FCN-159 for Neurofibromatosis

Not currently recruiting at 20 trial locations

Overseen By Vitalina Dzutseva

Age: Any Age

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Shanghai Fosun Pharmaceutical Development Co, Ltd.

Must not be taking: Chemotherapy, Strong CYP inhibitors

Disqualifiers: Malignant tumors, Uncontrolled hypertension, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of FCN-159, a promising oral treatment for individuals with neurofibromatosis type 1 (NF1), a condition that can cause nerve tumors known as plexiform neurofibromas. FCN-159 targets specific proteins to help reduce tumor growth. Participants must have NF1 with symptomatic plexiform neurofibromas requiring treatment and face significant risk if undergoing surgery. The study aims to find new ways to manage the physical and potentially life-altering symptoms associated with NF1. As a Phase 1 trial, this research seeks to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Will I have to stop taking my current medications?

The trial protocol does not specify if you must stop taking your current medications. However, you cannot participate if you have taken certain treatments like chemotherapy for NF1 within 3 months, or specific drugs or therapies within 14 days before starting FCN-159.

Is there any evidence suggesting that FCN-159 is likely to be safe for humans?

Research has shown that FCN-159, a treatment for neurofibromatosis, is generally well-tolerated. In studies, participants took doses up to 8 mg daily, experiencing manageable side effects. These side effects were not serious and were easy to control. The treatment also demonstrated promising results in combating tumors related to neurofibromatosis. Although further testing is ongoing, the initial safety data appears encouraging for potential trial participants. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for neurofibromatosis, which often focus on managing symptoms or involve surgical removal of tumors, FCN-159 is designed to target the underlying cause of the condition. FCN-159 works by inhibiting MEK, a key protein in the pathway that leads to tumor growth in neurofibromatosis patients. Researchers are excited about FCN-159 because this targeted approach could potentially slow or stop the growth of tumors, offering a more effective and less invasive option compared to current therapies.

What evidence suggests that FCN-159 might be an effective treatment for neurofibromatosis?

Research has shown that FCN-159 holds promise in treating neurofibromatosis type 1 (NF1), a condition that causes tumors to grow along nerves. Studies have found that this treatment is well-tolerated and can significantly shrink tumors, with some reducing by as much as 84.2%. FCN-159 blocks certain proteins that aid cell growth, potentially stopping the growth of NF1-related tumors. Trials have reported manageable side effects, indicating that the treatment is both effective and safe. Overall, these findings highlight FCN-159's potential as a targeted therapy for tumors related to NF1. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I will avoid too much sun and use sunscreen if I'm in the sun.

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

Coagulation function: International normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤ 1.5 ULN

Exclusion Criteria

Participation in other interventional clinical trials within 4 weeks before administration of FCN-159

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

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose-escalation

Participants receive escalating doses of FCN-159 to evaluate safety and tolerability

6 months

Dose-expansion

Participants receive the determined dose of FCN-159 to evaluate anti-tumor activity

18 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• FCN-159

Trial Overview 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.

How Is the Trial Designed?

Group I: Single Arm Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Shanghai Fosun Pharmaceutical Development Co, Ltd.

Lead Sponsor

Trials

5

Recruited

880+

Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.

Lead Sponsor

Trials

17

Recruited

5,400+

Citations

1. ascopubs.org ascopubs.org/doi/10.1200/JCO.2025.43.16_suppl.10044

Updated data of efficacy and safety of luvometinib (FCN ... Overall, luvometinib was well-tolerated and demonstrated promising anti-tumor activity in pediatric participants with NF1-related PN.

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/37400844/

Phase 1 dose-escalation study to evaluate the safety, ... FCN-159 was well tolerated up to 8 mg daily with manageable adverse events and showed promising anti-tumorigenic activity in patients with NF1-related PN.

3. ascopubs.org ascopubs.org/doi/10.1200/JCO.2024.42.16_suppl.3096

Efficacy and safety of FCN-159, a MEK1/2 inhibitor in ... Conclusions: FCN-159 demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN. Clinical ...

4. asco.org asco.org/abstracts-presentations/ABSTRACT371130

A multicenter, open-label, single-arm, phase 1 dose- ... The largest reduction in tumor size was 84.2%. Conclusions: Overall, FCN-159 at 8 mg is well tolerated, with easy to manage adverse events, and showed promising ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT04954001

NCT04954001 | Study to Evaluate the Safety, Tolerability, ... Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis ...