What side effects are associated with this type of intervention?

MEK1/2 inhibitors, such as FCN-159, are used in the treatment of Neurofibromatosis Type 1. Common side effects of MEK inhibitors include diarrhea, dermatitis acneiform, maculopapular rash, fatigue, dry skin, nausea, dyspnea, and vomiting. These side effects are generally manageable and vary in severity among patients.

What prior FDA approvals exist?

The FDA has approved selumetinib, a MEK1/2 inhibitor, for the treatment of neurofibromatosis type 1 (NF1) in pediatric patients with symptomatic, inoperable plexiform neurofibromas. This approval is based on clinical trials demonstrating its efficacy in reducing tumor size and improving symptoms. Other MEK inhibitors, such as trametinib, have also been studied for their potential benefits in treating NF1-related tumors.

What other types of research exist?

Promising alternatives to FCN-159 for Neurofibromatosis patients include trametinib and selumetinib, both of which are MEK inhibitors that have shown efficacy in related conditions.

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MEK1/2 inhibitor

FCN-159 for Neurofibromatosis

Phase 1 & 2

Waitlist Available

Research Sponsored by Shanghai Fosun Pharmaceutical Development Co, Ltd.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Cohort 1: 16-70 years of age with a body weight of ≥ 94 lbs or 42.5 kg

Participants must have a Karnofsky performance level of ≥70% (adults) or Lansky performance score ≥ 70% (pediatric participants)

Must not have

Radiotherapy, surgery, or immunotherapy within 4 weeks before administration of FCN-159

Participants with dysphagia, active digestive diseases, malabsorption syndrome

Timeline

Screening 3 weeks

Treatment Varies

Follow Up through study completion, an average of 2 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing FCN-159, a new drug taken by mouth, for patients with advanced solid tumors and neurofibromatosis type 1. The drug works by blocking specific proteins that help cancer cells grow. This targeted approach aims to slow down or stop the progression of these diseases.

Who is the study for?

This trial is for adults and children with Neurofibromatosis Type 1 (NF1) who have plexiform neurofibromas (PNs). Adults must be aged 16-70, weigh at least 94 lbs, and children should be aged 2-15. Participants need to have a measurable lesion suitable for MRI scans, not had certain treatments recently, can swallow tablets whole, and agree to use effective contraception if applicable.

What is being tested?

The study tests FCN-159's safety and effectiveness against tumors caused by NF1. FCN-159 is an oral drug designed as a MEK1/2 inhibitor targeting advanced solid tumors. The trial will assess how well it tolerates in different age groups and its impact on tumor size.

What are the potential side effects?

While the specific side effects of FCN-159 are not listed here, similar drugs often cause skin rash, diarrhea, fatigue or weakness. There may also be risks of eye problems like blurred vision or other organ-specific issues due to the inhibition of MEK pathways.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 16 and 70 years old and weigh at least 94 lbs (42.5 kg).

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I am mostly able to care for myself and carry out daily activities.

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I have been diagnosed with neurofibromatosis type 1 (NF1).

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I have a tumor that is at least 3 cm big and can be seen on an MRI.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have not had radiotherapy, surgery, or immunotherapy in the last 4 weeks.

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I have swallowing difficulties or digestive problems that affect how my body absorbs food.

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I am allergic to the study drug or similar medications.

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I have had issues with my eyes, like retinal vein occlusion or glaucoma.

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My high blood pressure is not under control.

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My NF1-related tumor needs treatment like chemo, radiation, or surgery.

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I have not been treated with selumetinib or any MEK 1/2 inhibitors.

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I have interstitial pneumonia.

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I do not have any ongoing infections.

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I've had chemotherapy for NF1 within the last 3 months and still experience significant side effects.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ through study completion, an average of 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~through study completion, an average of 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and through study completion, an average of 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Phase I Changes in NF1-related symptoms.

Phase II Clinical outcome variables: Changes in pain intensity.

Phase II Dose intensity

+6 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Single ArmExperimental Treatment1 Intervention

0 / 14Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

MEK1/2 inhibitors, such as FCN-159, work by blocking the MEK1/2 enzymes in the MAPK/ERK signaling pathway, which is often overactive in Neurofibromatosis type 1 (NF1). This inhibition helps to reduce tumor growth and proliferation. For patients with NF1, targeting this pathway is crucial because it directly addresses the underlying genetic mutation that leads to tumor development. By inhibiting MEK1/2, these treatments can potentially control tumor growth and improve patient outcomes, offering a targeted therapeutic approach that addresses the specific molecular abnormalities in NF1.

Summary statement novel agents in the treatment of lung cancer: Fifth Cambridge Conference assessing opportunities for combination therapy.Clinical, genetic and pharmacological data support targeting the MEK5/ERK5 module in lung cancer.Lorlatinib Treatment Elicits Multiple On- and Off-Target Mechanisms of Resistance in ALK-Driven Cancer.