New pill hopes to shrink painful nerve tumors in NF1 patients

NCT ID NCT06961565

Recruiting now Disease control Sponsor: Pasithea Therapeutics Corp. Source: ClinicalTrials.gov ↗

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage trial tests a daily pill called PAS-004 in 56 adults with NF1 who have at least one symptomatic, inoperable plexiform neurofibroma. The study has two parts: Part A finds a safe dose, and Part B checks safety and whether the drug can shrink tumors. Participants take the pill once daily for up to 6 months and undergo regular blood tests and MRIs.

What this could mean

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

Active substance: PAS-004 (a MEK inhibitor taken as a daily pill)
What this could lead to: If it works, this could point toward a new treatment option to shrink or control plexiform neurofibromas in people with NF1.
What could go wrong: This is an early Phase 1 trial with only 56 participants, so the main goal is safety, not proof of effectiveness. The drug may cause side effects or fail to shrink tumors.

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Conditions

The condition(s) this trial relates to.

neurofibromatosis neurofibromatosis type 1 plexiform neurofibroma

As listed by the trial registrant

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

NEUROFIBROMA, PLEXIFORM NEUROFIBROMATOSIS TYPE 1 (NF1) NEUROFIBROMATOSIS TYPE 1 (NF1)-RELATED PLEXIFORM NEUROFIBROMAS (PNS) NF1 MUTATION

Contacts and locations

Locations

Asan Medical Center
RECRUITING

Seoul, 05505, South Korea
Peter MacCallum Cancer Centre
RECRUITING

Melbourne, Victoria, 3000, Australia
Royal North Shore Hospital
RECRUITING

St Leonards, New South Wales, 2065, Australia
Severance Hospital, Yonsei University Health System
RECRUITING

Sinchŏn-dong, 6164, South Korea
University of Alabama at Birmingham
RECRUITING

Birmingham, Alabama, 35294, United States

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