Key Insights

Highlights

Success Rate

81% trial completion

Clinical Risk Assessment

Based on trial outcomes

High Risk

Score: 62/100

Termination Rate

10.5%

6 terminated out of 57 trials

Success Rate

80.6%

-5.9% vs benchmark

Late-Stage Pipeline

16%

9 trials in Phase 3/4

Results Transparency

32%

8 of 25 completed with results

Key Signals

8 with results81% success

Data Visualizations

Phase Distribution

40Total
Not Applicable (7)
Early P 1 (1)
P 1 (12)
P 2 (11)
P 3 (7)
P 4 (2)

Trial Status

Completed25
Recruiting10
Active Not Recruiting9
Terminated6
Unknown3
Enrolling By Invitation2

Trial Success Rate

80.6%

Benchmark: 86.5%

Based on 25 completed trials

Clinical Trials (57)

Showing 20 of 20 trials
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

NCT06872333Phase 2Recruiting

Allo HSCT for High Risk Hemoglobinopathies

NCT05329649Phase 3Active Not Recruiting

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

NCT03249831Phase 1Active Not Recruiting

A Blood Stem Cell Transplant for Sickle Cell Disease

NCT05356195Phase 3Active Not Recruiting

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

NCT07227155Not Yet Recruiting

The HOPE Biobank Resource (BMT CTN 2402 HOPE)

NCT03128996Phase 1Recruiting

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

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

NCT04671212RecruitingPrimary

Discarded Bone Marrow for Hematology Research

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

NCT01966367Phase 1Active Not Recruiting

CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation

NCT03745287Phase 2Completed

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease

NCT01666080Not ApplicableRecruiting

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

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Research Network

Activity Timeline