PAS-004 for Plexiform Neurofibromas in NF1
This study is testing a new oral medication called PAS-004 in adults with Neurofibromatosis Type 1 (NF1) who have plexiform neurofibromas (tumors that grow along nerves). PAS-004 is a MEK 1/2 inhibitor, which means it blocks certain proteins involved in cell growth. The main goals are to understand how safe PAS-004 is at different doses and what side effects it might cause. The study has two parts: Part A will help determine safe doses, and Part B will test those doses further. We are looking for 56 participants for this study, but the current recruitment status is unclear.
- Study design
- This is an interventional study with a planned enrollment of 56 participants. It is structured in two parts to evaluate safety and tolerability.
- What's involved
- Participants will take PAS-004 tablets once daily. Part A involves one 28-day treatment cycle, while Part B involves six 28-day treatment cycles.
- Compensation
- Not stated in the trial record.
- Follow-up
- Safety and tolerability will be measured through Day 28 for Part A and through Day 168 for Part B.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
At a glance
Conditions
Where it's being run
5 sites across 4 statesStudy leadership
- Tiago R Marques, MD · STUDY_DIRECTOR · Pasithea Therapeutics Corp.
Who to contact
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What this trial measures
- Part A: To evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cyclePart A from enrollment (Day 1) through Day 28 (completion of Cycle 1)
Endpoint/Outcome Measures: Number of participants with dose-limiting toxicities (DLTs), with Adverse Events (AEs), AEs leading to interruption or discontinuation of study drug, with clinically significant findings on clinical laboratory tests, with abnormal cardiac and visual function exams using the Common Terminology Criteria for Adverse Events (CTCAE Version 5).
- Part B: To evaluate the safety and tolerability of PAS-004 when administered for six 28-day treatment cyclePart B from enrollment (Day 1) through Day 168 (completion of Cycle 6) [each cycle is 28 days]
Endpoint/Outcome Measures: Number of participants with dose-limiting toxicities (DLTs), with Adverse Events (AEs), AEs leading to interruption or discontinuation of study drug, with clinically significant findings on clinical laboratory tests, with abnormal cardiac and visual function exams using the Common Terminology Criteria for Adverse Events (CTCAE Version 5).