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New treatment option for NF1 Mutation
Official title PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
ClinicalTrials.gov ID: NCT06961565
What this study is testing
What is PAS-004 Tablets?
PAS-004 Tablets is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for nf1 mutation.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
- Time commitment: about 6 months
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Participant is capable of providing informed consent, which includes compliance with the requirements, prohibitions and restrictions listed in the...
- Participant has been informed both verbally and in writing about the objectives of the clinical study, the methods, the anticipated benefits, the...
- 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...
You likely can't join if
- Participant has participated in another treatment clinical study within 28 days of starting PAS-004.
- Participant has received chemotherapy for any indication within 90 days of starting PAS-004.
- Participant has ongoing side effects from prior chemotherapy that are worse than mild (except alopecia). ("Mild" is defined as Asymptomatic or mild...
- Participant has received treatment with any PN-directed drug or biologic therapy within 14 days of starting PAS-004.
- Participant has received treatment with a strong CYP3A4 inhibitor or inducer, or moderate inducers for CYP2C8 and CYP2C9 within 14 days of starting...
- Participant has received growth factors to increase the number or function of platelets or white blood cells within 7 days of starting PAS-004.
See the full eligibility criteria
Who can join
- 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...
- 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...
- Participant has been diagnosed with NF1 based upon the following diagnostic criteria: a. Clinical and imaging confirmation meeting at least two of the following NF1 diagnostic criteria in accordance with the clinical...
- Measurable is defined as: 1. non-pedunculated (no stalk) 2. surrounded by visually uninvolved skin and not in physical contact with another CN, 3. measuring between 6 and 15 mm in the longest diameter and exophytic on...
- At least seven CN should be located on the trunk, neck, and/or limbs measuring between 6 and 15 mm in maximal diameter, and "measurable" as per the definition listed above.
- Participants with CN meeting the above criteria will undergo optional resections of at least two CN that meet eligibility criteria. If participant is willing to accept additional CN resections, this is permitted for up...
What rules you out
- Participant has participated in another treatment clinical study within 28 days of starting PAS-004.
- Participant has received chemotherapy for any indication within 90 days of starting PAS-004.
- Participant has ongoing side effects from prior chemotherapy that are worse than mild (except alopecia). ("Mild" is defined as Asymptomatic or mild symptoms, clinical or diagnostic observations only, or intervention not...
- Participant has received treatment with any PN-directed drug or biologic therapy within 14 days of starting PAS-004.
- 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...
- Participant has received growth factors to increase the number or function of platelets or white blood cells within 7 days of starting PAS-004.
- Participant has received radiotherapy, major surgery, or immunotherapy within 28 days of starting PAS-004.
- Participant has malignant tumors associated with NF1 requiring chemotherapy, radiotherapy, or surgery, such as intermediate- to high-grade gliomas or malignant peripheral nerve sheath tumors.
- Participant has a current malignancy (excluding cured non-melanomatous skin cancer, breast carcinoma in situ, or cervical cancer in situ) or has history of malignancy requiring active treatment within the past 5 years...
- Participant has uncontrolled hypertension defined as blood pressures \>150/90 mmHg on repeat examinations despite maximal medical management. Note: Participants with controlled hypertension with anti-hypertension...
- Participant has active dysphagia, digestive system disease, malabsorption syndrome, or other conditions that might affect the absorption of PAS-004.
- Participant has previous or current retinal vein occlusion (RVO), retinal pigment epithelial detachments (RPED), clinically active glaucoma, or other significant abnormality in screening ophthalmic examination.
- Participant has interstitial pneumonia, NF1-related pulmonary disease, including existing clinically significant radiation pneumonitis.
- Participant has impaired cardiac function or cardiac disease as indicated by:
- Average QTc interval \> 480 ms calculated using the Fridericia's QT interval correction formula.
- Grade ≥ 3 congestive heart failure per New York Heart Association (NYHA) guidelines.
- Clinically significant arrhythmias, including but not limited to, complete left bundle branch conduction abnormalities and 2nd degree atrioventricular block.
- Known concurrent clinically significant coronary artery disease, cardiomyopathy, or severe valvular disease.
- Echocardiogram or multi-gated acquisition (MUGA) scan performed during the screening showing impaired left ventricular ejection fraction (LVEF) \< 45%.
- Participant has taken a QTc-prolonging medication within seven days of IP initiation or longer if the half-life of the QTc prolonging medication is such that the drug is not cleared from the body within 7 days (5...
- Participant has an uncontrolled bacterial, fungal, or viral infections, including active hepatitis B (hepatitis B virus surface antigen positive and hepatitis B virus DNA \> 1000 IU/ml or meeting the study site's...
- Any clinically significant active or known history of liver disease or known hepatobiliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones).
- Participant has a known hypersensitivity to PAS-004, its excipients, or another MEK 1/2 inhibitor.
- Participant is pregnant or lactating.
- Participant has a clinically significant condition that, in the opinion of the investigator, would preclude study participation or compliance with safety requirements.
- Participant is unable to attend in-person clinic visits per clinical site guidelines and restrictions.
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- St Leonards, New South Wales, Australia
- Melbourne, Victoria, Australia
- Seoul, South Korea
- Sinchŏn-dong, South Korea
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 6 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; St Leonards, New South Wales, Australia; Melbourne, Victoria, Australia; Seoul, South Korea; Sinchŏn-dong, South Korea. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.