Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) - Healing Genes

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

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Study to Evaluate the Safety and Clinical Activity of UCART123 in Patients With BPDCN (ABC123)

Phase 1, Open-label Dose-escalation and Dose-expansion Study to Evaluate the Safety, Expansion, Persistence and Clinical Activity of a Single Dose of UCART123 (Allogeneic Engineered T-cells Expressing Anti-CD123 Chimeric Antigen Receptor), Administered in Patients With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Phase 1


Doctors at the MD Anderson Cancer Center seek patients with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) to participate in a clinical trial of a gene-modified immunotherapy. The investigatory treatment withdraws immune T-cells from the patient and, utilizing gene editing, the white blood cells will be trained to target receptors titled CD123 on their cancer cells. Then the modified T-cells will be infused to the patient after a course of chemotherapy. Follow up lasts at least 2 years.


  • Be 18 to 80 years of age
  • Be diagnosed with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
  • with DNA tests to confirm CD123 antigen presence on tumor cells
  • Not be pregnant and be willing to use contraception


  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. Have blood drawn, immune cells separated and blood cells returned (leukapheresis)
  3. After lab modification time, the patient will be admitted to the hospital and receive chemotherapy over 3 to 5 days.
  4. The gene-edited immune cells will be infused.
  5. Recovery in the hospital.
  6. Follow up continues for at least 2 years.

The study site is at the MD Anderson Cancer Center in Houston, TX. Map.

Naveen Pemmaraju, MD  |  713-792-4956  |  [email protected]
Aaron Logue, MBA  |  513-579-9911 ext 12889  |  [email protected]
Cellectis S.A.
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