Actively Recruiting
PAS-004 Oral Tablet Study to Assess Safety and Effects in Adults With Neurofibromatosis Type 1 and Plexiform Neurofibromas
Led by Pasithea Therapeutics Corp. · Updated on 2025-12-04
56
Participants Needed
5
Research Sites
13 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
This research aims to evaluate PAS-004 in adults with Neurofibromatosis Type 1 (NF1) who have symptomatic plexiform neurofibromas that are inoperable, incompletely resected, or recurrent. The study focuses on how well participants tolerate different doses of PAS-004 and to identify any side effects. It is a Phase 1/1b trial designed to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of this MEK 1/2 inhibitor in this population. The study is divided into two parts. Part A involves sequential dose escalation of PAS-004 tablets given orally once daily in 28-day cycles, with doses of 4 mg, 8 mg, 12 mg, and 18 mg tested to determine tolerability and safety. In Part B, two dose levels selected based on Part A results will be tested in parallel cohorts. Participants may continue on their assigned dose in Part B for up to six months if eligible. Throughout the study, participants will take PAS-004 daily and keep a diary to record their dosing. Participants will have regular visits for health and safety monitoring including blood tests and MRI scans to measure tumor response. Additional assessments include photography and caliper measurements of cutaneous neurofibromas, quality of life surveys, and physical function assessments. The primary outcome is safety and tolerability over one 28-day cycle in Part A and six 28-day cycles in Part B. Secondary outcomes include drug concentration levels, tumor response by MRI, appearance of neurofibromas, and patient-reported outcomes. The study continues until six months or earlier if side effects, disease progression, or other criteria occur.
CONDITIONS
Brief Title
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant can provide informed consent and comply with study requirements
- Participant has been informed about the study and given written consent
- Participant is at least 18 years old at consent signing
- Participant can swallow oral medication
- Karnofsky performance status of 70% or higher, including those in wheelchairs due to PN-related paralysis
- Diagnosed with NF1 by clinical and imaging criteria or genetic testing
- Has at least one symptomatic plexiform neurofibroma at least 3 cm in size causing symptoms or unsuitable for surgery
- Has at least seven measurable cutaneous neurofibromas between 6 and 15 mm on trunk, neck, or limbs (optional resections allowed)
- Willing and able to undergo serial MRI scans as per protocol
- Willing and able to undergo serial photography and caliper measurements
- INR and APTT laboratory values at or below 1.5 times the upper limit of normal
- Adequate organ and bone marrow function based on specified lab values
- Agrees to maintain abstinence or use effective contraception during treatment and for 90 days after last dose
- Sperm-producing participants agree to avoid sperm donation during treatment and for 90 days after last dose
You will not qualify if you...
- Participation in another interventional clinical study within 28 days before starting PAS-004
- Chemotherapy within 90 days before starting PAS-004
- Ongoing side effects from prior chemotherapy worse than mild (except hair loss)
- Treatment with any PN-directed drug or biologic therapy within 14 days before starting PAS-004
- Use of strong CYP3A4 inhibitors or inducers within 14 days before PAS-004
- Growth factors to increase platelets or white blood cells within 7 days before PAS-004
- Radiotherapy, major surgery, or immunotherapy within 28 days before starting PAS-004
- Malignant tumors associated with NF1 requiring active treatment
- Current malignancy or history of malignancy requiring treatment within past 5 years (with some exceptions)
- Uncontrolled hypertension over 150/90 mmHg despite treatment
- Active digestive diseases affecting drug absorption
- Eye conditions including retinal vein occlusion or glaucoma
- Interstitial pneumonia or NF1-related lung disease
- Impaired heart function or significant cardiac disease
- Use of QTc-prolonging medication within 7 days before starting study drug
- Active infections including hepatitis B or C, or HIV with detectable viral load
- Significant liver disease or abnormalities (except Gilbert's syndrome)
- Known hypersensitivity to PAS-004 or MEK 1/2 inhibitors
- Pregnant or lactating
- Any condition that would prevent safe participation or compliance
- Unable to attend in-person clinic visits as required
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - One 28-day treatment cycle for Part A; up to six 28-day treatment cycles for Part B
Participants receive PAS-004 oral tablets once daily in sequential dose escalation or selected dose cohorts to assess safety and effects.
Up to 6 monthly visits corresponding to treatment cycles
Duration - Up to 168 days (6 months) including treatment and observation
Participants are monitored for response and side effects including MRI scans, photography, and quality of life assessments.
Visits at Days 28, 56, 84, 112, 140, and 168 for assessments
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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