Actively Recruiting
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
Led by Pasithea Therapeutics Corp. · Updated on 2025-12-04
56
Participants Needed
5
Research Sites
130 weeks
Total Duration
On this page
Sponsors
P
Pasithea Therapeutics Corp.
Lead Sponsor
N
Novotech (Australia) Pty Limited
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participants must be able to give informed consent.
- Participants must be at least 18 years old at consent.
- Participants must be able to swallow oral medication.
- Participants must have a Karnofsky performance status of 70% or higher.
- Participants must have a confirmed diagnosis of NF1 based on clinical and imaging criteria or genetic testing.
- Participants must have at least one symptomatic plexiform neurofibroma measuring at least 3 cm, which is unsuitable for complete surgical removal or has recurred.
- Participants should have at least seven measurable cutaneous neurofibromas between 6 and 15 mm in size.
- Participants must be willing and able to undergo serial MRI scans as required.
- Participants must be willing and able to undergo serial 2-D and 3-D photography and caliper measurements.
- Participants must have adequate blood clotting function (INR and APTT ≤ 1.5 times upper limit of normal).
- Participants must have adequate organ and bone marrow function within specified lab value ranges.
- Participants must agree to maintain abstinence or use highly effective contraception during the study and for 90 days after.
- Sperm-producing participants must agree not to donate sperm during the study and for 90 days after.
You will not qualify if you...
- Participants who have been in another interventional study within 28 days before starting PAS-004.
- Participants who received chemotherapy within 90 days before starting PAS-004.
- Participants with ongoing side effects from prior chemotherapy worse than mild (except hair loss).
- Participants who received PN-directed drug or biologic therapy within 14 days before starting PAS-004.
- Participants who received strong CYP3A4 inhibitors or inducers within 14 days before starting PAS-004.
- Participants who received growth factors to increase blood cells within 7 days before starting PAS-004.
- Participants who received radiotherapy, major surgery, or immunotherapy within 28 days before starting PAS-004.
- Participants with malignant tumors requiring active treatment or history of malignancy requiring treatment within 5 years, excluding certain skin and in situ cancers.
- Participants with uncontrolled high blood pressure despite treatment.
- Participants with swallowing or digestive conditions affecting drug absorption.
- Participants with certain eye diseases or abnormalities.
- Participants with interstitial pneumonia or NF1-related lung disease.
- Participants with significant heart problems or abnormal heart function.
- Participants who have taken QTc-prolonging medication shortly before starting the study drug.
- Participants with uncontrolled infections including active hepatitis B, hepatitis C, or HIV with detectable viral load.
- Participants with significant liver disease except Gilbert's syndrome or asymptomatic gallstones.
- Participants allergic to PAS-004 or related drugs.
- Participants who are pregnant or lactating.
- Participants with other medical conditions that would interfere with study participation.
- Participants unable to attend required in-person clinic visits.
AI-Screening
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Trial Site Locations
Total: 5 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
Royal North Shore Hospital
St Leonards, New South Wales, Australia, 2065
Actively Recruiting
3
Peter MacCallum Cancer Centre
Melbourne, Victoria, Australia, 3000
Actively Recruiting
4
Asan Medical Center
Seoul, South Korea, 05505
Actively Recruiting
5
Severance Hospital, Yonsei University Health System
Sinchŏn-dong, South Korea, 6164
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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