Study of PAS-004 for adults with NF1 and plexiform neurofibromas
Treatments studied
Part of Brain & nervous system, Cancer, Genetic & congenital clinical trials.
This trial tests an oral medication called PAS-004 for adults with Neurofibromatosis Type 1 who have at least one symptomatic plexiform neurofibroma that cannot be surgically removed. The goal is to see if the drug can shrink these tumors or improve symptoms, and to understand side effects.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be at least 18 years old.
- You have been diagnosed with NF1 and have at least one plexiform neurofibroma that is at least 3 cm wide and causes symptoms like pain, deformity, or nerve problems.
- You must be able to swallow pills and have good organ function (kidneys, liver, bone marrow).
- You cannot have taken any NF1-directed drug or strong CYP3A4-affecting medications within 14 days before starting.
- You cannot have active cancer (except some skin cancers), uncontrolled high blood pressure, or certain heart or eye conditions.
- You must be willing to undergo MRI scans and possibly biopsies of small skin tumors.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This phase 1 trial tests an oral drug called PAS-004 for people with advanced solid tumors that have certain gene changes. It aims to find a safe dose and see if the drug can stop tumor growth.
This trial tests an experimental drug called HLX-1502 for people with NF1 who have a plexiform neurofibroma (a type of nerve tumor) that is growing or causing problems. It aims to see if the drug is safe and can shrink or stop the tumor from worsening.
This trial tests a combination of two targeted drugs (cabozantinib and selumetinib) for people with NF1 who have plexiform neurofibromas that are growing or causing significant problems. The goal is to see if this combination can shrink or slow the growth of these tumors.
This study tests whether the drug selumetinib can prevent the growth of plexiform neurofibromas (nerve tumors) in young children with NF1. Children are first monitored with MRI scans, and if a tumor appears or grows, they may receive treatment.
This Phase 2 trial tests HL-085 in adults with neurofibromatosis type 1 (NF1) who have a nerve tumor (plexiform neurofibroma) that cannot be removed by surgery and causes symptoms. It aims to shrink or control the tumor by measuring changes on MRI and monitoring side effects.
This study checks whether patient surveys (questionnaires) can reliably measure symptoms and pain in people with NF1 who have plexiform neurofibromas. It helps create better tools for tracking how you feel and how treatments may affect quality of life.
Hear when a new NF1 Mutation trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.