Key Insights

Highlights

Success Rate

81% trial completion

Clinical Risk Assessment

Based on trial outcomes

High Risk

Score: 62/100

Termination Rate

10.3%

6 terminated out of 58 trials

Success Rate

81.3%

-5.3% vs benchmark

Late-Stage Pipeline

16%

9 trials in Phase 3/4

Results Transparency

35%

9 of 26 completed with results

Key Signals

9 with results81% success

Data Visualizations

Phase Distribution

41Total
Not Applicable (7)
Early P 1 (1)
P 1 (12)
P 2 (12)
P 3 (7)
P 4 (2)

Trial Status

Completed26
Recruiting11
Active Not Recruiting8
Terminated6
Unknown3
Enrolling By Invitation2

Trial Success Rate

81.3%

Benchmark: 86.5%

Based on 26 completed trials

Clinical Trials (58)

Showing 20 of 20 trials
NCT01050855Phase 2Completed

Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders

NCT01666080Not ApplicableRecruiting

Second or Greater Allogeneic Hematopoietic Stem Cell Transplant Using Reduced Intensity Conditioning (RIC)

NCT05356195Phase 3Active Not Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT05329649Phase 3Active Not Recruiting

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

NCT07660783Phase 2Not Yet Recruiting

Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases

NCT07227155Recruiting

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

NCT06872333Phase 2Recruiting

Allo HSCT for High Risk Hemoglobinopathies

NCT03128996Phase 1Recruiting

Reduced Intensity Conditioning and Familial HLA-Mismatched BMT for Non-Malignant Disorders

NCT04671212RecruitingPrimary

Discarded Bone Marrow for Hematology Research

NCT06839456Phase 1Recruiting

Phase 1/2: CD45RA Depleted Stem Cell Addback to Prevent Viral or Fungal Infections Post TCRab/CD19 Depleted HSCT

NCT04208529Phase 3Enrolling By Invitation

A Long-term Follow-up Study in Participants Who Received CTX001

NCT00920972Phase 1Recruiting

Campath/Fludarabine/Melphalan Transplant Conditioning for Non-Malignant Diseases

NCT05477563Phase 3Recruiting

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT04644016Phase 2Recruiting

Cord Blood Transplant in Children and Young Adults With Blood Cancers and Non-malignant Disorders

NCT03249831Phase 1Active Not Recruiting

A Blood Stem Cell Transplant for Sickle Cell Disease

NCT07305181Completed

Prevalence of Abnormal Haemoglobin Variants in Metropolitan Chattogram, Bangladesh

NCT03655678Phase 2Completed

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

NCT05236764Not ApplicableActive Not Recruiting

Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion

NCT07207551CompletedPrimary

Prevalence of Hemoglobinopathies Associated With Significant Hb Variants in the Chattogram Region of Bangladesh

NCT07180836CompletedPrimary

Prevalence and Hematological Characteristics of Hemoglobinopathies

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Research Network

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