FCN-159 in Adult Patients with Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas

Fosun Pharma

Status and phase: Active, not recruiting; Phase 3

Conditions: NF1; Neurofibromatosis 1; Plexiform Neurofibroma

Treatments: Drug: Test group (Group A): FCN-159 8 mg, orally, once daily;; Drug: Control group (Group B): Placebo, orally, once daily;

Study type: Interventional

Funder types: Industry

Identifiers

NCT05913037

FCN-159-007

Details and patient eligibility

About

A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.

Full description

This is a randomized, double-blind, placebo-controlled, multi-center phase III clinical study to evaluate the efficacy and safety of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.

Enrollment: 167 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. ≥ 18 years old and ≤ 70 years old.
2. Patients must be diagnosed with symptomatic NF1-related plexiform neurofibromas (PNs) and require systemic therapy at the investigator's discretion.
3. Presence of measurable lesions, defined as ≥ 3 cm in length in at least one dimension, which can be evaluated for efficacy by MRI.
4. Karnofsky performance status score ≥ 70.
5. Patients with adequate organ and bone marrow functions.

Exclusion criteria

1. NF1-related malignancies requiring chemotherapy, radiotherapy, or surgery, such as medium to high grade optic glioma or malignant peripheral nerve sheath tumor.

2. Patients with a history of or concurrently with other malignancies (excluding cured non-melanoma skin basal cell carcinoma, breast cancer in situ or cervical cancer in situ, and other malignancies without evidence of disease within 5 years).

3. Patients who cannot undergo MRI and/or have contraindications to MRI.

4. Patients with previous or current retinal vein obstruction (RVO), retinal pigment epithelial detachment (RPED), glaucoma, and other abnormal ophthalmic examination with clinical significance.

5. Interstitial pneumonia, including clinically significant radiation pneumonia.

6. Cardiac function or combined diseases meet one of the following conditions:

   1. QTcF value of > 470 milliseconds; patients with risk factors for QTcF prolongation or patients receiving drugs that prolong the QTcF interval.
   2. Congestive heart failure per New York Heart Association (NYHA) classification ≥ Class 3.
   3. Arrhythmias with clinical significance.
   4. Known concurrent clinically significant coronary artery disease, cardiomyopathy, and severe valvular disease.
   5. LVEF < 50%.
   6. Patients with a heart rate of < 50 beats/min.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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167 participants in 2 patient groups, including a placebo group

FCN-159

Experimental group

Description:

Experimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 8 mg, orally, once daily Method of administration: Oral

Treatment:

Drug: Test group (Group A): FCN-159 8 mg, orally, once daily;

placebo

Placebo Comparator group

Description:

Experimental: placebo Dosage form:tablet Specification: 1mg，4mg Dose: placebo 8 mg, orally, once daily Method of administration: Oral

Treatment:

Drug: Control group (Group B): Placebo, orally, once daily;