Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH, ECD
Conditions
Brief summary
The purpose of this study was to assess safety, efficacy and PK in adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) given HLX208 (BRAF V600E inhibitor).
Interventions
DRUGHLX208
HLX208 450mg bid po
Sponsors
Shanghai Henlius Biotech
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Volunteer to participate in the clinical study; 2. Aged ≥ 18 years; 3. Confirmed adult patients with LCH and/or ECD with BRAF V600E mutation; 4. At least one measurable lesion as per PERCIST v1.0; 5. Expected survival time ≥ 3 months; 6. ECOG score 0-2;
Exclusion criteria
1. Previous treatment with BRAF inhibitors or MEK inhibitors; 2. A history of other malignancies within two years, except for cured cervical carcinoma in situ, basal cell carcinoma of the skin, adenocarcinoma in situ of the lung, or tumors that do not require interventional treatment after radical surgery; 3. Severe active infections requiring systemic anti-infective therapy; 4. Other anti-tumor treatments, such as chemotherapy, targeted therapy, or radiation therapy (except palliative radiation therapy), may be given during the study period.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ORR | up to 1 year | Objective response rate(assessed by independent review committee (IRC) based on the PERCIST Version 1.0) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| ORR | up to 1 year | Objective response rate(assessed by the investigator based on the PERCIST v1.0) |
| DCR | up to 1 year | Disease control rate (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 ) |
| TTR | up to 1 year | Time to response(assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1) |
| PFS | from the first dose until firstly confirmed and recorded disease progression or death (whichever occurs earlier),assessed up to 1 year | Progression-free survival (PFS) (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 ) |
| AEs | up to 1 year | Incidence and severity of adverse events |
| Cmax | from the date of first dose to 85 days | Maximum Plasma Concentration |
| Tmax | from the date of first dose to 85 days | Time of Maximum Plasma Concentration |
| AUC | from the date of first dose to 85 days | Area Under the Curve |
| OS | from the date of first dose until the date of death from any cause，assessed up to 1 year | Overall survival |
Countries
China
Outcome results
None listed