Malignant Brain TumorsPhase 1NCT07158710
Phase I Study of HSK42360 in Malignant Brain Tumors With BRAF V600 Mutation
A Phase 1 study testing HSK42360 in people with Malignant Brain Tumors, run by Haisco Pharmaceutical Group Co., Ltd. at 6 sites in China.
Trial summary
This is a phase I, open-label, dose-escalation and expansion study to evaluate the safety, tolerability, PK of HSK42360 when given orally in pediatric patients with active BRAF V600 mutation recurrent malignant brain tumors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Malignant Brain Tumors
- What is being tested?
- HSK42360
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 159 participants
- Where
- 6 sitesChina
- Study dates
- 15 Aug 2025to 3 Dec 2029
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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
- Age ≥6 and <18 years.
- Karnofsky/Lansky Performance Status >60.
- Life expectancy ≥ 3 months.
- Patients with recurrent malignant brain tumors confirmed by histology or cytology, who have failed standard treatment (disease progression after treatment or intolerable treatment); patients who have previously received BRAF and/or MEK inhibitor therapy are allowed to be included in this study.
- Positive BRAF V600 mutation result confirmed prior to the administration of HSK42360.
- Patients will provide blood or tumor sample according to their own willingness.
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- Measurable disease by RANO criteria.
- Patients with inactive CNS lesions, or patients treated with ≤5mg/day corticosteroid and without convulsion for ≥2 weeks.
- Adequate hematologic, hepatic, and renal function.
- Women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry, for the duration of study participation, and for 90 days after the last dose.
Who cannot join
- Patients with NF1 mutation.
- malignant tumor within 2 years, with the exception of cutaneous squamous cell carcinoma, cervical carcinoma in situ, papillary thyroid carcinoma, or other tumors with low malignancy.
- Uncontrollable pleural effusion, ascites, or pericardial effusion per protocol.
- Treatment with any of the following:
- Prior treatment with anti-tumor drug within 4 weeks or approximately 5 × t1/2 prior to the first dose of HSK42360, whichever is shorter; Prior treatment with nitrosourea or mitomycin C within 6 weeks prior to the first dose of HSK42360; Prior treatment with palliative radiotherapy or anti-tumor herbs within 2 weeks prior to the first dose of HSK42360; Prior treatment with radiotherapy, electric field therapy, or other anti-tumor therapies within 4 weeks prior to the first dose of HSK42360.
- Any unresolved toxicities from prior therapy greater than Common Terminology Criteria for Adverse Events (CTCAE) grade 1 at the time of starting study treatment, with the exception of alopecia, dermal toxicity, and other toxicity considering no safety risks by investigator.
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- Any disease which would preclude drug absorption, metabolism or pharmacokinetics, eg. active peptic ulcer or chronic gastroesophageal reflux disease.
- Patient who have clinically significant or uncontrolled cardiac disease, include: QTc interval ≥ 450 msec; any clinically significant arrhythmia; left ventricular ejection fraction < 50%; myocardial infarction, unstable angina, or class III/IV cardiac failure by the NYHA that occurred within 6 months prior to the first dose of HSK42360.
- Any thromboembolic events within 6 months prior to the first dose of HSK42360; any familial or aquired thrombophilia.
- Any unstable systemic disease, eg. severe metabolic disease: liver cirrhosis, renal failure, or uremia.
- Treatment with inhibitors/inducers for CYP3A4, or substrates of CYP3A4, CYP2C9, CYP2C8, OATP1B1, OATP1B3, OAT1, OAT3, P-gp or BCRP within 14 days or approximately 5 × t1/2 prior to the first dose of HSK42360, whichever is shorter.
- Patient with cognitive dysfunction, or history of mental illness, other uncontrolled comorbidities, alcohol dependence, hormone dependence or drug abuse.
- Autologous transplantation surgery within 3 months prior to the first dose of HSK42360; Allogeneic transplantation, or stem-cell Transplant surgery within 6 months prior to the first dose of HSK42360; Major surgery or significant traumatic injury occurring within 4 weeks prior to the first dose of HSK42360.
- Patient with a history of immunodeficiency, including HIV positive, or other acquired/congenital immunodeficiency diseases.
- Patient with severe retinal abnormalities and uveitis.
- Patient with active hepatitis B or hepatitis C.
- Allergic to any HSK42360 active constituent or ingredients.
- Participate in other clinical trials within 4 weeks prior to the first dose of HSK42360.
- Positive pregnancy test, or breastfeeding.
- Any other circumstances that would, in the investigator's judgment, prevent the subject's participation in the clinical study due to safety concerns or compliance with clinical study procedures.
Trial locations and contacts
6 sites in 1 country. Contact the study team or a site near you to ask about taking part.
6 sites in 1 country
China6 sites, 6 recruiting
- Recruiting
Xuanwu Hospital Capital Medical University
Beijing, Beijing Municipality, 100021
Cuibai Wei, 13910076457, xwgcpht@xwh.ccmu.edu.cn
- Recruiting
Department of Neuro-oncology, Cancer Center, Beijing Tiantan Hospital, Capital Medical University,Beijing, China
Beijing, Beijing Municipality, 100070
WenBin Li, 15301377998, liwenbin@ccmu.edu.cn
- Recruiting
The Third Bethune Hospital of Jilin University
Changchun, Jilin, 130000
Yufei Gao, 13331686535, 416566173@qq.com
- Recruiting
The first affiliated hospital of fujian medical university
Fuzhou, Fujian, 350200
Dezhi Kang, 15759413951, kdz99988@sina.com
- Recruiting
Zhujiang Hospital of Southern Medical University
Guangzhou, Guangdong, 510280
Hongbo Guo, 020-62783372, zjyygcp@163.com
- Recruiting
Fudan University Affiliated Huashan Hospital
Shanghai, Shanghai Municipality, 200000
Tianming Qiu, 13501665370, Tianming2100@126.com
About this study
Official title: A Phase I, Open-label, Dose-escalation and Expansion Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HSK42360 in Pediatric Patients With BRAF V600-Mutant Malignant Brain Tumors
This is a phase I, open-label, dose-escalation and expansion study to evaluate the safety, tolerability, PK of HSK42360 when given orally in pediatric patients with active BRAF V600 mutation recurrent malignant brain tumors.
- Conditions
- Malignant Brain Tumors
- Being tested
- HSK42360
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HSK42360Drug
Oral administration, QD/BID
| Study group | Role | What they receive |
|---|---|---|
| Phase Ia: HSK42360 as monotherapy | Experimental | Phase 1a: dose escalation of HSK42360 as monotherapy at various dose levels Receives: Drug: HSK42360 |
| Phase Ib: HSK42360 as monotherapy | Experimental | Phase 1b: dose expansion for HSK40118 as monotherapy at a dose determined during Phase 1a in patients with BRAF V600 recurrent mutation malignant brain tumors Receives: Drug: HSK42360 |
What is being measured
The primary outcome is the main question the study is designed to answer.
MTD
MTD determination: dose limiting toxicity (DLT) rate
Measured: Up to approximately 52 months
DLTs
Incidence of dose-limiting toxicities (DLTs) at Cycle1
Measured: Up to approximately 52 months
AEs
Rate and severity of adverse events of HSK42360 as monotherapy
Measured: Up to approximately 52 months
RP2D
RP2D determination: DLT, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary safety and anticancer activity data
Measured: Up to approximately 52 months
Karnofsky/Lansky Performance Scale
Change of the grade as a part of HSK43260 safety data
Measured: Up to approximately 52 months
Secondary outcomes (9)
Overall response rate (ORR)
ORR, defined as the proportion of patients who experience a best response of confirmed CR or PR according to RANO
Measured: Up to approximately 52 months
Disease control rate (DCR)
DCR, defined as the proportion of patients who experience a best response of CR, PR, MR or stable disease (SD) according to RANO
Measured: Up to approximately 52 months
Duration of response (DOR)
DOR, defined as the time from first documented response of CR, PR or MR to the date of first documented progressive disease or death due to any cause, whichever occurs first
Measured: Up to approximately 52 months
Progression free survival (PFS)
PFS, defined as the time frocease or death due to any cause, whichever occurs first
Measured: Up to approximately 52 months
Overall survival (OS)
OS, defined as the time from the first dose of HSK42360 until the date of death due to any cause
Measured: Up to approximately 52 months
Area under the curve (AUC)
Measured: Circle 1 (28days)
maximum plasma concentration (Cmax)
Measured: Circle 1 (28days)
half-life (t1/2)
Measured: Circle 1 (28days)
Tmax(Time to maximum plasma concentration)
Measured: Circle 1 (28days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2025
- Study start15 Aug 2025
- Primary completion23 May 2029 (estimated)
- Study completion3 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Malignant Brain TumorsStudies of the same condition, open and under way.
- More trials run by Haisco Pharmaceutical Group Co., Ltd.The 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 159 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Haisco Pharmaceutical Group Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07158710
- Sponsor's protocol ID
- HSK42360-103
- Status last verified
- Jul 2025
- First submitted
- 6 Aug 2025
- Last updated
- 8 Sep 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-label, Dose-escalation and Expansion Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HSK42360 in Pediatric Patients With BRAF V600-Mutant Malignant Brain Tumors
- ClinicalTrials.gov ID
- NCT07158710
- Acronym / sponsor ID
- HSK42360-103
- Conditions
- Malignant Brain Tumors
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 159 participants (estimated)
- Lead sponsor
- Haisco Pharmaceutical Group Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Aug 2025 (actual)
- Primary completion
- 23 May 2029 (estimated)
- Study completion
- 3 Dec 2029 (estimated)
- First posted
- 8 Sep 2025 (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.
This is a phase I, open-label, dose-escalation and expansion study to evaluate the safety, tolerability, PK of HSK42360 when given orally in pediatric patients with active BRAF V600 mutation recurrent malignant brain tumors.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phase Ia: HSK42360 as monotherapy | Experimental | Drug: HSK42360Phase 1a: dose escalation of HSK42360 as monotherapy at various dose levels |
| Phase Ib: HSK42360 as monotherapy | Experimental | Drug: HSK42360Phase 1b: dose expansion for HSK40118 as monotherapy at a dose determined during Phase 1a in patients with BRAF V600 recurrent mutation malignant brain tumors |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Age ≥6 and <18 years.
- Karnofsky/Lansky Performance Status >60.
- Life expectancy ≥ 3 months.
- Patients with recurrent malignant brain tumors confirmed by histology or cytology, who have failed standard treatment (disease progression after treatment or intolerable treatment); patients who have previously received BRAF and/or MEK inhibitor therapy are allowed to be included in this study.
- Positive BRAF V600 mutation result confirmed prior to the administration of HSK42360.
Key exclusion criteria (20 listed)
- Patients with NF1 mutation.
- malignant tumor within 2 years, with the exception of cutaneous squamous cell carcinoma, cervical carcinoma in situ, papillary thyroid carcinoma, or other tumors with low malignancy.
- Uncontrollable pleural effusion, ascites, or pericardial effusion per protocol.
- Treatment with any of the following:
- Prior treatment with anti-tumor drug within 4 weeks or approximately 5 × t1/2 prior to the first dose of HSK42360, whichever is shorter; Prior treatment with nitrosourea or mitomycin C within 6 weeks prior to the first dose of HSK42360; Prior treatment with palliative radiotherapy or anti-tumor herbs within 2 weeks prior to the first dose of HSK42360; Prior treatment with radiotherapy, electric field therapy, or other anti-tumor therapies within 4 weeks prior to the first dose of HSK42360.
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 |
|---|---|---|
| HSK42360Oral administration, QD/BID | Drug | Phase Ia: HSK42360 as monotherapy, Phase Ib: HSK42360 as monotherapy |
Primary outcome
MTD
Time frame: Up to approximately 52 months
MTD determination: dose limiting toxicity (DLT) rate
DLTs
Time frame: Up to approximately 52 months
Incidence of dose-limiting toxicities (DLTs) at Cycle1
AEs
Time frame: Up to approximately 52 months
Rate and severity of adverse events of HSK42360 as monotherapy
RP2D
Time frame: Up to approximately 52 months
RP2D determination: DLT, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary safety and anticancer activity data
Karnofsky/Lansky Performance Scale
Time frame: Up to approximately 52 months
Change of the grade as a part of HSK43260 safety data
9 secondary outcomes
Overall response rate (ORR)
Time frame: Up to approximately 52 months
Disease control rate (DCR)
Time frame: Up to approximately 52 months
Duration of response (DOR)
Time frame: Up to approximately 52 months
Progression free survival (PFS)
Time frame: Up to approximately 52 months
Overall survival (OS)
Time frame: Up to approximately 52 months
Area under the curve (AUC)
Time frame: Circle 1 (28days)
maximum plasma concentration (Cmax)
Time frame: Circle 1 (28days)
half-life (t1/2)
Time frame: Circle 1 (28days)
Tmax(Time to maximum plasma concentration)
Time frame: Circle 1 (28days)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Sep 2025.
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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.
Department of Neuro-oncology, Cancer Center, Beijing Tiantan Hospital, Capital Medical University,Beijing, China
Beijing, China
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HSK42360 in people with Malignant Brain Tumors, run by Haisco Pharmaceutical Group Co., Ltd. at 6 sites 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 10 inclusion and 20 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: looking for participants now, as of the sponsor's last update on 8 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
MTD (time frame: Up to approximately 52 months). The study lists 5 primary outcomes in all. 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 Sep 2025.