Clinical trial NCT06553365: Phase II Study of FCN-159 in NF2 Nerve Sheath Tumors
Official title: An Open, Single-Arm Phase II Study Evaluating the Efficacy and Safety of FCN-159 in Selected NF2-associated Nerve Sheath Tumors
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE2
Conditions: Nerve Sheath Tumor
Keywords: MEK
NF2
nerve sheath tumor
Lead sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Sponsor class: OTHER
First posted: 2024-08-14
Enrollment: 30
Sex: ALL
Minimum age (years): 16
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: 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..

Eligibility criteria: Inclusion Criteria:

1. ≥16 years of age, regardless of gender.
2. meet the revised 2022 diagnostic criteria for NF2-associated nerve sheath tumors or pathologically confirmed NF2-associated nerve sheath tumors.
3. should meet one of the following criteria: 1) Incomplete surgical resection, or postoperative recurrence. 2) Systemic therapy is required as determined by the Investigator.
4. the presence of a measurable lesion, as defined by REiNS or RECIST V1.1 criteria.
5. Karnofsky physical status score of ≥70.
6. the patient has adequate organ and bone marrow function.
7. International Normalized Ratio (INR) and activated partial thromboplastin time (APTT) ≤ 1.5 x ULN.
8. For patients of childbearing potential: During treatment and for at least 90 days after the last dose, patients should agree to use a highly effective method of contraception.
9. avoid excessive sun exposure and be willing to use an adequate amount of sunscreen in anticipation of sun exposure.
10. be able to understand and voluntarily sign a written informed consent form.

Exclusion Criteria:

* Previously received one of the following treatments:

  1. Pharmaceutical or biological therapy within 3 weeks or 5 half-lives prior to enrollment, whichever is longer.
  2. Use of growth factors that promote platelet, red blood cell, or white blood cell count or function within 7 days prior to enrollment.
  3. Patients who have received major surgery or anti-tumor immunotherapy within 4 weeks prior to enrollment.
  4. Radiation therapy for nerve sheath tumors within 4 weeks prior to enrollment.
  5. Dose adjustment for patients treated with dexamethasone or other corticosteroids within 1 week prior to enrollment.
  6. Patients who have participated in another interventional clinical trial within 4 weeks prior to enrollment.
  7. Prior treatment with Selumetinib or any other MEK 1/2 inhibitor. 2. history of or concurrent with other malignancies. 3. inability to undergo MRI and/or contraindications to MRI. 4. uncontrolled hypertension. 5. the presence of dysphagia, active gastrointestinal disease, malabsorption syndrome, or other condition that interferes with the absorption of the study medication.

     6. previous or current retinal vascular disease. 7. interstitial pneumonitis 8. cardiac function or co-morbidities 9. Immediate family history of sudden cardiac death before age 50. 10.History of any acute neurological condition 11. with active bacterial, fungal or viral infections 12. known hypersensitivity to the study drug, other MEK1/2 inhibitors, or their excipients.

Primary outcomes:
measure: Objective response rate (ORR) estimated by investigators
timeFrame: Every 4 cycles (each cycle is 28 days), assessed up to 2 years
Secondary outcomes:
measure: Clinical benefit rate (CBR) estimated by investigators
timeFrame: Every 4 cycles (each cycle is 28 days), assessed up to 2 years
measure: 24-week word recognition score (WRS)
timeFrame: up to 24 weeks
measure: pain estimated by NRS
timeFrame: Every 4 cycles (each cycle is 28 days), assessed up to 2 years
measure: Duration of response (DOR) assessed by investigator
timeFrame: 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
measure: progression free survival (PFS) assessed by investigator
timeFrame: 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
Other outcomes:
measure: life quality assessed by NFTI-QOL
timeFrame: Every 4 cycles (each cycle is 28 days), assessed up to 2 years
measure: Incidence of Treatment-Emergent Adverse Events as assessed by CTCAE 5.0
timeFrame: During the intervention
Locations:
Study officials:
affiliation: Cancer Institute and Hospital, Chinese Academy of Medical Sciences
name: Shuhang wang, PhD
role: STUDY_DIRECTOR
References: