New pill hopes to shrink painful nerve tumors in NF1 patients
NCT ID NCT06961565
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.
Disclaimer
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This is a summary of
the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Asan Medical Center
RECRUITINGSeoul, 05505, South Korea
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Peter MacCallum Cancer Centre
RECRUITINGMelbourne, Victoria, 3000, Australia
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Royal North Shore Hospital
RECRUITINGSt Leonards, New South Wales, 2065, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Severance Hospital, Yonsei University Health System
RECRUITINGSinchŏn-dong, 6164, South Korea
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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University of Alabama at Birmingham
RECRUITINGBirmingham, Alabama, 35294, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact