Back to resultsThe Efficacy and Safety of HLX208 in Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation
Primary Purpose
Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH
Status
Active
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
HLX208
Sponsored by
About this trial
This is an interventional treatment trial for Langerhans Cell Histiocytosis
Eligibility Criteria
Inclusion Criteria:
- Volunteer to participate in the clinical study;
- Aged ≥ 18 years;
- Confirmed adult patients with LCH and/or ECD with BRAF V600E mutation;
- At least one measurable lesion as per PERCIST v1.0;
- Expected survival time ≥ 3 months;
- ECOG score 0-2;
Exclusion Criteria:
- Previous treatment with BRAF inhibitors or MEK inhibitors;
- 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;
- Severe active infections requiring systemic anti-infective therapy;
- Other anti-tumor treatments, such as chemotherapy, targeted therapy, or radiation therapy (except palliative radiation therapy), may be given during the study period.
Sites / Locations
- Peking Union Medical College Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
HLX208
Arm Description
Participants receive HLX208 450mg bid po
Outcomes
Primary Outcome Measures
ORR
Objective response rate(assessed by independent review committee (IRC) based on the PERCIST Version 1.0)
Secondary Outcome Measures
AEs
Incidence and severity of adverse events
ORR
Objective response rate(assessed by the investigator based on the PERCIST v1.0)
ORR
Objective response rate(assessed by IRC and the investigator based on the RECIST v1.1)
DCR
Disease control rate (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )
TTR
Time to response(assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1)
PFS
Progression-free survival (PFS) (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )
OS
Overall survival
Cmax
Maximum Plasma Concentration
Tmax
Time of Maximum Plasma Concentration
AUC
Area Under the Curve
Full Information
NCT ID
NCT05092815
First Posted
September 24, 2021
Last Updated
August 7, 2023
Sponsor
Shanghai Henlius Biotech
1. Study Identification
Unique Protocol Identification Number
NCT05092815
Brief Title
The Efficacy and Safety of HLX208 in Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation
Official Title
A Single-arm, Open Label, Multicenter Phase II Clinical Study in Rare Diseases to Evaluate Safety, Efficacy and PK of HLX208 for Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation
Study Type
Interventional
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
December 6, 2021 (Actual)
Primary Completion Date
June 30, 2024 (Anticipated)
Study Completion Date
October 30, 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Shanghai Henlius Biotech
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH, ECD
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
HLX208
Arm Type
Experimental
Arm Description
Participants receive HLX208 450mg bid po
Intervention Type
Drug
Intervention Name(s)
HLX208
Other Intervention Name(s)
BRAF V600E inhibitor
Intervention Description
HLX208 450mg bid po
Primary Outcome Measure Information:
Title
ORR
Description
Objective response rate(assessed by independent review committee (IRC) based on the PERCIST Version 1.0)
Time Frame
up to 1 year
Secondary Outcome Measure Information:
Title
AEs
Description
Incidence and severity of adverse events
Time Frame
up to 1 year
Title
ORR
Description
Objective response rate(assessed by the investigator based on the PERCIST v1.0)
Time Frame
up to 1 year
Title
ORR
Description
Objective response rate(assessed by IRC and the investigator based on the RECIST v1.1)
Time Frame
up to 1 year
Title
DCR
Description
Disease control rate (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )
Time Frame
up to 1 year
Title
TTR
Description
Time to response(assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1)
Time Frame
up to 1 year
Title
PFS
Description
Progression-free survival (PFS) (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )
Time Frame
from the first dose until firstly confirmed and recorded disease progression or death (whichever occurs earlier),assessed up to 1 year
Title
OS
Description
Overall survival
Time Frame
from the date of first dose until the date of death from any cause，assessed up to 1 year
Title
Cmax
Description
Maximum Plasma Concentration
Time Frame
from the date of first dose to 85 days
Title
Tmax
Description
Time of Maximum Plasma Concentration
Time Frame
from the date of first dose to 85 days
Title
AUC
Description
Area Under the Curve
Time Frame
from the date of first dose to 85 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Volunteer to participate in the clinical study;
Aged ≥ 18 years;
Confirmed adult patients with LCH and/or ECD with BRAF V600E mutation;
At least one measurable lesion as per PERCIST v1.0;
Expected survival time ≥ 3 months;
ECOG score 0-2;
Exclusion Criteria:
Previous treatment with BRAF inhibitors or MEK inhibitors;
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;
Severe active infections requiring systemic anti-infective therapy;
Other anti-tumor treatments, such as chemotherapy, targeted therapy, or radiation therapy (except palliative radiation therapy), may be given during the study period.
Facility Information:
Facility Name
Peking Union Medical College Hospital
City
Beijing
Country
China
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
The Efficacy and Safety of HLX208 in Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation
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