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

Phase 2

Active, not recruiting

• Conditions: Langerhans Cell Histiocytosis; LCH; ECD; Erdheim-Chester Disease
• Interventions: Drug: HLX208

• Registration Number: NCT05092815
• Lead Sponsor: Shanghai Henlius Biotech

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).

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-09-24
• Study First Submitted QC: 2021-10-12
• Study First Posted: 2021-10-26
• Study Start: 2021-12-06
• Status Verified: 2023-08
• Last Update Submitted: 2023-08-07
• Last Update Posted: 2023-08-08
• Primary Completion: 2024-06-30
• Study Completion: 2024-10-30

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 25

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HLX208	HLX208	Participants receive HLX208 450mg bid po

Primary Outcome Measures

Name	Time	Method
ORR	up to 1 year	Objective response rate(assessed by independent review committee (IRC) based on the PERCIST Version 1.0)

Secondary Outcome Measures

Name	Time	Method
AEs	up to 1 year	Incidence and severity of adverse events
Tmax	from the date of first dose to 85 days	Time of Maximum Plasma Concentration
DCR	up to 1 year	Disease control rate (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )
ORR	up to 1 year	Objective response rate(assessed by IRC and the investigator based on the RECIST v1.1)
Cmax	from the date of first dose to 85 days	Maximum Plasma Concentration
AUC	from the date of first dose to 85 days	Area Under the Curve
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 )
OS	from the date of first dose until the date of death from any cause，assessed up to 1 year	Overall survival

Trial Locations

Locations (1)

Peking Union Medical College Hospital; 🇨🇳 Beijing, China