FCN-159 for Neurofibromatosis

Recruiting at 19 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

160 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

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.

How does the drug FCN-159 differ from other treatments for neurofibromatosis?

FCN-159 is unique because it is being specifically studied for neurofibromatosis, a condition with limited standard treatment options, unlike other drugs that have been primarily tested for different cancers like colorectal cancer. ^{1 2 3 4 5}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

References

1. United States pubmed.ncbi.nlm.nih.gov

Phase I-II study of ftorafur and methyl-CCNU in advanced colorectal cancer. [2019]

2. United States pubmed.ncbi.nlm.nih.gov

Phase III study of ICRF-159 versus 5-FU in the treatment of advanced metastatic colorectal carcinoma. [2013]

3. United States pubmed.ncbi.nlm.nih.gov

A randomized clinical trial of combination chemotherapy in advanced colorectal cancer. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Cytotoxicity and in-vivo tolerance of FdUMP[10]: a novel pro-drug of the TS inhibitory nucleotide FdUMP. [2019]

5. England pubmed.ncbi.nlm.nih.gov

Effect of stereoisomers related to ICRF-159 on metastasis of B16 melanoma. [2019]