All > Neurofibromatosis Type 1 > Neurofibromatosis

FCN-159 for Neurofibromatosis

Research Site, Madrid, Spain
Neurofibromatosis+2 More ConditionsFCN-159 - Drug
Eligibility
2 - 70
All Sexes

Study Summary

This trial is testing a new drug, FCN-159, to see if it can help treat patients with advanced solid tumors or neurofibromatosis type 1.

Eligible Conditions
  • Neurofibromatosis Type 1
  • Plexiform Neurofibromas

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 1 & 2

Similar Trials

1
Curcumin
1
IK-930
1
Experimental Monotherapy Dose Escalation

Study Objectives

5 Primary · 19 Secondary · Reporting Duration: Through study completion, an average of 2 years

Day 28
Phase I: Safety: Dose-limiting toxicity (DLT) incidence rate.
Month 9
Primary objectives of Phase I
Month 9
Phase I: Safety: MTD and RP2D.
Day 28
Phase I PD marker: ERK phosphorylation inhibition in peripheral blood mononuclear cells (PBMCs)
Day 28
Phase I: Maximum Observed Plasma Concentration (Cmax) After Single Dose and Multiple Dose of FCN-159
Week 104
Phase I: AUC From Time Zero to Last Measurable Concentration (AUClast) After Single Dose and Multiple Dose of FCN-159
Phase II Ctrough After Multiple Dose of FCN-159
Year 2
Phase I Changes in NF1-related symptoms.
Phase I Clinical outcome variables: patient- and observer-reported outcomes and functional measures
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;
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
Phase II Changes in NF1-related symptoms
Phase II Clinical outcome variables: patient- and observer-reported outcomes and functional measures
Phase II Dose intensity
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;
Phase II: Objective response rate (ORR) by BIRC assessment
Phase II: Other efficacy endpoints: Clinical Benefit Rate (CBR)
Phase II: Other efficacy endpoints: Disease Control Rate (DCR)
Phase II: Other efficacy endpoints: Duration Of Response (DOR)
Phase II: Other efficacy endpoints: Objective Response Rate (ORR)
Phase II: Other efficacy endpoints: Progression-Free Survival (PFS)
Phase II: Other efficacy endpoints: Time To Progression (TTP)
Phase II: Other efficacy endpoints: Time To Response (TTR)
Primary objective of Phase II

Trial Safety

Phase-Based Safety

1 of 3

Similar Trials

1
Curcumin
1
IK-930
1
Experimental Monotherapy Dose Escalation

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.

Trial Design

1 Treatment Group

Single Arm
1 of 1

Experimental Treatment

160 Total Participants · 1 Treatment Group

Primary Treatment: FCN-159 · No Placebo Group · Phase 1 & 2

Single Arm
Drug
Experimental Group · 1 Intervention: FCN-159 · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: through study completion, an average of 2 years

Who is running the clinical trial?

Shanghai Fosun Pharmaceutical Development Co, Ltd.Lead Sponsor
4 Previous Clinical Trials
681 Total Patients Enrolled
Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.Lead Sponsor
12 Previous Clinical Trials
4,665 Total Patients Enrolled

Eligibility Criteria

Age 2 - 70 · All Participants · 2 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You agree to limit your time in the sun and apply sunscreen if you know you'll be exposed to sunlight.
These are the basic requirements that all participants need to meet to be considered for Phase I and II clinical trials.
References

Frequently Asked Questions

Are there numerous centers offering this research study in Canada?

"This clinical trial is running out of 4 sites across the country, including Detroit, Gainesville and Charleston. To reduce time spent traveling for patients enrolled in this study, it may be beneficial to choose a clinic closest to your home." - Anonymous Online Contributor

Unverified Answer

To what extent is the participant pool for this clinical trial expanding?

"Affirmative. Per the information housed on clinicaltrials.gov, this medical trial is in search of volunteer participants and was first launched on March 26th 2021. The study needs to find 160 patients from 4 different locations before it can be completed; its records were most recently updated on August 24th 2022." - Anonymous Online Contributor

Unverified Answer

Am I eligible to partake in the current investigation?

"This medical study is seeking 160 individuals between the ages of 2 and 70 diagnosed with Watson Syndrome. To qualify, applicants must meet one or more of the following criteria: have a body weight above 94 lbs/42.5 kg., possess axillary freckling or inguinal region freckles; be genetically confirmed to have an NF1 germline mutation (either from testing at the central laboratory for FCN-159-002 or documentation from a CLIA-approved centre); display six cafe-au-lait macules bigger than 0.5 cm pre-puberty, 1.5 cm post puberty; two neurof" - Anonymous Online Contributor

Unverified Answer

Is there an age requirement to participate in this medical study?

"As per the requirements of this trial, potential enrollees must be at least 2 years old and no older than 70." - Anonymous Online Contributor

Unverified Answer

Are there any opportunities currently available to volunteers for this research project?

"Clinicaltrials.gov affirms that this research is still actively looking for subjects to enrol, having been first posted on March 26th 2021 and most recently updated August 24th 2022." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Unbiased ResultsWe believe in providing patients with all the options.
Your Data Stays Your DataWe only share your information with the clinical trials you're trying to access.
Verified Trials OnlyAll of our trials are run by licensed doctors, researchers, and healthcare companies.
Back to top