RecruitingPhase 1

PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas

United States, Australia, South Korea56 participantsStarted 2025-05-30

Plain-language summary

The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1). The main questions it aims to answer are: * How well participants are able tolerate different doses of PAS-004, and * What side effects PAS-004 might have. This study will have two parts, Part A and Part B. The main goal of Part A of this study is to learn more about how participants tolerate different doses of PAS-004, and what side effects PAS-004 might have. What we learn from Part A of the study will help decide what doses of the study drug (PAS-004) should be used in Part B of the study, and if it is safe. In Part B, two different doses from Part A will be tested. The main goal of this part of the study is to keep studying any side effects of PAS-004 at those two dose levels, and to learn more about if the doses picked for this part of the study might have an effect on plexiform neurofibromas. Participants in Part A of the study who were taking doses selected for Part B may be able to continue on to Part B and keep taking the same dose of PAS-004 for 6 more months. Study participants in both parts will have regular visits to the study doctor and be asked to have tests and exams done to check on their health and safety, including blood draws and MRIs. Everyone participating in the study will take PAS-004 by mouth once a day during the study, in 28-day cycles. Participants will be asked to keep a diary to record their daily dose of study drug. Participants will continue on daily PAS-004 for up to 6 months, or until: * They decide to withdraw from the study, or * They experience unacceptable side effects, or * Their disease progresses, or another illness interferes with taking the study drug, or * The sponsor selects a dose level to study further in the next part of the study, or * The sponsor stops the study.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Participant is capable of providing informed consent, which includes compliance with the requirements, prohibitions and restrictions listed in the informed consent form.
. Participant has been informed both verbally and in writing about the objectives of the clinical study, the methods, the anticipated benefits, the potential risks, and the discomfort to which they may be exposed and has given written consent to participation in the study prior to study start and any study-related procedure.
. Participant must be at least 18 years of age at the signing of the informed consent form (ICF).
. Participant must be able to swallow oral medication.
. Performance status: Participant must have a Karnofsky performance level of ≥70%. Note: Participants who are wheelchair bound because of paralysis secondary to a PN should be considered ambulatory when they are in the wheelchair. Similarly, participants with limited mobility secondary to the need for mechanical support (such as an airway PN requiring tracheostomy or CPAP) will also be considered ambulatory for the purposes of this study.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Part A: To evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle

Timeframe: Part A from enrollment (Day 1) through Day 28 (completion of Cycle 1)

Part B: To evaluate the safety and tolerability of PAS-004 when administered for six 28-day treatment cycle

Timeframe: Part B from enrollment (Day 1) through Day 168 (completion of Cycle 6) [each cycle is 28 days]

Trial details

NCT IDNCT06961565

SponsorPasithea Therapeutics Corp.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-09

View on ClinicalTrials.gov More NF1 Mutation trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Participant is capable of providing informed consent, which includes compliance with the requirements, prohibitions and restrictions listed in the informed consent form.
. Participant has been informed both verbally and in writing about the objectives of the clinical study, the methods, the anticipated benefits, the potential risks, and the discomfort to which they may be exposed and has given written consent to participation in the study prior to study start and any study-related procedure.
. Participant must be at least 18 years of age at the signing of the informed consent form (ICF).
. Participant must be able to swallow oral medication.
. Performance status: Participant must have a Karnofsky performance level of ≥70%. Note: Participants who are wheelchair bound because of paralysis secondary to a PN should be considered ambulatory when they are in the wheelchair. Similarly, participants with limited mobility secondary to the need for mechanical support (such as an airway PN requiring tracheostomy or CPAP) will also be considered ambulatory for the purposes of this study.

What they're measuring

Part A: To evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle

Timeframe: Part A from enrollment (Day 1) through Day 28 (completion of Cycle 1)

Part B: To evaluate the safety and tolerability of PAS-004 when administered for six 28-day treatment cycle

Timeframe: Part B from enrollment (Day 1) through Day 168 (completion of Cycle 6) [each cycle is 28 days]

PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas

Plain-language summary

Who can participate

Inclusion criteria

Questions worth asking your doctor

Questions for the trial coordinator

What they're measuring

Trial details

Exclusion criteria