Actively Recruiting
A Multi-center, Open-label, Single Arm Phase 2 Study to Evaluate the Efficacy, Safety, and Pharmacokinetic Characteristics of FCN-159 in Pediatric Patients With Refractory/Recurrent Langerhans Cell Histiocytosis
Led by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. · Updated on 2025-01-24
56
Participants Needed
11
Research Sites
104 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating FCN-159 monotherapy in pediatric patients aged 2 to 16 years with refractory or recurrent Langerhans cell histiocytosis (LCH), a rare disease. This Phase 2, single-arm, open-label study aims to assess the drug's effectiveness, safety, and how it behaves in the body. About 56 children will participate across multiple centers. Patients must have confirmed LCH and have received at least one prior systemic treatment or be unable to tolerate chemotherapy due to severe toxicity. Participants will receive FCN-159 tablets orally once daily at a dose of 5 mg per square meter of body surface area, not exceeding 8 mg, in 28-day cycles. Treatment continues until disease progression, intolerable side effects, withdrawal, death, or other protocol-defined reasons. Tumor assessments will be done regularly using PET response criteria and LCH treatment guidelines to evaluate the disease's status by both investigators and an independent review committee. Throughout the study, patients will undergo tumor response evaluations, safety monitoring, and pharmacokinetic testing for up to 24 months. The primary outcome measured is the objective response rate based on PET criteria. Secondary outcomes include disease control, clinical benefit rates, survival rates, and treatment-emergent side effects. Follow-up continues through disease progression, death, or withdrawal, with comprehensive assessments to track treatment effects and patient health.
CONDITIONS
Brief Title
To Evaluate the Efficacy, Safety, and PK Characteristics of FCN-159 in Pediatric Patients With Refractory/Recurrent LCH
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 2 and 16 years inclusive
- Histologically confirmed Langerhans cell histiocytosis diagnosed by central laboratory
- Available tumor tissue or blood samples for biomarker testing or local gene testing if tissue unavailable
- Received at least prior first-line systemic treatment including vinblastine or alternatives, or unable to tolerate chemotherapy due to severe toxicity
- Refractory or relapsed LCH defined by failure of prior treatment, disease reactivation, positive gene mutation tests, lack of CNS lesion regression, or bone marrow/HLH involvement
- Presence of evaluable lesions based on PET response criteria
- Recovery from prior anti-tumor therapy toxic effects to grade 1 or less except alopecia and ototoxicity
- Expected survival of at least 3 months
- Performance status scores of 50% or higher (Lansky or Karnofsky scale)
- Written informed consent obtained from patient or legal guardian
- Negative pregnancy test for females of childbearing potential within 7 days before treatment
- Agreement to use effective contraception during treatment and 90 days after last dose
- Adequate bone marrow, liver, renal function, and coagulation parameters as specified
You will not qualify if you...
- Prior chemotherapy, targeted, immunotherapy, biologic, or herbal anti-tumor treatment for LCH within 4 weeks or less than 5 half-lives before starting study drug
- Use of strong CYP3A4, CYP2C8, CYP2C9 inhibitors or inducers within 14 days before study drug start (except topical skin application)
- Use of growth factors affecting blood counts within 7 days before study drug start
- Radiotherapy or major surgery within 4 weeks before study drug start
- Participation in other interventional trials within 4 weeks before study drug start
- Use of MEK 1/2 inhibitors unless treatment lasted 2 weeks or less
- Use of anticoagulants within 7 days before study drug for brain tumor patients
- Prednisone treatment below 0.5 mg/kg/day allowed one month before enrollment but must stop 14 days before study
- History or current other malignant tumors except certain cured skin or breast conditions
- Uncontrolled hypertension
- Conditions affecting drug absorption like dysphagia or malabsorption
- Significant eye disorders including retinal or glaucoma issues
- Interstitial pneumonia except due to primary disease
- Cardiac conditions including prolonged QTcF, heart failure class 2 or above, arrhythmias, coronary disease, or low heart function
- Active infections including hepatitis B or C, or HIV
- Known allergies to study drug or MEK inhibitors
- Genetic mutations in tumor tissue indicating MAP2K1 exon 3 deletions
- Any other condition deemed by investigator to interfere with study participation or safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Repeated 28-day cycles until disease progression or discontinuation
Participants receive FCN-159 orally once daily in 28-day cycles until disease progression, intolerable toxicity, withdrawal, death, or other protocol-specified reasons.
Visits may occur regularly during treatment cycles for assessments and dosing
Duration - Up to 24 months
Participants are monitored after treatment ends for safety and efficacy outcomes up to 24 months.
Visits scheduled as needed for outcome assessments
Trial Site Locations
Total: 11 locations
1
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality, China
Actively Recruiting
2
Children's Hospital Affiliated to the Capital Institute of Pediatrics
Beijing, Beijing Municipality, China
Not Yet Recruiting
3
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, China
Actively Recruiting
4
Sun Yat-Sen Memorial Hpsipital,Sun Yat-Sen Unniversity
Guangzhou, Guangdong, China
Not Yet Recruiting
5
The First Affiliated Hospital,Sun Yat-sen University
Guangzhou, Guangdong, China
Not Yet Recruiting
6
Shenzhen Children's Hospital
Shenzhen, Guangdong, China
Not Yet Recruiting
7
Henan Children's Hospital Zhengzhou Children's Hospital
Zhengzhou, Henan, China
Not Yet Recruiting
8
Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei, China
Not Yet Recruiting
9
West China Second University Hospital,Sihuan University/West China women's and Children's Hospital
Chengdu, Sichuan, China
Not Yet Recruiting
10
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
Not Yet Recruiting
11
Children's Hospital of Soochow University
Suzhou, China
Not Yet Recruiting
Research Team
R
Rui Zhang, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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