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

Part of paid clinical trials in Birmingham, Alabama.

Sponsor
Pasithea Therapeutics Corp.
Study ID
NCT06961565
Phase
PHASE1
Status
Recruiting
Apply to this trial

Conditions

  • NF1 Mutation
  • Neurofibroma Plexiform
  • Neurofibroma, Plexiform
  • Neurofibromatosis Type 1 (NF1)
  • Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)

Eligibility Criteria

Sex
ALL
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • PAS-004 Tablets — DRUG
    A mitogen-activated protein kinase/extracellular signal-regulated kinase kinase (MAPK/ERK kinase, or MEK) 1/2 inhibitor presented in 1m and 4mg strength tablets, intended for oral administration once daily.

Study Details

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.

Key Dates

Start date
May 30, 2025
Status verified
Nov 2025
Primary completion
Sep 30, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
56 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Part A
    Sequential dose escalation: 4 mg, 8 mg, 12 mg, and 18 mg
  • Experimental: Part B
    Two parallel cohorts dosing at 2 levels selected based on Part A safety results

Primary Outcome Measure

Part A: To evaluate the safety and tolerability of PAS-004 when administered for one 28-day treatment cycle [ Time Frame: Part A from enrollment (Day 1) through Day 28 (completion of Cycle 1) ]

Locations (1)

FacilityCityStateZIPSite coordinators
University of Alabama at BirminghamBirminghamAlabama35294
Rebecca Brown, MD PhD
[email protected]
205-934-1813
Rebecca Brown, MD PhD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Birmingham, AL

By research site
University of Alabama at Birmingham· Birmingham, AL

Related Studies