OncoMatch/Clinical Trials/NCT06961565
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
Is NCT06961565 recruiting? Yes, currently enrolling (May 2026). This Phase 1 trial studies PAS-004 Tablets for nf1 mutation.
Treatment: PAS-004 Tablets — 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.
Check if I qualifyExtracted eligibility criteria
Biomarker criteria
Required: NF1 pathogenic germline mutation
Prior therapy
Cannot have received: chemotherapy
Participant has received chemotherapy for any indication within 90 days of starting PAS-004
Cannot have received: PN-directed drug or biologic therapy
Participant has received treatment with any PN-directed drug or biologic therapy within 14 days of starting PAS-004
Cannot have received: strong CYP3A4 inhibitor or inducer; moderate inducers for CYP2C8 and CYP2C9; major substrate of the enzymes above with a narrow therapeutic index
Exception: except for topical skin use
Participant has received treatment with a strong CYP3A4 inhibitor or inducer, or moderate inducers for CYP2C8 and CYP2C9 within 14 days of starting PAS-004, or any drug considered a major substrate of the enzymes above with a narrow therapeutic index except for topical skin use
Cannot have received: growth factors to increase the number or function of platelets or white blood cells
Participant has received growth factors to increase the number or function of platelets or white blood cells within 7 days of starting PAS-004
Cannot have received: radiotherapy
Participant has received radiotherapy, major surgery, or immunotherapy within 28 days of starting PAS-004
Cannot have received: major surgery
Participant has received radiotherapy, major surgery, or immunotherapy within 28 days of starting PAS-004
Cannot have received: immunotherapy
Participant has received radiotherapy, major surgery, or immunotherapy within 28 days of starting PAS-004
Lab requirements
Blood counts
ANC ≥ 1.5 × 10^9/L; Hemoglobin ≥ 90 g/dL; Platelets ≥ 100 × 10^9/L
Kidney function
Creatinine clearance ≥ 60 mL/min
Liver function
Serum total bilirubin ≤ 1.5 × ULN for age (≤ 3.0 × ULN in participants with Gilbert's syndrome); AST ≤ 2.0 × ULN; ALT ≤ 2.0 × ULN; Albumin ≥ 3 g/dL
Cardiac function
Average QTc interval ≤ 480 ms (Fridericia's formula); LVEF ≥ 45%; No grade ≥ 3 congestive heart failure (NYHA); No clinically significant arrhythmias; No significant coronary artery disease, cardiomyopathy, or severe valvular disease
Participant must have adequate organ and bone marrow function at screening as indicated by the following laboratory value ranges: ANC ≥ 1.5 × 10^9/L; Hemoglobin ≥ 90 g/dL; Platelets ≥ 100 × 10^9/L; Serum total bilirubin ≤ 1.5 × ULN for age (≤ 3.0 × ULN in participants with Gilbert's syndrome); AST ≤ 2.0 × ULN; ALT ≤ 2.0 × ULN; Albumin ≥ 3 g/dL; Creatinine clearance ≥ 60 mL/min. Cardiac: Average QTc interval ≤ 480 ms (Fridericia's formula); LVEF ≥ 45%; No grade ≥ 3 congestive heart failure (NYHA); No clinically significant arrhythmias; No significant coronary artery disease, cardiomyopathy, or severe valvular disease
Structured fields extracted by AI. May contain errors — verify against the official protocol.
US trial sites
- University of Alabama at Birmingham · Birmingham, Alabama
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