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Active, Not Recruiting

NCT Number: NCT05913037

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

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Neurofibromatosis 1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System NF1 Neoplasms Neoplasms by Histologic Type Neoplasms, Nerve Tissue Neoplastic Syndromes, Hereditary Nerve Sheath Neoplasms Nervous System Diseases Nervous System Neoplasms Neurocutaneous Syndromes Neurodegenerative Diseases Neurofibroma Neurofibroma, Plexiform Neurofibromatoses Neuromuscular Diseases Peripheral Nervous System Diseases Peripheral Nervous System Neoplasms Plexiform Neurofibroma

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Cancer Hospital Chinese Academy of Medical Sciences, Beijing, Beijing Municipality, China

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • NF1-related malignancies requiring chemotherapy, radiotherapy, or surgery, such as medium to high grade optic glioma or malignant peripheral nerve sheath tumor.
  • 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).
  • Patients who cannot undergo MRI and/or have contraindications to MRI.
  • Patients with previous or current retinal vein obstruction (RVO), retinal pigment epithelial detachment (RPED), glaucoma, and other abnormal ophthalmic examination with clinical significance.
  • Interstitial pneumonia, including clinically significant radiation pneumonia.
  • Cardiac function or combined diseases meet one of the following conditions:
  • QTcF value of > 470 milliseconds; patients with risk factors for QTcF prolongation or patients receiving drugs that prolong the QTcF interval.
  • Congestive heart failure per New York Heart Association (NYHA) classification ≥ Class 3.
  • Arrhythmias with clinical significance.
  • Known concurrent clinically significant coronary artery disease, cardiomyopathy, and severe valvular disease.
  • LVEF < 50%.
  • Patients with a heart rate of < 50 beats/min.

Treatment and study plan

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

Drug

After completing all screening visit items, qualified patients will be randomly assigned to the test group (Group A) or control group (Group B) in a 2:1 ratio, and receive FCN-159 or placebo within 3 days after randomization.

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

Drug

After completing all screening visit items, qualified patients will be randomly assigned to the test group (Group A) or control group (Group B) in a 2:1 ratio, and receive FCN-159 or placebo within 3 days after randomization.

Primary outcomes

  1. Objective response rate (ORR) evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria)

    Time frame: Through study completion, an average of 2 years

    ORR is defined as the proportion of patients who have a confirmed complete response or confirmed partial response as determined by ICR per REiNS criteria.

Secondary outcomes

  1. Objective response rate (ORR) evaluated by the investigator (REiNS criteria)

    Time frame: Through study completion, an average of 2 years

    ORR is defined as the proportion of patients who have a confirmed complete response or confirmed partial response as determined by ICR per REiNS criteria.

  2. Duration of response (DOR) evaluated by BIRC and the investigator;

    Time frame: Through study completion, an average of 2 years

    DOR is defined as the time from the date of first documented response (which is subsequently confirmed) until progression by BIRC and the investigator per REiNS criteria or death due to any cause.

  3. Disease control rate (DCR) evaluated by BIRC and the investigator;

    Time frame: Through study completion, an average of 2 years

    DCR is defined as the proportion of patients who have a confirmed complete response or confirmed partial response or stable disease as determined by BIRC and the investigator per REiNS criteria.

  4. Clinical benefit rate (CBR)evaluated by BIRC and the investigator;

    Time frame: Through study completion, an average of 2 years

    CBR is defined as the proportion of patients who have a confirmed complete response or confirmed partial response or stable disease>48 weeks as determined by BIRC and the investigator per REiNS criteria.

  5. Progression free survival (PFS) evaluated by BIRC and the investigator;

    Time frame: Through study completion, an average of 2 years

    PFS is defined as the time from randomization until date of disease progression by BIRC and investigator per REiNS criteria or death due to any cause.

  6. Time to progression (TTP) evaluated by BIRC and the investigator;

    Time frame: Through study completion, an average of 2 years

    TTP is defined as the time from randomization until date of disease progression by BIRC and investigator per REiNS criteria.

  7. Time to response (TTR) evaluated by BIRC and the investigator;

    Time frame: Through study completion, an average of 2 years

    TTR is defined as the time from date of randomization until the date of objective response by BIRC and investigator per REiNS criteria.

  8. Change from baseline in pain intensity score

    Time frame: Through study completion, an average of 2 years

    Difference in mean change from baseline in overall tumor and target PN pain intensity score between Arm A and Arm B as assessed by the 11-point Numerical Rating Scale (NRS-11),which uses the range 0-10,higher scores mean worse outcome.

  9. Change from baseline in appearance

    Time frame: Through study completion, an average of 2 years

    Change in appearance from baseline for Arm A versus Arm B as assessed using a sponsor-customized 'appearance evaluation'PRO questionnaire, which is descriptive.

Sponsors and collaborators

Lead sponsor

Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2025
Study completion
2026
First posted
Jun 22, 2023
Registry last updated
Mar 6, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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