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Neurofibromatosis 1Phase 3NCT05913037

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

A Phase 3 study testing Test group (Group A): FCN-159 8 mg, orally, once daily; in people with Neurofibromatosis 1 and Plexiform Neurofibroma, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 15 sites in China.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

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

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Neurofibromatosis 1, Plexiform Neurofibroma, NF1
What is being tested?
Test group (Group A): FCN-159 8 mg, orally, once daily;
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
167 participants
Where
15 sitesChina
Study dates
20 Jun 2023to 30 Jun 2026
Phase
Phase 3
Are there results?
Not yet: the study is under way

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 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • ≥ 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.

Who cannot join

  • 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:
Show all 12Show fewer
  • 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.

Trial locations and contacts

15 sites in 1 country.

15 sites in 1 country

China15 sites

  • Cancer Hospital Chinese Academy of Medical Sciences

    Beijing, Beijing Municipality

  • Chinese Academy of Medical Sciences & Peking Union Medical College

    Beijing, Beijing Municipality

  • Plastic Surgery Hospital,Chinese Academy of Medical Sciences

    Beijing, Beijing Municipality

  • West China Hospital，Sichuan University

    Chengdu, Sichuan

  • Nanfang Hospital, Southern Medical University

    Guangzhou, Guangdong

  • Sun Yat-sen University Cancer Center

    Guangzhou, Guangdong

  • Hangzhou First People's Hospital

    Hangzhou, Zhejiang

  • Zhejiang Provincial People'S Hospital

    Hanzhou, Zhejiang

  • Fudan University Shanghai Cancer center

    Shanghai, Shanghai Municipality

  • The First Hospial of China Medical University

    Shenyang, Liaoning

  • Peking University Shenzhen Hospital

    Shenzhen, Guangdong

  • The Fourth Hospital of Hebei Medical University

    Shijiazhuang, Hebei

  • The Second Hospital of Hebei Medical University

    Shijiazhuang, Hebei

  • Renmin Hospital of Wuhan University

    Wuhan, Hubei

  • TongJi Hospital，TongJi Medical College Huazhong University of Science and Technology

    Wuhan, Hubei

Other trials in China

About this study

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

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

Read the 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.

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

  • 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.

Study groups in NCT05913037
Study groupRoleWhat they receive
FCN-159ExperimentalExperimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 8 mg, orally, once daily Method of administration: Oral
Receives: Drug: Test group (Group A): FCN-159 8 mg, orally, once daily;
placeboPlacebo comparatorExperimental: placebo Dosage form:tablet Specification: 1mg，4mg Dose: placebo 8 mg, orally, once daily Method of administration: Oral
Receives: Drug: Control group (Group B): Placebo, orally, once daily;

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Through study completion, an average of 2 years

Secondary outcomes (9)

  1. 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.

    Measured: Through study completion, an average of 2 years

  2. 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.

    Measured: Through study completion, an average of 2 years

  3. 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.

    Measured: Through study completion, an average of 2 years

  4. 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.

    Measured: Through study completion, an average of 2 years

  5. 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.

    Measured: Through study completion, an average of 2 years

  6. 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.

    Measured: Through study completion, an average of 2 years

  7. 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.

    Measured: Through study completion, an average of 2 years

  8. 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.

    Measured: Through study completion, an average of 2 years

  9. 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.

    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.

  1. First posted22 Jun 2023
  2. Study start20 Jun 2023
  3. Primary completion19 Aug 2025 (estimated)
  4. Study completion30 Jun 2026 (estimated)

Resources

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 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
167 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05913037
Sponsor's protocol ID
FCN-159-007
Status last verified
Feb 2025
First submitted
6 Jun 2023
Last updated
6 Mar 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Test group (Group A): FCN-159 8 mg, orally, once daily; in people with Neurofibromatosis 1 and Plexiform Neurofibroma, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 15 sites in China.

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 5 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 6 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 15 sites in China. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

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?

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). 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 NCT05913037.

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 6 Mar 2025.

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