5 Percentage of participants — p=<0.0001
Sickle Cell Disease · Thalassemia Major
Primary: Rate of Graft Failure — 2; 0 participants
Beta Thalassemia Major Anemia · Iron Overload
Primary: The Percentage of Patients in Each Treatment Group Who Still Have a Serum Ferritin Level < 1000 Micrograms Per Liter (μg/L) at Month 12 — 21; 12 Participants — p=0.0446
B-Thalassemia
Primary: Percentage of Non-transfusion Dependent (NTD) Participants With a Hemoglobin Increase of ≥1.5 g/dL From Baseline for ≥14 Days — 0.0; 0.0; 0.0; 66.7 Percentage of…
Thalassemia Major
Primary: Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC) — -5.934 % change of hematocrit-adjusted volume
Thalassemia
Primary: Change in Lumbar Spine Bone Mineral Density (BMD) by DXA (Baseline to 18 Months) — 5.9; 1.5 Percent Change — p=0.13
Thalassemia Major With Severe Transfusional Iron Overload
Primary: Number of Participants With Improvement in Liver Iron Concentration — 3 Participants
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 Disease · Thalassemia · Severe Congenital Neutropenia
Primary: Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity — 0; 0; 2 Participants
Transfusional Iron Overload in β-thalassemia
Primary: Long Term Safety and Tolerability Profile of ICL670 Based on the Number of Participants Who Experienced Any Adverse Event — 109; 113; 85; 110 Participants
Anemia · Hemosiderosis
Primary: The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths — 85; 99; 36; 50 Participants
Sickle Cell Disease
Primary: Percentage of Participants With Two-Year Post-Transplant Event Free Survival (EFS) — 88.0; 79.3 Percentage of Participants
Thalassemia · Iron Overload
Primary: Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months — -6.2 mg/g
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Primary: Number of Participants With Adverse Events, Serious Adverse Events and Deaths as a Measure of Safety and Tolerability — 25; 3; 0 Participants
Sickle Cell Disease & Thalassemia
Primary: Safety and Tolerability Will be Measured by Acute NiCord® Infusional Toxicity. — 0; 0; 3; 0 participants