Langerhans Cell HistiocytosisPhase 2NCT05092815
The Efficacy and Safety of HLX208 in Adult Langerhans Cell Histiocytosis (LCH) and Erdheim-Chester Disease (ECD) With BRAF V600E Mutation
A Phase 2 study testing HLX208 in people with Langerhans Cell Histiocytosis and Erdheim-Chester Disease, run by Shanghai Henlius Biotech at 1 site in China.
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH
- What is being tested?
- HLX208
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteChina
- Study dates
- 6 Dec 2021to 30 Oct 2024
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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).
- Conditions
- Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH, ECD
- Being tested
- HLX208
- Medical subject headings
- Histiocytosis, Langerhans-Cell, Erdheim-Chester Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HLX208Drug
Also called BRAF V600E inhibitor
HLX208 450mg bid po
| Study group | Role | What they receive |
|---|---|---|
| HLX208 | Experimental | Participants receive HLX208 450mg bid po Receives: Drug: HLX208 |
What is being measured
The primary outcome is the main question the study is designed to answer.
ORR
Objective response rate(assessed by independent review committee (IRC) based on the PERCIST Version 1.0)
Measured: up to 1 year
Secondary outcomes (10)
AEs
Incidence and severity of adverse events
Measured: up to 1 year
ORR
Objective response rate(assessed by the investigator based on the PERCIST v1.0)
Measured: up to 1 year
ORR
Objective response rate(assessed by IRC and the investigator based on the RECIST v1.1)
Measured: up to 1 year
DCR
Disease control rate (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )
Measured: up to 1 year
TTR
Time to response(assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1)
Measured: up to 1 year
PFS
Progression-free survival (PFS) (assessed by IRC and the investigator as per PERCIST v1.0 and RECIST v1.1 )
Measured: from the first dose until firstly confirmed and recorded disease progression or death (whichever occurs earlier),assessed up to 1 year
OS
Overall survival
Measured: from the date of first dose until the date of death from any cause，assessed up to 1 year
Cmax
Maximum Plasma Concentration
Measured: from the date of first dose to 85 days
Tmax
Time of Maximum Plasma Concentration
Measured: from the date of first dose to 85 days
AUC
Area Under the Curve
Measured: from the date of first dose to 85 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2021
- Study start6 Dec 2021
- Primary completion30 Jun 2024 (estimated)
- Study completion30 Oct 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Langerhans Cell HistiocytosisStudies of the same condition, open and under way.
- More trials run by Shanghai Henlius BiotechThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Henlius Biotech
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05092815
- Sponsor's protocol ID
- HLX208-LCH/ECD201
- Status last verified
- Aug 2023
- First submitted
- 24 Sep 2021
- Last updated
- 8 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05092815
- Acronym / sponsor ID
- HLX208-LCH/ECD201
- Conditions
- Langerhans Cell Histiocytosis, Erdheim-Chester Disease, LCH, ECD
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Shanghai Henlius BiotechIndustry (pharma, biotech, devices)
- Study start
- 6 Dec 2021 (actual)
- Primary completion
- 30 Jun 2024 (estimated)
- Study completion
- 30 Oct 2024 (estimated)
- First posted
- 26 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HLX208 | Experimental | Drug: HLX208Participants receive HLX208 450mg bid po |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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;
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HLX208Also: BRAF V600E inhibitorHLX208 450mg bid po | Drug | HLX208 |
Primary outcome
ORR
Time frame: up to 1 year
Objective response rate(assessed by independent review committee (IRC) based on the PERCIST Version 1.0)
10 secondary outcomes
AEs
Time frame: up to 1 year
ORR
Time frame: up to 1 year
ORR
Time frame: up to 1 year
DCR
Time frame: up to 1 year
TTR
Time frame: up to 1 year
PFS
Time frame: from the first dose until firstly confirmed and recorded disease progression or death (whichever occurs earlier),assessed up to 1 year
OS
Time frame: from the date of first dose until the date of death from any cause，assessed up to 1 year
Cmax
Time frame: from the date of first dose to 85 days
Tmax
Time frame: from the date of first dose to 85 days
AUC
Time frame: from the date of first dose to 85 days
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Aug 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HLX208 in people with Langerhans Cell Histiocytosis and Erdheim-Chester Disease, run by Shanghai Henlius Biotech at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 8 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
ORR (time frame: up to 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Aug 2023.