Neurofibromatosis 1Phase 1/2NCT04954001
Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
A Phase 1/2 study testing FCN-159 in people with Neurofibromatosis 1 and Plexiform Neurofibroma, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 14 sites in 3 countries.
Trial summary
FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib. FCN-159 is an orally available and highly potent selective inhibitor of MEK1/2, which is expected to be a targeted therapy for the treatment of advanced solid tumors and… Read more about this study
- Who is it for?
- Ages 2 to 70 years
- Conditions the study is for
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- What is being tested?
- FCN-159
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 160 participants
- Where
- 14 sitesin 3 countries
- Study dates
- 26 Mar 2021to 30 Mar 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- General inclusion criteria for Phase I and II:
- 1.Cohort 1: 16-70 years of age (inclusive) with a body weight of ≥ 94 lbs or 42.5 kg.
- Cohort 2: 2-15 years of age (inclusive) and able to swallow whole tablet. 2.Participants must be diagnosed with NF1-related plexiform neurofibromas (PN) and symptomatic with requirement of systematic therapy per investigator's judgment. A PN is defined as a neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches. A spinal PN involves two or more levels with connection between the levels or extending laterally along the nerve. Diagnosis of neurofibromatosis type 1 (NF1) is based on meeting at least 1 of the following 2 diagnostic criteria:
- Genetic testing confirmation: i.e., positive for NF1 germline mutation per CLIA-certified laboratory (or equivalent) testing. Note: NF1 germline mutation positive must either be confirmed by the FCN-159-002 central laboratory or have documentation of NF1 mutation issued by a CLIA-certified laboratory (or equivalent) - OR -
- Clinical and imaging confirmation: Meets at least 2 of the following 7 NF1 diagnostic criteria according to the clinical NIH consensus criteria:
- ≥ 6 cafe-au-lait macules (>0.5 cm in prepubertal participants and > 1.5 cm in post-pubertal participants);
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- Axillary freckling or freckling in inguinal regions;
- ≥2 neurofibromas of any type, or ≥ 1 plexiform neurofibroma;
- An optic pathway glioma;
- ≥2 Lisch nodules (iris hamartomas);
- A distinctive bony lesion such as dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex);
- First-degree relative with NF1. 3. Participants should meet one of the following criteria
- <!-- -->
- Must be judged by the investigator to be inoperable for complete resection without causing substantial damage, or unsuitable for surgery with high surgical risks , e.g. due to encasement of or close proximity to vital structures, invasiveness, or high vascularity、Extensive lesion scope surgery is not feasible. NF1 has to cause or has the potential to cause significant morbidity, such as (but not limited to) head and neck lesions that could compromise the airway or great vessels, paraspinal lesions that can cause myelopathy, brachial or lumbar plexus lesions that could cause nerve compression and loss of function, lesions that could result in major deformity (e.g., orbital lesions) or are significantly disfiguring, lesions of the extremity that cause limb hypertrophy or loss of function, and painful lesions.
- The participants who have previously received surgical treatment, if the PN resection is incomplete, the postoperative residual exceeds 15% of the primary lesion, or relapse after surgery, and the lesions of at least 3 cm are measured in one dimension, are eligible for enrollment. At least a 28-day interval is required between surgery and the first dose of FCN-159.
- 4. Participants must have a measurable lesion, defined as at least 3 cm in length in at least one dimension, amenable to MRI for efficacy assessment.
- 5. Adult participants: Karnofsky performance level of ≥70%; Pediatric participants: Lansky performance score ≥ 70%, see Appendix 18.
- Note: Participants who are wheelchair bound because of paralysis secondary to a plexiform neurofibroma should be considered ambulatory when they are in the wheelchair. Similarly, participants with limited mobility secondary to the need for mechanical support (such as an airway PN requiring tracheostomy or CPAP) will also be considered ambulatory for the purposes of this study.
- 6. Coagulation function: International normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤ 1.5 ULN.
- 7. Participants or their legal guardians (if the participant is <18 years old) are able to understand and voluntarily sign a written informed consent form.
- 8. For participants of childbearing potential: participants must agree to take effective contraception, and receive double barrier contraception, condom, oral or injectable contraceptives, intrauterine device and other contraceptive methods during treatment and for at least 90 days after the last dose. Male participants must agree to avoid sperm donation for at least 90 days after the last dose.
- 9. Willing to avoid excessive sun exposure and use adequate amounts of sunscreen if sun exposure is anticipated.
Who cannot join
- Participants who meet any of the following conditions shall not be included in this clinical study:
- Participants who have previously received one of the following:
- Chemotherapy for NF1 within 3 months of enrollment. Ongoing side effects of that treatment > Grade 1 (except alopecia).
- Treatment with any drug or biologic therapy within 14 days of starting FCN-159, such as: tipifarnib, pirfenidone, Peg-Interferon, sorafenib or other VEGFR inhibitors
- Strong CYP3A4, CYP2C8 and CYP2C9 inhibitors or inducers (moderate inducers for CYP2C8 and CYP2C9) within 14 days before treatment of the study drug, except for topical skin use.
- Use of growth factors to increase the number or function of platelets or white blood cells within 7 days before administration of FCN-159.
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- Radiotherapy, surgery or immunotherapy within 4 weeks before administration of FCN-159.
- Participation in other interventional clinical trials within 4 weeks before administration of FCN-159.
- Prior treatment with selumetinib or any other MEK 1/2 inhibitors (specific for phase 2 part).
- Participants with malignant tumors associated with NF1 requiring chemotherapy, radiotherapy, or surgery, such as intermediate- to high-grade optic gliomas or malignant peripheral nerve sheath tumors.
- Patients have other malignant tumor history or with other malignant tumors simultaneously (excluding cured non-melanoma skin basal cell carcinoma, breast carcinoma in situ or cervix cancer in situ, and other malignant tumors without disease evidence for the past 5 years);
- Participants who are unable to undergo MRI examination and/or for whom MRI examination is contraindicated (e.g., due to prostheses, orthotics or dental appliances or due to interference with volumetric analysis of target PN on MRI).
- Uncontrolled hypertension (despite medical therapy)
- Adult participants: defined as systolic or diastolic blood pressures > 140/90 mmHg on repeat examination with existing anti-hypertension therapy.
- Pediatric participants: Blood pressure (BP) greater than or equal to the 95th percentile for age, height, and gender measured as described in (Appendix 19).
- Participants with dysphagia, active digestive diseases, malabsorption syndrome, or other conditions that might affect the absorption of the study drug.
- Previous or current retinal vein occlusion (RVO), retinal pigment epithelial detachments (RPED), glaucoma and other significant abnormality in ophthalmic examination.
- Interstitial pneumonia, including existing clinically significant radiation pneumonitis.
- Cardiac dysfunction or concomitant diseases meeting any one of the following conditions will be excluded:
- Three 12-lead electrocardiogram (ECG) measurements performed at the study site during the screening period for which the mean value of three measurements was calculated according to the QTcF formula using the instrument, with QTcF > 470 milliseconds; Participants with risk factors for QTcF prolongation, such as uncorrectable hypokalemia, hereditary long QT syndrome; or receiving drugs that prolong QTcF interval (mainly class Ia, Ic, III antiarrhythmic drugs). Drugs with potential to prolong QTcF interval, See Appendix 20.
- New York Heart Association (NYHA) Class ≥ 3 congestive heart failure;
- Clinically significant arrhythmia, including but not limited to complete left bundle branch block, second degree atrioventricular block;
- Known concurrent clinically significant coronary artery disease, cardiomyopathy, severe valvular disease.
- Ultrasound Cardiogram performed during the screening showing. Left ventricular ejection fraction LVEF < 50%.
- Participants with active bacterial, fungal or viral infections, including active hepatitis B (hepatitis B virus surface antigen positive and hepatitis B virus DNA > 1000 IU/ml or meeting the study site's diagnostic criteria for active hepatitis B infection), hepatitis C (hepatitis C virus RNA positive), or human immunodeficiency virus infection (HIV positive).
- Pregnant or lactating women.
- Known hypersensitivity to the study drug, other MEK 1/2 inhibitor or its excipients.
- Clinically significant condition that, in the opinion of the investigator, would preclude study participation or compliance with safety requirements.
- Inability to attend in-person appointments per current clinical site COVID 19 guidelines and restrictions.
Trial locations and contacts
14 sites in 3 countries.
14 sites in 3 countries
China6 sites
Research Site
Beijing
Research Site
Guangzhou
Research Site
Hangzhou
Research Site
Shanghai
Research Site
Shijiazhuang
Research Site
Wuhan
United States5 sites
Medical University of South Carolina - Hollings Cancer Center - PPDS
Charleston, South Carolina, 29425
Henry Ford Health System
Detroit, Michigan, 48202
Principal Investigator Hans
Gainesville, Florida, 32608
Children's Hospital Los Angeles
Los Angeles, California, 90027
John Hopkins All Children Hospital
St. Petersburg, Florida, 33701
Spain3 sites
Hospital Universitario Vall d'Hebron
Barcelona
Hospital Universitario 12 de Octubre
Madrid
Hospital Universitario La Paz
Madrid
About this study
Official title: A Multi-center, Open-label, Single-arm Phase I Dose-escalation and Phase II Dose-expansion Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib. FCN-159 is an orally available and highly potent selective inhibitor of MEK1/2, which is expected to be a targeted therapy for the treatment of advanced solid tumors and neurofibromatosis type 1.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Being tested
- FCN-159
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FCN-159Drug
FCN-159 is administered orally in once daily schedule for 28 days a cycle.
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | Not described Receives: Drug: FCN-159 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase I: Safety: Dose-limiting toxicity (DLT) incidence rate.
Measured: 28 days after the dose of FCN-159 for DLT
Phase I: Safety: MTD and RP2D.
Measured: Approximately 6-9 months for MTD and RP2D (phase I duration)
Phase II: Objective response rate (ORR) by investigator assessment
Measured: Through study completion, an average of 2 years
Secondary outcomes (18)
Phase I Other safety: - The type and frequency of adverse events (AE) - Treatment-Emergent Serious Adverse Events (SAE) - The frequency and causes of death events - Laboratory safety test results - Changes in vital signs
Measured: Through study completion, an average of 2 years
Phase I Efficacy: objective response rate (ORR) by investigator /BIRC assessment, clinical benefit rate (CBR) per investigator/BIRC assessment including CR, PR, and SD lasting more than 6 months;
Measured: Through study completion, an average of 2 years
Phase I: Maximum Observed Plasma Concentration (Cmax) After Single Dose and Multiple Dose of FCN-159
Measured: Pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 10, 24 hours post-dose on Cycle 1 Day 1 (1 cycle = 28 days) for single dose, Pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 10, 24 hours post-dose on Cycle 1 Day 28 for multiple dose
Phase I: AUC From Time Zero to Last Measurable Concentration (AUClast) After Single Dose and Multiple Dose of FCN-159
Measured: Pre-dose (0 hour), 0.5~1, 1.5~3, 4~6, 24 hours on Day 1 of Cycles 2, Pre-dose (0 hour) on Cycle 5 and every 4 cycles thereafter (assessed up to 104 weeks) (1 cycle = 28 days)
Phase I PD marker: ERK phosphorylation inhibition in peripheral blood mononuclear cells (PBMCs)
Measured: During Cycle 1 (cycle is 28 days): Day 1, Day 8 and Day 28
Phase I Changes in NF1-related symptoms.
Each participant will undergo standardized functional evaluations according to the relevant category or categories of PN-related complications. The incidence of patients with improved function or quality of life will be measured. Key measurements are chosen from each complication category to assess for change over time.
Measured: Through study completion, an average of 2 years]
Phase II: Critical secondary endpoint: Objective response rate (ORR) by BIRC assessment
Measured: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Clinical Benefit Rate (CBR)
Per investigator/BIRC assessment including CR (Complete Response), PR (Partial Response), and SD (Stable Disease) lasting more than 6 months;
Measured: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Disease Control Rate (DCR)
Defined as the percentage of cases with best response (PR or CR or stable disease (SD) after treatment in evaluable cases.
Measured: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Progression-Free Survival (PFS)
Defined as the time from participant enrollment to disease progression or death (whichever occurs first). Participants without an event (no progression or death) were censored at the date of last tumor evaluation.
Measured: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Time To Progression (TTP)
Defined as the time from participant enrollment to disease progression, and participants without events (without progression or death) were censored at the date of tumor evaluation.
Measured: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Time To Response (TTR)
Defined as the time from participant enrollment to the first observation of tumor response among participants with an objective response
Measured: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Duration Of Response (DOR)
Defined as the time from first observation of tumor response to tumor progression or death from any cause in participants with an objective response, (whichever occurs first).
Measured: Through study completion, an average of 2 years
Phase II Other safety endpoints: - The type and frequency of adverse events (AE) - Treatment-Emergent Serious Adverse Events (SAE) - The frequency and causes of death events - Laboratory safety test results; - Changes in vital signs;
Measured: Through study completion, an average of 2 years
Phase II Dose intensity
Dose intensity, planned dose intensity and relative dose intensity will be measured.
Dose Intensity (mg/day) = Actual Cumulative Dose (mg)/ Total Duration of Exposure (Days).
Plan Dose Intensity (mg/day) = Plan Cumulative Dose (mg) / Total Duration of Exposure (Days).
Relative Dose Intensity (%) = Dose Intensity / Plan Dose Intensity.
Measured: Through study completion, an average of 2 years
Phase II Ctrough After Multiple Dose of FCN-159
Measured: Pre-dose (0 hour), 0.5~1, 1.5~3, 4~6, 24 hours on Day 1 of Cycles 2, Pre-dose (0 hour) on Cycle 5 and every 4 cycles thereafter (assessed up to 104 weeks) (1 cycle = 28 days)
Phase II Clinical outcome variables: Changes in pain intensity.
Each participant will undergo standardized assessments to evaluate changes in pain intensity, using validated pain scales and questionnaires. The incidence of patients experiencing a decrease in pain intensity, as well as the reduction in pain intensity scores, will be evaluated.
Measured: Through study completion, an average of 2 years
Phase II Other clinical outcome variables: Changes in COA variables other than pain intensity.
Measured: Through study completion, an average of 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2021
- Study start26 Mar 2021
- Primary completion28 Feb 2025 (estimated)
- Study completion30 Mar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Neurofibromatosis 1Studies of the same condition, open and under way.
- More trials run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 160 participants (estimated)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04954001
- Sponsor's protocol ID
- FCN-159-002
- Status last verified
- Oct 2024
- First submitted
- 17 Jun 2021
- Last updated
- 12 Nov 2024
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Hu X, Wu Z, Wang J, Li W, Zeng K, Li Y, Tao J, Guan Z, Kang Z, Xu Z, Ma Y, Yang L, Wang X, Han P, Lin H, Diao L, Tan Y, Zhong W, Hui AM, Li C, Lin X. Phase 1 Study of Luvometinib Use in Pediatric Patients with Neurofibromatosis Type 1-Related Unresectable Plexiform Neurofibromas. Target Oncol. 2025 Nov;20(6):991-1001. doi: 10.1007/s11523-025-01176-y. Epub 2025 Oct 26.
PubMed 41139346Hu X, Li W, Zeng K, Xu Z, Li C, Kang Z, Li S, Huang X, Han P, Lin H, Hui AM, Tan Y, Diao L, Li B, Wang X, Wu Z, Lin X. Phase 1 dose-escalation study to evaluate the safety, tolerability, pharmacokinetics, and anti-tumor activity of FCN-159 in adults with neurofibromatosis type 1-related unresectable plexiform neurofibromas. BMC Med. 2023 Jul 3;21(1):230. doi: 10.1186/s12916-023-02927-2.
PubMed 37400844
Study at a glance
From the registry record, last updated by the sponsor on 12 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-label, Single-arm Phase I Dose-escalation and Phase II Dose-expansion Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
- ClinicalTrials.gov ID
- NCT04954001
- Acronym / sponsor ID
- FCN-159-002
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 160 participants (estimated)
- Lead sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.Industry (pharma, biotech, devices)
- Study start
- 26 Mar 2021 (actual)
- Primary completion
- 28 Feb 2025 (estimated)
- Study completion
- 30 Mar 2025 (estimated)
- First posted
- 8 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
FCN-159 is a highly active MEK1/2 inhibitor that was designed, synthesized and screened on the basis of the structure of trametinib. FCN-159 is an orally available and highly potent selective inhibitor of MEK1/2, which is expected to be a targeted therapy for the treatment of advanced solid tumors and neurofibromatosis type 1.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: FCN-159 |
Who may qualify
- Ages
- 2 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (22 listed)
- 1.Cohort 1: 16-70 years of age (inclusive) with a body weight of ≥ 94 lbs or 42.5 kg.
- Cohort 2: 2-15 years of age (inclusive) and able to swallow whole tablet. 2.Participants must be diagnosed with NF1-related plexiform neurofibromas (PN) and symptomatic with requirement of systematic therapy per investigator's judgment. A PN is defined as a neurofibroma that has grown along the length of a nerve and may involve multiple fascicles and branches. A spinal PN involves two or more levels with connection between the levels or extending laterally along the nerve. Diagnosis of neurofibromatosis type 1 (NF1) is based on meeting at least 1 of the following 2 diagnostic criteria:
- Genetic testing confirmation: i.e., positive for NF1 germline mutation per CLIA-certified laboratory (or equivalent) testing. Note: NF1 germline mutation positive must either be confirmed by the FCN-159-002 central laboratory or have documentation of NF1 mutation issued by a CLIA-certified laboratory (or equivalent) - OR -
- Clinical and imaging confirmation: Meets at least 2 of the following 7 NF1 diagnostic criteria according to the clinical NIH consensus criteria:
- <!-- -->
Key exclusion criteria (29 listed)
- Participants who meet any of the following conditions shall not be included in this clinical study:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FCN-159FCN-159 is administered orally in once daily schedule for 28 days a cycle. | Drug | Single Arm |
Primary outcome
Phase I: Safety: Dose-limiting toxicity (DLT) incidence rate.
Time frame: 28 days after the dose of FCN-159 for DLT
Phase I: Safety: MTD and RP2D.
Time frame: Approximately 6-9 months for MTD and RP2D (phase I duration)
Phase II: Objective response rate (ORR) by investigator assessment
Time frame: Through study completion, an average of 2 years
18 secondary outcomes
Phase I Other safety: - The type and frequency of adverse events (AE) - Treatment-Emergent Serious Adverse Events (SAE) - The frequency and causes of death events - Laboratory safety test results - Changes in vital signs
Time frame: Through study completion, an average of 2 years
Phase I Efficacy: objective response rate (ORR) by investigator /BIRC assessment, clinical benefit rate (CBR) per investigator/BIRC assessment including CR, PR, and SD lasting more than 6 months;
Time frame: Through study completion, an average of 2 years
Phase I: Maximum Observed Plasma Concentration (Cmax) After Single Dose and Multiple Dose of FCN-159
Time frame: Pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 10, 24 hours post-dose on Cycle 1 Day 1 (1 cycle = 28 days) for single dose, Pre-dose (0 hour), 0.5, 1, 2, 3, 4, 6, 10, 24 hours post-dose on Cycle 1 Day 28 for multiple dose
Phase I: AUC From Time Zero to Last Measurable Concentration (AUClast) After Single Dose and Multiple Dose of FCN-159
Time frame: Pre-dose (0 hour), 0.5~1, 1.5~3, 4~6, 24 hours on Day 1 of Cycles 2, Pre-dose (0 hour) on Cycle 5 and every 4 cycles thereafter (assessed up to 104 weeks) (1 cycle = 28 days)
Phase I PD marker: ERK phosphorylation inhibition in peripheral blood mononuclear cells (PBMCs)
Time frame: During Cycle 1 (cycle is 28 days): Day 1, Day 8 and Day 28
Phase I Changes in NF1-related symptoms.
Time frame: Through study completion, an average of 2 years]
Phase II: Critical secondary endpoint: Objective response rate (ORR) by BIRC assessment
Time frame: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Clinical Benefit Rate (CBR)
Time frame: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Disease Control Rate (DCR)
Time frame: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Progression-Free Survival (PFS)
Time frame: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Time To Progression (TTP)
Time frame: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Time To Response (TTR)
Time frame: Through study completion, an average of 2 years
Phase II: Other efficacy endpoints: Duration Of Response (DOR)
Time frame: Through study completion, an average of 2 years
Phase II Other safety endpoints: - The type and frequency of adverse events (AE) - Treatment-Emergent Serious Adverse Events (SAE) - The frequency and causes of death events - Laboratory safety test results; - Changes in vital signs;
Time frame: Through study completion, an average of 2 years
Phase II Dose intensity
Time frame: Through study completion, an average of 2 years
Phase II Ctrough After Multiple Dose of FCN-159
Time frame: Pre-dose (0 hour), 0.5~1, 1.5~3, 4~6, 24 hours on Day 1 of Cycles 2, Pre-dose (0 hour) on Cycle 5 and every 4 cycles thereafter (assessed up to 104 weeks) (1 cycle = 28 days)
Phase II Clinical outcome variables: Changes in pain intensity.
Time frame: Through study completion, an average of 2 years
Phase II Other clinical outcome variables: Changes in COA variables other than pain intensity.
Time frame: Through study completion, an average of 2 years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Nov 2024.
- Derived from this study: PubMed 41139346Hu X, Wu Z, Wang J, Li W, Zeng K, Li Y, Tao J, Guan Z, Kang Z, Xu Z, Ma Y, Yang L, Wang X, Han P, Lin H, Diao L, Tan Y, Zhong W, Hui AM, Li C, Lin X. Phase 1 Study of Luvometinib Use in Pediatric Patients with Neurofibromatosis Type 1-Related Unresectable Plexiform Neurofibromas. Target Oncol. 2025 Nov;20(6):991-1001. doi: 10.1007/s11523-025-01176-y. Epub 2025 Oct 26.
- Derived from this study: PubMed 37400844Hu X, Li W, Zeng K, Xu Z, Li C, Kang Z, Li S, Huang X, Han P, Lin H, Hui AM, Tan Y, Diao L, Li B, Wang X, Wu Z, Lin X. Phase 1 dose-escalation study to evaluate the safety, tolerability, pharmacokinetics, and anti-tumor activity of FCN-159 in adults with neurofibromatosis type 1-related unresectable plexiform neurofibromas. BMC Med. 2023 Jul 3;21(1):230. doi: 10.1186/s12916-023-02927-2.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
14 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FCN-159 in people with Neurofibromatosis 1 and Plexiform Neurofibroma, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 14 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 22 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 12 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in China, United States, Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase I: Safety: Dose-limiting toxicity (DLT) incidence rate. (time frame: 28 days after the dose of FCN-159 for DLT). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04954001, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Nov 2024.