Neurofibromatosis 1Phase 1/2NCT07024394
Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1
A Phase 1/2 study testing Luvometinib Tablets in people with Neurofibromatosis 1 and Plexiform Neurofibroma, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 1 site in China.
Trial summary
FCN-159 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study NCT04954001.This study is a 5-year long-term follow-up of the FCN-159-002 study, involving all… Read more about this study
- Who is it for?
- Ages 2 to 20 years
- Conditions the study is for
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- What is being tested?
- Luvometinib Tablets
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 65 participants
- Where
- 1 siteChina
- Study dates
- 12 Jun 2025to 23 Sep 2029
- Phase
- Phase 1/2
- 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 2 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients from the pediatric cohort of the FCN-159-002 study. Willing to participate in the long-term follow-up study and capable of understanding and voluntarily signing the informed consent form.
Who cannot join
- Any clinically significant condition that, in the investigator's judgment, may interfere with study participation or compliance with safety requirements.
- Patients unable to comply with visit-related requirements.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Shanghai Ninth People's Hospital
Shanghai, Shanghai Municipality, 200011
Xiaojie Hu, +86 13701997136, hxjie2002@hotmail.com
About this study
Official title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Ruvometinib Tablets (FCN-159 Tablets) in Pediatric Participants With Neurofibromatosis Type 1
FCN-159 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study NCT04954001.This study is a 5-year long-term follow-up of the FCN-159-002 study, involving all enrolled patients to further assess safety, growth and development effects, and treatment efficacy.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Being tested
- Luvometinib Tablets
- 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.
Luvometinib TabletsDrug
Also called FCN-159
5mg/m² (Maximum dose does not exceed 8mg, the recommended oral dose for adults), orally, once daily, until disease progression or study completion, whichever occurs first
| Study group | Role | What they receive |
|---|---|---|
| FCN-159 | Experimental | Study Drug: FCN-159 Dosing Regimen: FCN-159 to be administered once daily until disease progression or study completion, whichever occurs first Receives: Drug: Luvometinib Tablets |
What is being measured
The primary outcome is the main question the study is designed to answer.
long term safty
Adverse events during treatment of FCN-159 will be assessed by the investigators according to CTCAE 5.0.
Measured: Through study completion, about 5 years
Secondary outcomes (6)
Height in centimeters
Height will be measured in centimeters, with subsequent plotting of height growth curves and calculation of growth velocity.
Measured: Through study completion, about 5 years
Weight in kilograms
Weight will be measured in kilograms, with subsequent plotting of weight growth curves.
Measured: Through study completion, about 5 years
Tanner stage
Tanner stage will be examined, and the mean age for each Tanner stage will be calculated.
Measured: Through study completion, about 5 years
Progression-Free Survival (PFS)
PFS assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator
Measured: Through study completion, about 5 years
Objective response rate (ORR)
ORR assessed per REiNS (Response Evaluation in Neurofibromatosis and Schwannomatosis) criteria by inverstigator.
Measured: Through study completion, about 5 years
Changes in pain intensity
Changes in pain intensity score relative to baseline will be assessed according to 11-point Numerical Rating Scale (NRS-11).NRS-11 quantifies pain intensity on a scale from 0 to 10, 0 represents "No pain" ,10 represents "The worst pain imaginable".
Measured: Through study completion, about 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2025
- Study start12 Jun 2025 (estimated)
- Primary completion23 Sep 2029 (estimated)
- Study completion23 Sep 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- 65 participants (estimated)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07024394
- Sponsor's protocol ID
- FCN-159-002-LT
- Status last verified
- Jun 2025
- First submitted
- 3 Jun 2025
- Last updated
- 17 Jun 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Ruvometinib Tablets (FCN-159 Tablets) in Pediatric Participants With Neurofibromatosis Type 1
- ClinicalTrials.gov ID
- NCT07024394
- Acronym / sponsor ID
- FCN-159-002-LT
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 65 participants (estimated)
- Lead sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.Industry (pharma, biotech, devices)
- Study start
- 12 Jun 2025 (estimated)
- Primary completion
- 23 Sep 2029 (estimated)
- Study completion
- 23 Sep 2029 (estimated)
- First posted
- 17 Jun 2025 (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 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study NCT04954001.This study is a 5-year long-term follow-up of the FCN-159-002 study, involving all enrolled patients to further assess safety, growth and development effects, and treatment efficacy.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FCN-159 | Experimental | Drug: Luvometinib TabletsStudy Drug: FCN-159 Dosing Regimen: FCN-159 to be administered once daily until disease progression or study completion, whichever occurs first |
Who may qualify
- Ages
- 2 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (1 listed)
- Patients from the pediatric cohort of the FCN-159-002 study. Willing to participate in the long-term follow-up study and capable of understanding and voluntarily signing the informed consent form.
Key exclusion criteria (2 listed)
- Any clinically significant condition that, in the investigator's judgment, may interfere with study participation or compliance with safety requirements.
- Patients unable to comply with visit-related requirements.
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 |
|---|---|---|
| Luvometinib TabletsAlso: FCN-1595mg/m² (Maximum dose does not exceed 8mg, the recommended oral dose for adults), orally, once daily, until disease progression or study completion, whichever occurs first | Drug | FCN-159 |
Primary outcome
long term safty
Time frame: Through study completion, about 5 years
Adverse events during treatment of FCN-159 will be assessed by the investigators according to CTCAE 5.0.
6 secondary outcomes
Height in centimeters
Time frame: Through study completion, about 5 years
Weight in kilograms
Time frame: Through study completion, about 5 years
Tanner stage
Time frame: Through study completion, about 5 years
Progression-Free Survival (PFS)
Time frame: Through study completion, about 5 years
Objective response rate (ORR)
Time frame: Through study completion, about 5 years
Changes in pain intensity
Time frame: Through study completion, about 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jun 2025.
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Luvometinib Tablets in people with Neurofibromatosis 1 and Plexiform Neurofibroma, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 1 site 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 1 inclusion and 2 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: opening soon, as of the sponsor's last update on 17 Jun 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. 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?
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?
Contact Xiaojie Hu (+86 13701997136, hxjie2002@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
long term safty (time frame: Through study completion, about 5 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Jun 2025.