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Tests treatment safety and results for Langerhans Cell Histiocytosis
Official title To Evaluate the Efficacy, Safety, and PK Characteristics of FCN-159 in Pediatric Patients With Refractory/Recurrent LCH
ClinicalTrials.gov ID: NCT05997602
What this study is testing
What is FCN-159?
FCN-159 is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for langerhans cell histiocytosis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is a rare disease, single-arm, open-label,multi-center, non-randomized Phase 2 clinical study to evaluate the efficacy, safety, and pharmacokinetic characteristics of FCN-159 monotherapy in pediatric patients with refractory/recurrent Langerhans cell histiocytosis (LCH).
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 16
You may be able to join if
- Age 2-16 (inclusive)
- Patients with histologically confirmed Langerhans cell histiocytosis (LCH) diagnosed by the central laboratory.
- If sufficient tumor tissue samples and peripheral blood samples are available, central laboratory biomarker testing is required as follows: including...
- Patients who have received at least prior first-line systemic treatment, defined as treatment including vinblastine (VBL) and glucocorticoids for at...
- Refractory/relapsed LCH is defined as the presence of one of the following:
You likely can't join if
- Patients who have received any of the following prior treatments:
- Chemotherapy, targeted therapy, immunotherapy, biologic therapy, or herbal anti-tumor therapy for LCH within 4 weeks or \< 5 half-lives (whichever is...
- Strong CYP3A4, CYP2C8, and CYP2C9 inhibitors or inducers within 14 days before the start of the study drug, except for topical skin application.
- Gowth factors that promote platelet or white blood cell count or function within 7 days before the start of the study drug.
- Radiotherapy or major surgical treatment (including craniotomy, thoracotomy, laparotomy, open bone or joint surgery, etc.) within 4 weeks before the...
- Participated in other treatment clinical trials within 4 weeks before the start of the study drug.
See the full eligibility criteria
Who can join
- Age 2-16 (inclusive)
- Patients with histologically confirmed Langerhans cell histiocytosis (LCH) diagnosed by the central laboratory.
- If sufficient tumor tissue samples and peripheral blood samples are available, central laboratory biomarker testing is required as follows: including but not limited to ERBB3, BRAF, ARAF, HRAS, KRAS, NRAS, MEK (MAP2K1...
- Patients who have received at least prior first-line systemic treatment, defined as treatment including vinblastine (VBL) and glucocorticoids for at least 2 weeks. VBL can be substituted with vincristine (VCR) or...
- Refractory/relapsed LCH is defined as the presence of one of the following:
- Failure of prior treatment, i.e., no regression in risk organs after at least 2 weeks of systemic treatment, or overall evaluation of AD-progression or AD-mix;
- Initial response of the disease to first or second-line systemic treatment is NAD or AD-better or AD-stable, followed by disease reactivation after maintenance therapy for more than 3 months. Second-line treatment...
- Persistent mutated gene positive in plasma free DNA testing during prior treatment (confirmed by 2 consecutive tests) or retest positive after treatment discontinuation;
- Lack of regression in the affected central nervous system (including the pituitary gland) after treatment;
- Presence of bone marrow involvement and/or hemophagocytic lymphohistiocytosis (HLH);
- Presence of evaluable lesions based on PET response criteria (PRC).
- Patients who have to have recovered from all acute toxic effects of prior anti-tumor therapy, and all relevant toxicities must be ≤ grade 1 (except for alopecia and ototoxicity).
- Expected survival at least ≥ 3 months;
- Lansky (≤ 15 years old) and Karnofsky (≥ 16 years old) performance status scores should be ≥ 50%, as shown in Appendix 4.
- Patients or their legal guardians must be able to understand and willingly sign a written informed consent form.
- For women of childbearing potential, a serum human chorionic gonadotropin (HCG) pregnancy test must be negative within 7 days before starting treatment.
- For female patients of childbearing potential: Patients should agree to use effective contraception methods during the treatment period and for at least 90 days after the last dose of study treatment, using dual barrier...
- Adequate bone marrow function: Absolute neutrophil count ≥ 1.0×10\^9/L, hemoglobin ≥ 90g/L, and platelets ≥ 75×10\^9/L without the use of blood transfusions, blood products, or granulocyte colony-stimulating factors...
- Adequate hepatic and renal function: Serum total bilirubin ≤ 1.5 × the upper limit of normal (ULN), or ≤ 5× ULN for patients with Gilbert's syndrome or liver involvement; aspartate aminotransferase (AST), alanine...
- Coagulation: International normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤ 1.5 ULN.
What rules you out
- Patients who have received any of the following prior treatments:
- Chemotherapy, targeted therapy, immunotherapy, biologic therapy, or herbal anti-tumor therapy for LCH within 4 weeks or \< 5 half-lives (whichever is shorter)before the start of the study drug .
- Strong CYP3A4, CYP2C8, and CYP2C9 inhibitors or inducers within 14 days before the start of the study drug, except for topical skin application.
- Gowth factors that promote platelet or white blood cell count or function within 7 days before the start of the study drug.
- Radiotherapy or major surgical treatment (including craniotomy, thoracotomy, laparotomy, open bone or joint surgery, etc.) within 4 weeks before the start of the study drug.
- Participated in other treatment clinical trials within 4 weeks before the start of the study drug.
- MEK 1/2 inhibitors (those who have received this treatment for a short period of ≤ 2 weeks may be included).
- Anticoagulants within 7 days before the start of the study drug for patients with brain tumors (intracranial masses).
- Prednisone treatment \< 0.5mg/kg/day (or equivalent dose of other corticosteroids) is allowed within one month before enrollment, but must be discontinued 14 days before the start of the study drug. Patients with brain...
- Patients with a history of other malignant tumors or concurrent other malignant tumors (excluding cured non-melanoma skin basal cell carcinoma, ductal carcinoma in situ of the breast, or cervical carcinoma in situ).
- Uncontrolled hypertension (with medication treatment): Blood pressure (BP) greater than or equal to the 95th percentile for age, height, and sex, as described in Appendix 6.
- Patients with dysphagia, active gastrointestinal disease, malabsorption syndrome, or other conditions that may affect the absorption of the study drug.
- Prior or current history of retinal vein obstruction (RVO), retinal pigment epithelial detachment (RPED), glaucoma, and other clinically significant abnormal ophthalmologic examination results.
- Interstitial pneumonia, including clinically significant radiation pneumonitis. Except for interstitial pneumonia caused by pulmonary involvement of the primary disease.
- Patients will be excluded if their cardiac function or comorbidities meet any of the following criteria:
- During the screening period, 12-lead electrocardiogram (ECG) measurements will be taken three times at the study center with a mean value calculated using the QTcF formula provided by the instrument; patients with a...
- New York Heart Association (NYHA) Class 2 and above congestive heart failure as shown in Appendix 5.
- Clinically significant arrhythmias, including but not limited to complete left bundle branch block, and second-degree atrioventricular block.
- Known presence of clinically significant coronary heart disease, cardiomyopathy, or severe valvular disease.
- Echocardiography examination indicating left ventricular ejection fraction (LVEF) \< 50%.
- Patients with active bacterial, fungal, or viral infections, including active hepatitis B (defined as positive hepatitis B surface antigen and hepatitis B virus DNA \> 1000IU/ml or meeting the diagnostic criteria for...
- Patients with known allergies to the study drug, other MEK1/2 inhibitors, or their excipients.
- Patients with known tumor tissue genetic testing that indicates the presence of MAP2K1 exon 3 deletions (del) or deletion-insertion type (delins/indels) mutations.
- The investigator considers clinically significant cases that will impede participation in the study or prevent compliance with safety requirements.
The study team makes the final eligibility decision.
Where it's taking place
- Beijing, Beijing Municipality, China
- Chongqing, Chongqing Municipality, China
- Guangzhou, Guangdong, China
- Shenzhen, Guangdong, China
- Zhengzhou, Henan, China
- Wuhan, Hubei, China
- Chengdu, Sichuan, China
- Hangzhou, Zhejiang, China
- Suzhou, China
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 16 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Beijing, Beijing Municipality, China; Chongqing, Chongqing Municipality, China; Guangzhou, Guangdong, China; Shenzhen, Guangdong, China; Zhengzhou, Henan, China; Wuhan, Hubei, China and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.