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NCT04557436COMPLETEDanonymous

Phase 1, Open Label Study of CRISPR-CAR Genome Edited T Cells (PBLTT52CAR19) in Relapsed /Refractory B Cell Acute Lymphoblastic Leukaemia

Sponsor

Source record

Great Ormond Street Hospital for Children NHS Foundation Trust

Phase

Source record

Phase 1

Modality

AI-normalized

gene therapy

Target

AI-normalized

CD19+ B cells, utilizing CRISPR-CAR genome editing to modify T cells for enhanced anti-leukemic activity.

Indication / condition

AI-normalized

B Acute Lymphoblastic Leukemia

Intervention

Source record

PBLTT52CAR19

Source & freshness

Source record

NCT ID

NCT04557436

Original source

ClinicalTrials.gov

Source last updated

Mar 07, 2024

Ingested at

Jun 12, 2026

Internal sync

Jun 12, 2026

Model version

trialsignal-ai-v1

Normalized confidence

96%

Validation status

validated

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View original source fields

NCT ID

NCT04557436

Title

Phase 1, Open Label Study of CRISPR-CAR Genome Edited T Cells (PBLTT52CAR19) in Relapsed /Refractory B Cell Acute Lymphoblastic Leukaemia

Sponsor

Great Ormond Street Hospital for Children NHS Foundation Trust

Status

COMPLETED

Phase

Phase 1

Condition raw

B Acute Lymphoblastic Leukemia

Condition normalized

B Acute Lymphoblastic Leukemia

Modality raw

gene therapy

Modality normalized

gene therapy

Target raw

CD19+ B cells, utilizing CRISPR-CAR genome editing to modify T cells for enhanced anti-leukemic activity.

Target normalized

CD19+ B cells, utilizing CRISPR-CAR genome editing to modify T cells for enhanced anti-leukemic activity.

Interventions

PBLTT52CAR19

Public preview

Source record

The PBLTT52CAR19 asset, developed by Great Ormond Street Hospital for Children NHS Foundation Trust, targets relapsed/refractory B-cell acute lymphoblastic leukaemia (B-ALL) in pediatric patients. Given the high unmet need in this patient population, the potential for PBLTT52CAR19 to secure molecular remission prior to allogeneic stem cell transplantation positions it favorably in the CAR-T therapy market. The asset's innovative use of CRISPR technology for genome editing may provide a competitive edge over traditional CAR-T therapies, particularly in terms of safety and efficacy. The completion of this Phase 1 trial will be critical for advancing to larger studies and attracting potential partnerships or acquisitions from larger biopharma entities focused on oncology.

AI-generated analysis supports research triage only. Verify source records, publications, sponsor disclosures and IP databases before making diligence decisions. Model: trialsignal-ai-v1.

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