30 closest matches · ranked by relevance
Thalassemia Major
Primary: Change of Hematocrit Adjusted Volume of Red Blood Cells (RBC) — -5.934 % change of hematocrit-adjusted volume
Severe Thalassemia
Primary: Primary Objective: Event-free Survival at 1 Year. — 17 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
Thalassemia
Primary: Report of Pain by Age Group — 23; 61; 18; 51 percentage of participant-cycles — p=<.001
Beta-Thalassemia
Primary: Percentage of Participants Who Have Achieved Transfusion Independence (TI) — 88.9 Percentage of 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
Beta-Thalassemia
Primary: Percentage of Participants Who Meet the Definition of Transfusion Independence (TI) — 91.3 Percentage of 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
Thalassemia
Primary: Prevalence of Pain — 75; 7; 11 participants — p=0.45
Beta-Thalassemia Major · Sickle Cell Disease
Primary: Number of Treated Participants With Successful Neutrophil and Platelet Engraftment — 3; 4; 3; 4 Participants
Thalassemia
Primary: Number of Evaluable Patients With an Increase From Baseline in Hemoglobin (Hb) of ≥1.5 g/dL — 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
Thalassemia · Iron Overload
Primary: Efficacy of Combined Treatment With Deferasirox and Deferoxamine Over 12 Months — -6.2 mg/g
Thalassemia Major With Severe Transfusional Iron Overload
Primary: Number of Participants With Improvement in Liver Iron Concentration — 3 Participants
Sickle Cell Disease · Beta Thalassemia-Major
Primary: Number of Participants With Sustained Cell Engraftment of Donor Cells — 29 Participants
Sickle Cell Disease · Thalassemia · Diamond-Blackfan Anemia
Primary: Count of Participants With Stable Engraftment Post Hematopoietic Cell Transplantation (HCT) — 3 Participants
Non-transfusion Dependent Thalassemia
Primary: Core Study: Change in Liver Iron Concentration (LIC) From Baseline to Week 52 — -1.95; -3.80; 0.38 mg iron (Fe)/g dry weight (dw)
Anemia · Hemosiderosis
Primary: The Number of Participants With Adverse Events (AEs), Serious Adverse Events (SAEs) or Deaths — 85; 99; 36; 50 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…
Transfusional Iron Overload · β-thalassemia Major · Pediatric Rare Anemia
Primary: Participants With Adverse Events by Primary System Organ Class (SOC) — 20; 20; 19; 20 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)
Dyskeratosis Congenita · Aplastic Anemia
Primary: Incidence of Neutrophil Engraftment — 100; 100; 96; 100 Percentage of participants
Non-transfusion Dependent Thalassemia
Primary: Absolute Change in Liver Iron Content (LIC) at 52 Weeks From Baseline — -6.68 mg Fe/g dw
Thrombotic Microangiopathies
Primary: Percent of Total Doses of Defibrotide That Were Missed [Feasibility] — 0.7 percentage of missed doses
Sickle Cell Disease · Thalassemia Major
Primary: Rate of Graft Failure — 2; 0 participants
Beta-Thalassemia · Non-transfusion-dependent Thalassemia
Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 6; 7; 3 Participants
Thalassemia · Sickle Cell Disease · Diamond Blackfan Anemia
Primary: Safety Profile of Deferasirox Based Upon Drug Administration and Reporting of Serious Adverse Events — 0; 0; 1; 4 Participants
Thalassemia, Non-transfusional-dependent Thalassemia (NTDT), Myeloplastic Dysplasia (MDS), Other Anemia
Primary: Percentage of Participants With Cardiac and Liver Iron Overload. — 10; 22; 4; 0 Percentage of participants
Infant, Newborn, Diseases · Infant, Extremely Low Birth Weight · Infant, Small for Gestational Age
Primary: Death or Neurodevelopmental Impairment — 423; 422; 422; 425 Participants — p=0.93
Chronic Iron Overload
Primary: Percentage of Successfully Chelated Patients — 69; 80 Participants