Experimental drug targets rare blood cancer in tiny trial

NCT ID NCT03485547

Completed Disease control Sponsor: Dana-Farber Cancer Institute Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase study tested the drug venetoclax in 5 adults with a rare blood cancer called BPDCN that had come back or not responded to prior treatment. The goal was to check safety and see if the drug could shrink tumors. Venetoclax works by helping cancer cells self-destruct.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Venetoclax (Venclexta), a drug that helps cancer cells self-destruct
What this could lead to: If successful, this could point toward a new treatment option for BPDCN, a rare blood cancer.
What could go wrong: This is a very early Phase 1 trial with only 5 participants, so results may not apply broadly. The drug may cause side effects or not work for all patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Blastic Plasmacytoid Dendritic Cell Neoplasm CD4+/CD56+ hematodermic neoplasm

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

BLASTIC PLASMACYTOID DENDRITIC CELL NEOPLASM

Contacts and locations

Locations

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States
MD Anderson Cancer Center

Houston, Texas, 77030, United States

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