Actively Recruiting
Study to Evaluate the Effects and Safety of FCN-159 in Children with Refractory or Recurrent Langerhans Cell Histiocytosis
Led by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. · Updated on 2025-01-24
56
Participants Needed
11
Research Sites
189 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a rare disease Phase 2 clinical trial to study the effects of FCN-159 monotherapy in pediatric patients aged 2 to 16 years with refractory or recurrent Langerhans cell histiocytosis (LCH). This open-label, single-arm, multi-center study aims to evaluate the drug's efficacy, safety, and how the body processes it (pharmacokinetics). Approximately 56 children will participate, providing valuable data on this rare condition and treatment. Participants will receive FCN-159 orally once daily at a dose of 5 mg/m², not exceeding 8 mg, taken continuously in 28-day treatment cycles. Treatment will continue until the disease worsens, unacceptable side effects occur, consent is withdrawn, death, or other protocol-specified reasons. The study includes a screening period, treatment period, and follow-up period, with tumor assessments done regularly based on PET response criteria and LCH guidelines. During the study, patients will be closely monitored through evaluations by investigators and an independent review committee. Researchers will assess tumor response, safety, and pharmacokinetics for up to 24 months. Assessments include imaging tests, laboratory tests, and clinical evaluations to track disease progression, treatment effects, and adverse events. The study aims to provide comprehensive information on FCN-159's impact in this pediatric population.
CONDITIONS
Official 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 2 to 16 years inclusive
- Histologically confirmed diagnosis of Langerhans cell histiocytosis (LCH) by central laboratory
- Central or local laboratory biomarker testing for specified genes if sufficient tissue or blood samples are available
- Prior treatment with at least first-line systemic therapy including vinblastine or equivalent for at least 2 weeks, or inability to tolerate chemotherapy due to severe toxicity
- Refractory or relapsed LCH defined by specific criteria including treatment failure, disease reactivation, persistent mutated gene positivity, CNS involvement, or bone marrow/HLH involvement
- Presence of evaluable lesions based on PET response criteria
- Recovery from acute toxic effects of prior anti-tumor therapy with toxicities ≤ grade 1 (except alopecia and ototoxicity)
- Expected survival of at least 3 months
- Lansky (≤15 years) or Karnofsky (≥16 years) performance status score ≥ 50%
- Ability and willingness to provide informed consent
- Negative serum pregnancy test within 7 days before treatment for females of childbearing potential
- Agreement to use effective contraception during treatment and for 90 days after last dose (dual barrier methods for females; males to refrain from sperm donation)
- Adequate bone marrow function with specified blood count thresholds, or inclusion based on investigator judgment if low counts due to underlying disease
- Adequate liver and kidney function within specified laboratory limits, with possible inclusion based on investigator judgment
- Coagulation parameters (INR and APTT) ≤ 1.5 times upper limit of normal
You will not qualify if you...
- Prior chemotherapy, targeted therapy, immunotherapy, biologic therapy, or herbal anti-tumor therapy for LCH within 4 weeks or less than 5 half-lives before study drug start
- Use of strong CYP3A4, CYP2C8, or CYP2C9 inhibitors or inducers within 14 days before study drug start (except topical skin application)
- Use of growth factors affecting platelet or white blood cell function within 7 days before study drug start
- Radiotherapy or major surgery within 4 weeks before study drug start
- Participation in other interventional clinical trials within 4 weeks before study drug start
- Prior MEK 1/2 inhibitor treatment exceeding 2 weeks
- Use of anticoagulants within 7 days before study drug start in patients with brain tumors
- Prednisone or equivalent corticosteroid use below specified dose within 1 month prior, with required discontinuation 14 days before study drug start
- History or presence of other malignant tumors except certain cured skin or breast cancers
- Uncontrolled hypertension (≥ 95th percentile for age, height, and sex)
- Dysphagia, active gastrointestinal disease, malabsorption syndrome, or conditions affecting drug absorption
- History or current retinal vein obstruction, retinal pigment epithelial detachment, glaucoma, or significant eye exam abnormalities
- Interstitial pneumonia or significant radiation pneumonitis except due to primary disease
- Specified cardiac conditions including QTcF >470 ms, NYHA class ≥2 heart failure, significant arrhythmias, coronary heart disease, cardiomyopathy, severe valvular disease, or LVEF <50%
- Active bacterial, fungal, or viral infections including active hepatitis B or C, or HIV
- Known allergies to study drug, MEK1/2 inhibitors, or excipients
- Genetic mutations MAP2K1 exon 3 deletions or deletion-insertion mutations in tumor tissue
- Any other clinically significant conditions impeding study participation or safety compliance as determined by investigator
AI-Screening
AI-Powered Screening
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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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