The Efficacy and Safety of HLX208 in Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation

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

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).

Study Overview

• Status: Active, not recruiting
• Conditions: Langerhans Cell Histiocytosis; Erdheim-Chester Disease; LCH; ECD
• Intervention / Treatment: Drug: HLX208

• Study Type: Interventional
• Enrollment (Estimated): 25
• Phase: Phase 2

Contacts and Locations

Study Locations

• China
  • Beijing, China
    • Peking Union Medical College Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: HLX208; Participants receive HLX208 450mg bid po	Drug: HLX208; HLX208 450mg bid po; Other Names: BRAF V600E inhibitor

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
ORR	Objective response rate(assessed by independent review committee (IRC) based on the PERCIST Version 1.0)	up to 1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
AEs	Incidence and severity of adverse events	up to 1 year
ORR	Objective response rate(assessed by the investigator based on the PERCIST v1.0)	up to 1 year
ORR	Objective response rate(assessed by IRC and the investigator based on the RECIST v1.1)	up to 1 year
DCR	Disease control rate (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )	up to 1 year
TTR	Time to response(assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1)	up to 1 year
PFS	Progression-free survival (PFS) (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )	from the first dose until firstly confirmed and recorded disease progression or death (whichever occurs earlier),assessed up to 1 year
OS	Overall survival	from the date of first dose until the date of death from any cause，assessed up to 1 year
Cmax	Maximum Plasma Concentration	from the date of first dose to 85 days
Tmax	Time of Maximum Plasma Concentration	from the date of first dose to 85 days
AUC	Area Under the Curve	from the date of first dose to 85 days

Collaborators and Investigators

• Sponsor: Shanghai Henlius Biotech

Study record dates

Study Major Dates

• Study Start (Actual): December 6, 2021
• Primary Completion (Estimated): June 30, 2024
• Study Completion (Estimated): October 30, 2024

Study Registration Dates

• First Submitted: September 24, 2021
• First Submitted That Met QC Criteria: October 12, 2021
• First Posted (Actual): October 26, 2021

Study Record Updates

• Last Update Posted (Actual): August 8, 2023
• Last Update Submitted That Met QC Criteria: August 7, 2023
• Last Verified: August 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lymphatic Diseases; Lung Diseases; Lung Diseases, Interstitial; Histiocytosis, Non-Langerhans-Cell; Histiocytosis, Langerhans-Cell; Histiocytosis; Erdheim-Chester Disease
• Other Study ID Numbers: HLX208-LCH/ECD201

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No