Back to resultsFCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
Primary Purpose
Neurofibromatosis 1, Plexiform Neurofibroma, NF1
Status
Not yet recruiting
Phase
Phase 3
Locations
Study Type
Interventional
Intervention
Test group (Group A): FCN-159 8 mg, orally, once daily;
Control group (Group B): Placebo, orally, once daily;
Sponsored by
About this trial
This is an interventional treatment trial for Neurofibromatosis 1
Eligibility Criteria
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.
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
FCN-159
placebo
Arm Description
Experimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 8 mg, orally, once daily Method of administration: Oral
Experimental: placebo Dosage form:tablet Specification: 1mg，4mg Dose: placebo 8 mg, orally, once daily Method of administration: Oral
Outcomes
Primary Outcome Measures
Objective response rate (ORR) evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria)
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 Outcome Measures
Objective response rate (ORR) evaluated by the investigator (REiNS criteria)
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.
Duration of response (DOR) evaluated by BIRC and the investigator;
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.
Disease control rate (DCR) evaluated by BIRC and the investigator;
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.
Clinical benefit rate (CBR)evaluated by BIRC and the investigator;
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.
Progression free survival (PFS) evaluated by BIRC and the investigator;
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.
Time to progression (TTP) evaluated by BIRC and the investigator;
TTP is defined as the time from randomization until date of disease progression by BIRC and investigator per REiNS criteria.
Time to response (TTR) evaluated by BIRC and the investigator;
TTR is defined as the time from date of randomization until the date of objective response by BIRC and investigator per REiNS criteria.
Change from baseline in pain intensity score
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.
Change from baseline in appearance
Change in appearance from baseline for Arm A versus Arm B as assessed using a sponsor-customized 'appearance evaluation'PRO questionnaire, which is descriptive.
Full Information
NCT ID
NCT05913037
First Posted
June 6, 2023
Last Updated
June 16, 2023
Sponsor
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT05913037
Brief Title
FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
Official 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
Study Type
Interventional
2. Study Status
Record Verification Date
June 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
June 16, 2023 (Anticipated)
Primary Completion Date
June 30, 2025 (Anticipated)
Study Completion Date
June 30, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
Detailed 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neurofibromatosis 1, Plexiform Neurofibroma, NF1
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
162 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
FCN-159
Arm Type
Experimental
Arm Description
Experimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 8 mg, orally, once daily Method of administration: Oral
Arm Title
placebo
Arm Type
Placebo Comparator
Arm Description
Experimental: placebo Dosage form:tablet Specification: 1mg，4mg Dose: placebo 8 mg, orally, once daily Method of administration: Oral
Intervention Type
Drug
Intervention Name(s)
Test group (Group A): FCN-159 8 mg, orally, once daily;
Intervention Description
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.
Intervention Type
Drug
Intervention Name(s)
Control group (Group B): Placebo, orally, once daily;
Intervention Description
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 Outcome Measure Information:
Title
Objective response rate (ORR) evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria)
Description
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.
Time Frame
Through study completion, an average of 2 years
Secondary Outcome Measure Information:
Title
Objective response rate (ORR) evaluated by the investigator (REiNS criteria)
Description
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.
Time Frame
Through study completion, an average of 2 years
Title
Duration of response (DOR) evaluated by BIRC and the investigator;
Description
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.
Time Frame
Through study completion, an average of 2 years
Title
Disease control rate (DCR) evaluated by BIRC and the investigator;
Description
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.
Time Frame
Through study completion, an average of 2 years
Title
Clinical benefit rate (CBR)evaluated by BIRC and the investigator;
Description
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.
Time Frame
Through study completion, an average of 2 years
Title
Progression free survival (PFS) evaluated by BIRC and the investigator;
Description
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.
Time Frame
Through study completion, an average of 2 years
Title
Time to progression (TTP) evaluated by BIRC and the investigator;
Description
TTP is defined as the time from randomization until date of disease progression by BIRC and investigator per REiNS criteria.
Time Frame
Through study completion, an average of 2 years
Title
Time to response (TTR) evaluated by BIRC and the investigator;
Description
TTR is defined as the time from date of randomization until the date of objective response by BIRC and investigator per REiNS criteria.
Time Frame
Through study completion, an average of 2 years
Title
Change from baseline in pain intensity score
Description
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.
Time Frame
Through study completion, an average of 2 years
Title
Change from baseline in appearance
Description
Change in appearance from baseline for Arm A versus Arm B as assessed using a sponsor-customized 'appearance evaluation'PRO questionnaire, which is descriptive.
Time Frame
Through study completion, an average of 2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
12. IPD Sharing Statement
Learn more about this trial
FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
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