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NCT05444894 (EDIT-301 in Transfusion-Dependent Beta Thalassemia): BVCT ex-ante prediction vs. reported outcome

On 14 Feb 2024, BioinvestGPT's BVCT predicted technical success and commercial partial success for this trial. The 12 Dec 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistical significance undisclosed and commercially insufficient.

Clinical Trial

NCTID
NCT05444894
Acronym
EdiTHAL
Drug Name
EDIT-301
Drug MoA
AsCas12a-mediated HBG1-HBG2-promoter-deletion CD34+ autologous hematopoietic stem and progenitor cell therapy
Drug Modality
cell therapy
Drug Class
First-In-Class
Therapeutic Area
Hemic and Lymphatic Diseases
Indication
Transfusion-Dependent Beta Thalassemia
Human Patients
9
Sponsor
Editas Medicine
Phase
Phase 1 Phase 2

Prediction of , index 1243

Prospective Prediction
Prediction Index
1243
Prediction Date
Commercial Prediction
partial SUCCESS (commercially insufficient clinical benefit non-superior to Casgevy)
Technical Prediction
SUCCESS (statistically significant clinical benefit)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
302 days in advance
Readout Data Interpretation
EDIT301's further development discontinued without a commercial partner
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistical significance undisclosed and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

Official title
A Multicenter Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of Autologous Clustered Regularly Interspaced Short Palindromic Repeats Gene-edited Cluster of Differentiation 34 (CD34+) Human Hematopoietic Stem and Progenitor Cells (HSPC) (EDIT-301) in Transfusion-Dependent Beta Thalassemia (TDT)
Phase
Phase 1, Phase 2
Status
Active not recruiting
Enrollment
9
Lead sponsor
Editas Medicine, Inc.
Conditions
Transfusion Dependent Beta Thalassemia; Hemoglobinopathies; Thalassemia Major; Thalassemia Intermedia
Interventions
GENETIC: EDIT-301
Design
Single group · None
Primary purpose
Treatment
Start
2022-04-29
Primary completion
2025-09
Completion
2025-12
Primary outcomes
  • Proportion of participants achieving engraftment defined as neutrophil engraftment (defined as demonstrating absolute neutrophil count (ANC) ≥ 0.5 x 10^9/L post EDIT-301 infusion for 3 consecutive measurements obtained on different days) (EDIT-301 infusion (Day 0) to 42 days post EDIT-301 infusion)
  • Frequency and severity of adverse events (AEs) (incidence of AEs and Grade 3 or higher serious adverse events, using National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] v.5.0) (Screening through up to 24 months post EDIT-301 infusion)

NCT05444894 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials