Langerhans Cell HistiocytosisPhase 2NCT05997602
To Evaluate the Efficacy, Safety, and PK Characteristics of FCN-159 in Pediatric Patients With Refractory/Recurrent LCH
A Phase 2 study testing FCN-159 in people with Langerhans Cell Histiocytosis and LCH, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 11 sites in China.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 2 to 16 years
- Conditions the study is for
- Langerhans Cell Histiocytosis, LCH
- What is being tested?
- FCN-159
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 56 participants
- Where
- 11 sitesChina
- Study dates
- 28 Sep 2023to 14 May 2027
- Phase
- Phase 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 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to 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 and MAP2K2), and other MEK upstream genes.If inability to get tissue, the gene testing results from a local laboratory also can be accepted.
- 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 vindesine (VDS). Alternatively, patients may be unable to tolerate chemotherapy due to severe chemotherapy toxicity. Inability to tolerate chemotherapy is defined as one of the following: Severe liver impairment (liver enzyme elevation ≥ 5 × upper limit of normal (ULN) and bilirubin elevation ≥ 1.5 × ULN), severe neurotoxicity related to vinca alkaloids, chemotherapy-related intracranial hypertension, or grade 4 bone marrow depression with severe infection (sepsis, severe pneumonia, etc.) after chemotherapy.
- 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;
Show all 20Show fewer
- 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 includes cytarabine and/or cladribine.
- 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 contraception methods such as condoms, oral or injectable contraceptives, intra-uterine contraceptive devices, etc. Male patients should agree to refrain from donating sperm for at least 90 days after the last dose of study treatment.
- 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. Patients with hematocytopenia below these thresholds due to the underlying disease may be considered for inclusion based on the investigator's comprehensive judgment.
- 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 aminotransferase (ALT), and alkaline phosphatase (AKP) ≤ 2.5 × ULN, or ≤ 10 × ULN for patients with liver involvement; albumin ≥ 3g/dL; and creatinine clearance or isotopic glomerular filtration rate (GFR) ≥ 50ml/min/1.73㎡or serum creatinine based on age; hepatic and renal impairment caused by the primary disease may be considered for inclusion based on the investigator's comprehensive judgment.
- Coagulation: International normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤ 1.5 ULN.
Who cannot join
- 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 interventional clinical trials within 4 weeks before the start of the study drug.
Show all 24Show fewer
- 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 lesions receiving corticosteroid therapy for brain edema must maintain a stable dose for 14 days before enrollment. Hormone replacement therapy is allowed for patients with hypopituitarism due to primary disease involvement of the pituitary.
- 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 mean value of QTcF > 470 milliseconds or with risk factors for QTcF prolongation, such as uncorrected hypokalemia, congenital long QT syndrome, or receiving drugs known to prolong QTcF interval (mainly class Ia, Ic, and III antiarrhythmic drugs) will be excluded from the study. Drugs with the potential to prolong the QTcF interval are listed in Appendix 7.
- 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 active hepatitis B infection at the study center) or hepatitis C (positive hepatitis C virus RNA), or human immunodeficiency virus (HIV positive) infection.
- 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.
Trial locations and contacts
11 sites in 1 country. Contact the study team or a site near you to ask about taking part.
11 sites in 1 country
China11 sites, 11 recruiting
- Recruiting
Beijing Children's Hospital, Capital Medical University
Beijing, Beijing Municipality
Rui Zhang
Rui Zhang, MD
- Not yet recruiting
Children's Hospital Affiliated to the Capital Institute of Pediatrics
Beijing, Beijing Municipality
Rong Liu, MD, 13601123876, liurong@shouer.com.cn
Rong Liu, MD
- Not yet recruiting
West China Second University Hospital,Sihuan University/West China women's and Children's Hospital
Chengdu, Sichuan
Ju Gao, MD, 18180609278, gaoju651220@126.com
Ju Gao, MD
- Recruiting
Children's Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality
Jie Yu, 13983762652, 1808106657@qq.com
Jie Yu, MD
- Not yet recruiting
Sun Yat-Sen Memorial Hpsipital,Sun Yat-Sen Unniversity
Guangzhou, Guangdong
Jianpei Fang, MD
Jianpei Fang, MD
- Not yet recruiting
The First Affiliated Hospital,Sun Yat-sen University
Guangzhou, Guangdong
Xuequn Luo, 18902233573, L-xuequn@126.com
Xuequn Luo, MD
- Not yet recruiting
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang
yongmin Tang, 13858024301, y_m_tang@zju.edu.cn
yongmin Tang, MD
- Not yet recruiting
Shenzhen Children's Hospital
Shenzhen, Guangdong
Sixi Liu, MD, 18938690206, tiger647@126.com
Sixi Liu, MD
- Not yet recruiting
Children's Hospital of Soochow University
Suzhou
Shaoyan Hu, 13771870462, hsy139@126.com
Shaoyan Hu, MD
- Not yet recruiting
Tongji Hospital Affiliated to Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei
Aiguo Liu, 13807196944, drliuaiguo@163.com
Aiguo Liu, MD
- Not yet recruiting
Henan Children's Hospital Zhengzhou Children's Hospital
Zhengzhou, Henan
Wei Liu, 13673710016, liuweixinxiang@163.com
Wei Liu, MD
About this study
Official title: 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
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).
Read the detailed description
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). Approximately 56 pediatric patients will be enrolled in this study.The study included screening period, treatment period and follow-up period. Subjects will receive FCN-159 5mg/m² (NMT 8mg, the recommended oral dose for adults), orally, once daily, continuously for 28 days per cycle. Subjects will be treated until disease progression (PD), intolerable toxicity, withdrawal of consent, death, or other protocol-specified reasons. According to the PET Response Criteria and Langerhans Cell Histiocytosis Evaluation and Treatment Guidelines, tumor assessment will be performed until disease progression, death, and withdrawal of information Intention, loss of follow-up, initiation of new antitumor therapy, or study termination.Efficacy evaluation will be performed by the investigator and the Independent Review Committee (IRC), respectively.
- Conditions
- Langerhans Cell Histiocytosis, LCH
- Being tested
- FCN-159
- Medical subject headings
- Histiocytosis, Langerhans-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FCN-159Drug
5mg/m² (Maximum dose does not exceed 8mg, the recommended oral dose for adults), orally, once daily, continuously for 28 days per cycle.
| Study group | Role | What they receive |
|---|---|---|
| FCN-159 | Experimental | Experimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 5mg/m² (Maximum dose does not exceed 8mg), orally, once daily Receives: Drug: FCN-159 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR) Evaluated by Independent Review Committee (IRC) Based on PET Response Criteria (PRC)
ORR Evaluated by Independent Review Committee (IRC) Based on PET Response Criteria (PRC) is defined as the percentage of participants who achieved a best overall response of CMR or PMR assessed by IRC .
Measured: up to 24months
Secondary outcomes (12)
Objective Response Rate (ORR) Evaluated by Investigator Based on PET Response Criteria (PRC) and Langerhans Cell Histiocytosis Evaluation and Treatment Guidelines (Histiocyte Society, April 2009, hereinafter referred to as "Guidelines")
The ORR Evaluated by Investigator Based on PRC is defined as the percentage of participants who achieved a best overall response of CMR or PMR assessed by investigator.
The ORR Evaluated by Investigator Based on "Guidelines" is defined as the percentage of participants who achieved confirmed NAD or AD-better assessed by investigator.
Measured: up to 24months
Disease Control Rate (DCR) Evaluated by Investigator Based on PET Response Criteria (PRC) and "Guidelines".
The DCR Evaluated by Investigator based on PRC is defined as the percentage of participants who achieved a best overall response of CMR or PMR or SMD assessed by investigator.
The DCR Evaluated by Investigator Based on "Guidelines" is defined as the percentage of participants who achieved a confirmed NAD or AD-better or AD-stable assessed by investigator.
Measured: up to 24months
Clinical benefit rate (CBR) Evaluated by Investigator Based on PRC and "Guidelines".
The CBR Evaluated by Investigator based on PRC is defined as the percentage of participants who achieved a best overall response of CMR or PMR or SMD for a duration of ≥ 24 weeks assessed by investigator.
The CBR Evaluated by Investigator Based on "Guidelines" is defined as the percentage of participants who achieved confirmed NAD or AD-better or AD stable for a duration of ≥ 24 weeks assessed by investigator.
Measured: up to 24months
Time to Response (TTR) Evaluated by Investigator Based on PET Response Criteria (PRC) and "Guidelines"
The TTR Evaluated by Investigator based on PRC is defined as the time from the first documented achievement of CMR or PMR (whichever comes first) assessed by investigator.
The TTR Evaluated by Investigator based on the "Guidelines" criteria is defined as the time from the first documented achievement of NAD or AD-better (whichever comes first) assessed by investigator.
Measured: up to 24months
One-year Progressive Free Survival (PFS) Rate Evaluated by Investigator Based on PET Response Criteria (PRC) and "Guidelines"
The 1-year PFS Rate Evaluated by Investigator based on PRC is defined as the percentage of participants who did not achieve the first documented occurrence of PMD or death (whichever comes first) within one year after first administration of the study drug assessed by investigator.
The 1-year PFS Rate Evaluated by Investigator based on "Guidelines" is defined as the percentage of participants who did not achieve the first documented occurrence of AD-progression or death (whichever comes first) within one year after first administration of the study drug assessed by investigator.
Measured: up to 12months
Progressive Free Survival (PFS) Evaluated by Investigator Based on PET Response Criteria (PRC) and "Guidelines"
The PFS Evaluated by Investigator based on PRC is defined as the time from the first administration of the study drug to the first documented occurrence of PMD or death (whichever comes first) assessed by investigator.
The PFS Evaluated by Investigator based on "Guidelines" is defined as the time from the first administration of the study drug to the first documented occurrence of AD-progression or death (whichever comes first) assessed by investigator.
Measured: up to 24months
Overall Survival (OS)
The OS is defined as the time from the first administration of the study drug in patients to death from any cause. Data from the patient without events will be censored at the date the patient is last known to be alive
Measured: up to 24months
One-year Overall Survival (OS) Rate
The 1year OS Rate is defined as as the percentage of participants who did not achieve death due to any cause within one year after first administration of the study drug.
Measured: up to 12months
Two-year Progressive Free Survival (PFS) rate Evaluated by the PRC and "Guidelines"
The 2-year PFS Rate Evaluated by Investigator based on PRC is defined as the percentage of participants who did not achieve the first documented occurrence of PMD or death (whichever comes first) within two year after first administration of the study drug.
The 2-year PFS Rate Evaluated by Investigator based on "Guidelines" is defined as the percentage of participants who did not achieve the first documented occurrence of AD-progression or death (whichever comes first) within two year after first administration of the study drug.
Measured: up to 24months
Two-year Overall Survival (OS) Rate
The 2 year OS Rate is defined as the percentage of participants who did not achieve death due to any cause within two year after first administration of the study drug.
Measured: up to 24months
Treatment-emergent adverse events (TEAEs)
Type and frequency of treatment-emergent adverse events (TEAEs) with toxicity grades evaluated according to the National Cancer Institute-Common Toxicity Criteria Adverse Event (NCI-CTCAE) version 5.0.
Measured: up to 24months
Plasma Concentration of FCN-159
Evaluation of the pharmacokinetic characteristics of FCN-159
Measured: up to 24months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2023
- Study start28 Sep 2023
- Primary completion14 May 2025 (estimated)
- Study completion14 May 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Langerhans Cell HistiocytosisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rui Zhang, MD, Principal investigator, Beijing Children's Hospital,Captial Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05997602
- Sponsor's protocol ID
- FCN-159-004
- Status last verified
- Jan 2025
- First submitted
- 8 Aug 2023
- Last updated
- 24 Jan 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05997602
- Acronym / sponsor ID
- FCN-159-004
- Conditions
- Langerhans Cell Histiocytosis, LCH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.Industry (pharma, biotech, devices)
- Study start
- 28 Sep 2023 (actual)
- Primary completion
- 14 May 2025 (estimated)
- Study completion
- 14 May 2027 (estimated)
- First posted
- 18 Aug 2023 (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.
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).
Detailed description
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). Approximately 56 pediatric patients will be enrolled in this study.The study included screening period, treatment period and follow-up period. Subjects will receive FCN-159 5mg/m² (NMT 8mg, the recommended oral dose for adults), orally, once daily, continuously for 28 days per cycle. Subjects will be treated until disease progression (PD), intolerable toxicity, withdrawal of consent, death, or other protocol-specified reasons. According to the PET Response Criteria and Langerhans Cell Histiocytosis Evaluation and Treatment Guidelines, tumor assessment will be performed until disease progression, death, and withdrawal of information Intention, loss of follow-up, initiation of new antitumor therapy, or study termination.Efficacy evaluation will be performed by the investigator and the Independent Review Committee (IRC), respectively.
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: FCN-159Experimental: FCN-159 Dosage form:tablet Specification: 1mg，4mg Dose: FCN-159 5mg/m² (Maximum dose does not exceed 8mg), orally, once daily |
Who may qualify
- Ages
- 2 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (20 listed)
- 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 and MAP2K2), and other MEK upstream genes.If inability to get tissue, the gene testing results from a local laboratory also can be accepted.
- 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 vindesine (VDS). Alternatively, patients may be unable to tolerate chemotherapy due to severe chemotherapy toxicity. Inability to tolerate chemotherapy is defined as one of the following: Severe liver impairment (liver enzyme elevation ≥ 5 × upper limit of normal (ULN) and bilirubin elevation ≥ 1.5 × ULN), severe neurotoxicity related to vinca alkaloids, chemotherapy-related intracranial hypertension, or grade 4 bone marrow depression with severe infection (sepsis, severe pneumonia, etc.) after chemotherapy.
- Refractory/relapsed LCH is defined as the presence of one of the following:
Key exclusion criteria (24 listed)
- Patients who have received any of the following prior treatments:
- 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.
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 |
|---|---|---|
| FCN-1595mg/m² (Maximum dose does not exceed 8mg, the recommended oral dose for adults), orally, once daily, continuously for 28 days per cycle. | Drug | FCN-159 |
Primary outcome
Objective Response Rate (ORR) Evaluated by Independent Review Committee (IRC) Based on PET Response Criteria (PRC)
Time frame: up to 24months
ORR Evaluated by Independent Review Committee (IRC) Based on PET Response Criteria (PRC) is defined as the percentage of participants who achieved a best overall response of CMR or PMR assessed by IRC .
12 secondary outcomes
Objective Response Rate (ORR) Evaluated by Investigator Based on PET Response Criteria (PRC) and Langerhans Cell Histiocytosis Evaluation and Treatment Guidelines (Histiocyte Society, April 2009, hereinafter referred to as "Guidelines")
Time frame: up to 24months
Disease Control Rate (DCR) Evaluated by Investigator Based on PET Response Criteria (PRC) and "Guidelines".
Time frame: up to 24months
Clinical benefit rate (CBR) Evaluated by Investigator Based on PRC and "Guidelines".
Time frame: up to 24months
Time to Response (TTR) Evaluated by Investigator Based on PET Response Criteria (PRC) and "Guidelines"
Time frame: up to 24months
One-year Progressive Free Survival (PFS) Rate Evaluated by Investigator Based on PET Response Criteria (PRC) and "Guidelines"
Time frame: up to 12months
Progressive Free Survival (PFS) Evaluated by Investigator Based on PET Response Criteria (PRC) and "Guidelines"
Time frame: up to 24months
Overall Survival (OS)
Time frame: up to 24months
One-year Overall Survival (OS) Rate
Time frame: up to 12months
Two-year Progressive Free Survival (PFS) rate Evaluated by the PRC and "Guidelines"
Time frame: up to 24months
Two-year Overall Survival (OS) Rate
Time frame: up to 24months
Treatment-emergent adverse events (TEAEs)
Time frame: up to 24months
Plasma Concentration of FCN-159
Time frame: up to 24months
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 24 Jan 2025.
- Individual participant dataSharing planned: No
Related trials for Langerhans Cell Histiocytosis
RecruitingObservational study
Hemophagocytic Lymphohistiocytosis (HLH) Evaluation and Research of Clinical, ImmUnoLogic and TranscriptomE Study
Lymphohistiocytosis, Hemophagocytic, Secondary Hemophagocytic Lymphohistiocytosis
RecruitingObservational study
Registry for Patients With Erdheim-Chester Disease and Other Histiocytoses
Erdheim-Chester Disease
Not yet recruitingObservational study
Long Term Safety Study in Patients With HLH/MAS in Still's Disease Exposed to Gamifant.
Hemophagocytic Lymphohistiocytosis (HLH), Macrophage Activation Syndrome (MAS)
RecruitingPhase 2
A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis
Recurrent Langerhans Cell Histiocytosis, Refractory Langerhans Cell Histiocytosis
RecruitingObservational study
Hemophagocytic Lymphohistiocytosis After Allogeneic Stem Cell Transplantation
Hemophagocytic Lymphohistiocytosis (HLH)
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.
Rui Zhang, MD principal investigator
Beijing Children's Hospital,Captial Medical University
11 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing FCN-159 in people with Langerhans Cell Histiocytosis and LCH, run by Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. at 11 sites 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 20 inclusion and 24 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: looking for participants now, as of the sponsor's last update on 24 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites 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 phase mean?
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 Rui Zhang, MD (18611106187, ruizh1973@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) Evaluated by Independent Review Committee (IRC) Based on PET Response Criteria (PRC) (time frame: up to 24months). 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 24 Jan 2025.