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

Langerhans Cell Histiocytosis Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05092815
• Other study ID #: HLX208-LCH/ECD201
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: December 6, 2021
• Est. completion date: October 30, 2024

Study information

• Verified date: August 2023
• Source: Shanghai Henlius Biotech
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 25
• Est. completion date: October 30, 2024
• Est. primary completion date: June 30, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• ECD
• Erdheim-Chester Disease
• Histiocytosis
• Histiocytosis, Langerhans-Cell
• Langerhans Cell Histiocytosis

Intervention

Drug:
• HLX208
HLX208 450mg bid po

Locations

Country	Name	City	State
China	Peking Union Medical College Hospital	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Shanghai Henlius Biotech

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	ORR	Objective response rate(assessed by independent review committee (IRC) based on the PERCIST Version 1.0)	up to 1 year
Secondary	AEs	Incidence and severity of adverse events	up to 1 year
Secondary	ORR	Objective response rate(assessed by the investigator based on the PERCIST v1.0)	up to 1 year
Secondary	ORR	Objective response rate(assessed by IRC and the investigator based on the RECIST v1.1)	up to 1 year
Secondary	DCR	Disease control rate (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )	up to 1 year
Secondary	TTR	Time to response(assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1)	up to 1 year
Secondary	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
Secondary	OS	Overall survival	from the date of first dose until the date of death from any cause,assessed up to 1 year
Secondary	Cmax	Maximum Plasma Concentration	from the date of first dose to 85 days
Secondary	Tmax	Time of Maximum Plasma Concentration	from the date of first dose to 85 days
Secondary	AUC	Area Under the Curve	from the date of first dose to 85 days