The goal of this clinical trial is to evaluate the efficacy and safety of FCN-159 in patients with specific NF2-associated nerve sheath tumors.
1. A phase 2 single arm single center study, total 30 patients will be enrolled; 2. To evaluate the safety and efficacy of FCN-159, a Mek inhibitor, to treat NF2; associated nerve sheath tumors, age≥16, including benign and malignant tumors; 3. Primary endpoint: Objective response rate (ORR);Secondary Outcome Measures: Clinical benefit rate (CBR);24w WRS, OS et al..
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
FCN-159 8mg qd po
Shuhang Wang
Beijing, Beijing Municipality, China
RECRUITINGObjective response rate (ORR) estimated by investigators
Investigator-assessed objective mitigation rate ORR (Reponse evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria and RECIST 1.1 criteria, respectively)
Time frame: Every 4 cycles (each cycle is 28 days), assessed up to 2 years
Clinical benefit rate (CBR) estimated by investigators
Clinical benefit rate CBR will be assessed by the investigator (REiNS criteria and RECIST 1.1 criteria respectively)
Time frame: Every 4 cycles (each cycle is 28 days), assessed up to 2 years
24-week word recognition score (WRS)
Hearing response changes will be assessed in patients with hearing impairment (24-week word recognition scores and pure tone audiometry).
Time frame: up to 24 weeks
pain estimated by NRS
Change in pain intensity score relative to baseline will be assessed according to NRS scale.
Time frame: Every 4 cycles (each cycle is 28 days), assessed up to 2 years
Duration of response (DOR) assessed by investigator
Duration of response (DoR) is estimated by the investigators as the time from treatment initiation to disease progression or death in patients who achieve complete or partial response, using both RECIST V1.1 and REINS criteria.
Time frame: From date of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 2 years
progression free survival (PFS) assessed by investigator
PFS is estimated by the investigators as the time from treatment initiation to disease progression or death in patients, using both RECIST V1.1 and REINS criteria.
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Time frame: From date of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 2 years