Key Insights

Highlights

Success Rate

79% trial completion

Published Results

18 trials with published results (26%)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 37/100

Termination Rate

8.6%

6 terminated out of 70 trials

Success Rate

79.3%

-7.2% vs benchmark

Late-Stage Pipeline

9%

6 trials in Phase 3/4

Results Transparency

78%

18 of 23 completed with results

Key Signals

18 with results79% success

Data Visualizations

Phase Distribution

64Total
Not Applicable (9)
Early P 1 (1)
P 1 (17)
P 2 (31)
P 3 (3)
P 4 (3)

Trial Status

Completed23
Recruiting16
Unknown12
Active Not Recruiting7
Terminated6
Not Yet Recruiting3

Trial Success Rate

79.3%

Benchmark: 86.5%

Based on 23 completed trials

Clinical Trials (70)

Showing 20 of 20 trials
NCT05012111RecruitingPrimary

Natural History of Acquired and Inherited Bone Marrow Failure Syndromes

NCT05998408Phase 1RecruitingPrimary

JAK1/2 Inhibitor Ruxolitinib for Relapsed/Refractory Immune Bone Marrow Failure

NCT02099747Phase 3CompletedPrimary

hATG+CsA vs hATG+CsA+Eltrombopag for SAA

NCT02828592Phase 2RecruitingPrimary

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

NCT03192397Phase 1Active Not Recruiting

Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant

NCT01623167Phase 1Active Not RecruitingPrimary

Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

NCT01351545Recruiting

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

NCT02918292Phase 2CompletedPrimary

Optimizing Haploidentical Aplastic Anemia Transplantation (BMT CTN 1502)

NCT03173937Phase 1RecruitingPrimary

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS Using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome

NCT04304820Phase 2RecruitingPrimary

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

NCT06517641Phase 2RecruitingPrimary

Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia

NCT05600426Phase 3Active Not RecruitingPrimary

A Trial Comparing Unrelated Donor BMT With IST for Pediatric and Young Adult Patients With Severe Aplastic Anemia (TransIT, BMT CTN 2202)

NCT07299123Phase 1Enrolling By Invitation

Phase I Clinical Study of Haplo-HSCT Combined With Hypoxic 3D-Cultured Umbilical Cord MSC for the Treatment of SAA

NCT03579875Phase 2Recruiting

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

NCT02979873Phase 2Active Not RecruitingPrimary

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

NCT01174108Phase 2RecruitingPrimary

Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells

NCT07297550Phase 1Not Yet RecruitingPrimary

Safety and Efficacy of the JAK1 Inhibitor Combined With Intensive Immunosuppressive Therapy in Severe Aplastic Anemia

NCT05236764Not ApplicableActive Not Recruiting

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

NCT01966367Phase 1Active Not Recruiting

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

NCT04099966Phase 2Recruiting

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

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