Hemoglobinopathies
59
11
19
26
Key Insights
Highlights
Success Rate
81% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
10.2%
6 terminated out of 59 trials
81.3%
-5.3% vs benchmark
15%
9 trials in Phase 3/4
38%
10 of 26 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 26 completed trials
Clinical Trials (59)
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
The HOPE Biobank Resource (BMT CTN 2402 HOPE)
A Clinical Exploratory Study of YOLT-204 in the Treatment of Hemoglobinopathies
A Long-term Follow-up Study in Participants Who Received CTX001
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases
Allo HSCT for High Risk Hemoglobinopathies
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Discarded Bone Marrow for Hematology Research
Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease
Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders
A Blood Stem Cell Transplant for Sickle Cell Disease
Prevalence of Abnormal Haemoglobin Variants in Metropolitan Chattogram, Bangladesh
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion