Hemoglobinopathies
57
10
19
25
Key Insights
Highlights
Success Rate
81% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
10.5%
6 terminated out of 57 trials
80.6%
-5.9% vs benchmark
16%
9 trials in Phase 3/4
32%
8 of 25 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 25 completed trials
Clinical Trials (57)
Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT
A Long-term Follow-up Study in Participants Who Received CTX001
Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases
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
Allo HSCT for High Risk Hemoglobinopathies
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
A Blood Stem Cell Transplant for Sickle Cell Disease
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
The HOPE Biobank Resource (BMT CTN 2402 HOPE)
Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders
Prevalence of Abnormal Haemoglobin Variants in Metropolitan Chattogram, Bangladesh
A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia
Discarded Bone Marrow for Hematology Research
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
Prevalence of Hemoglobinopathies Associated With Significant Hb Variants in the Chattogram Region of Bangladesh
Prevalence and Hematological Characteristics of Hemoglobinopathies
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease
Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)