MedulloblastomaPhase 2NCT07856069
Arsenic Trioxide Combined With Chemotherapy for Relapsed SHH-Activated and/or TP53-Mutant Medulloblastoma
A Phase 2 study testing Arsenic Trioxide (ATO), ETOPOSIDE - Study and Cisplatin in people with Medulloblastoma, run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University at 1 site in China.
Trial summary
This prospective, open-label, single-arm, multicenter study will evaluate the efficacy and safety of arsenic trioxide (ATO) combined with chemotherapy in pediatric patients with relapsed or refractory SHH-activated medulloblastoma and/or TP53-mutant medulloblastoma. Read more about this study
- Who is it for?
- Ages 18 months to 18 years
- Conditions the study is for
- Medulloblastoma
- What is being tested?
- Arsenic Trioxide (ATO), ETOPOSIDE - Study and Cisplatin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 78 participants
- Where
- 1 siteChina
- Study dates
- Oct 2026to Oct 2034
- Phase
- Phase 2
- 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 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Histologically confirmed SHH-activated medulloblastoma meeting at least one of the following conditions: Tumor recurrence demonstrated by imaging or cerebrospinal fluid examination after standard treatment consisting of surgery, radiotherapy, and chemotherapy; or Refractory medulloblastoma, defined as an increase in tumor size of more than 25% or the appearance of new lesions after two cycles of standard first-line chemotherapy.
- Medulloblastoma with a TP53 mutation identified by genetic testing of tumor tissue, including at least one of the following: A TP53 mutation present at initial diagnosis; A newly detected TP53 mutation during subsequent treatment in a patient with relapsed medulloblastoma; or A newly detected TP53 mutation during subsequent treatment in a patient with refractory medulloblastoma.
- Age ≥18 months and ≤18 years.
- Written informed consent obtained from the participant's legal guardian. Participants must meet either Inclusion Criterion 1 or Inclusion Criterion 2, in addition to Inclusion Criteria 3 and 4.
Who cannot join
- History of another malignancy for which the participant received chemotherapy and abdominal radiotherapy.
- Severe progressive or persistent heart failure, defined as New York Heart Association (NYHA) class III or IV heart failure or a left ventricular ejection fraction (LVEF) <50%.
- Severe progressive or persistent renal failure, defined as a glomerular filtration rate (GFR) <30 mL/min/1.73 m² or serum creatinine >5 mg/dL (442 μmol/L).
- Severe hepatic dysfunction, defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥5 times the age-appropriate upper limit of normal (ULN), or total serum bilirubin ≥3 times the age-appropriate ULN.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
Guangzhou, Guangdong, 510120
Yang Li, MD, PhD, +8613570209818, drliyang@126.com
Yang Li, MD, PhD
About this study
Official title: A Prospective, Single-Arm, Open-Label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Arsenic Trioxide Combined With Chemotherapy in Patients With Relapsed SHH-Activated and/or TP53-Mutant Medulloblastoma
This prospective, open-label, single-arm, multicenter study will evaluate the efficacy and safety of arsenic trioxide (ATO) combined with chemotherapy in pediatric patients with relapsed or refractory SHH-activated medulloblastoma and/or TP53-mutant medulloblastoma. Eligible participants will be 18 months to 18 years of age.
Participants will receive six cycles of induction chemotherapy containing ATO in combination with etoposide and either cisplatin or cyclophosphamide. The primary outcome is the objective response rate (ORR), defined as the proportion of participants achieving a complete response or partial response, assessed according to the Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria by an independent review committee.
Secondary outcomes include overall survival, progression-free survival, and treatment-related safety, including adverse events and serious adverse events. A total of 78 participants are planned for enrollment.
Read the detailed description
Medulloblastoma is a malignant pediatric central nervous system tumor. Patients with relapsed or refractory disease, particularly those with SHH-activated tumors and/or TP53 mutations, have limited treatment options and generally poor outcomes. Arsenic trioxide (ATO) has demonstrated antitumor activity through mechanisms involving inhibition of the SHH signaling pathway and restoration of the function of selected mutant TP53 proteins.
This is a prospective, open-label, single-arm, multicenter clinical study designed to investigate the efficacy and safety of ATO combined with chemotherapy in pediatric patients with relapsed or refractory SHH-activated medulloblastoma and/or TP53-mutant medulloblastoma.
Induction treatment consists of six 21-day cycles. Cycles 1, 3, and 5 include ATO, etoposide, and cisplatin, while cycles 2, 4, and 6 include ATO, etoposide, and cyclophosphamide. Intrathecal methotrexate with dexamethasone may also be administered according to the study protocol. Following induction therapy, subsequent consolidation or maintenance treatment may be selected according to the participant's clinical condition and treatment plan.
Tumor response will be assessed according to RAPNO criteria. The primary efficacy endpoint is objective response rate after completion of induction therapy. Overall survival, progression-free survival, adverse events, and serious adverse events will also be evaluated during treatment and follow-up.
- Conditions
- Medulloblastoma
- Being tested
- Arsenic Trioxide (ATO), ETOPOSIDE - Study, Cisplatin, Cyclophosphamide, Methotrexate
- Keywords
- SHH-Activated Medulloblastoma, TP53 Mutation, TP53-Mutant Medulloblastoma, Relapsed Medulloblastoma, Refractory Medulloblastoma, Arsenic Trioxide, Pediatric Medulloblastoma
- Medical subject headings
- Medulloblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Arsenic Trioxide (ATO)Drug
Arsenic trioxide 0.18 mg/kg/day is administered by intravenous infusion over more than 8 hours on Days 1-10 of each 21-day induction cycle.
ETOPOSIDE - StudyDrug
Etoposide 100 mg/m²/day is administered by intravenous infusion over more than 3 hours on Days 3-5 of each induction cycle.
CisplatinDrug
Cisplatin 90 mg/m² is administered by intravenous infusion over 6 hours on Day 3 of induction Cycles 1, 3, and 5.
CyclophosphamideDrug
Cyclophosphamide 800 mg/m² is administered by intravenous infusion over 3 hours on Day 3 of induction Cycles 2, 4, and 6.
MethotrexateDrug
Intrathecal methotrexate is administered on Day 5 according to age-based dosing specified in the protocol.
DexamethasoneDrug
Intrathecal dexamethasone is administered with methotrexate on Day 5 according to age-based dosing specified in the protocol.
| Study group | Role | What they receive |
|---|---|---|
| Arsenic Trioxide Combined With Chemotherapy | Experimental | Participants will receive six 21-day cycles of induction chemotherapy. Cycles 1, 3, and 5 consist of arsenic trioxide (ATO), etoposide, and cisplatin. Cycles 2, 4, and 6 consist of ATO, etoposide, and cyclophosphamide. Intrathecal methotrexate with dexamethasone may be administered according to the protocol. Receives: Drug: Arsenic Trioxide (ATO); Drug: ETOPOSIDE - Study; Drug: Cisplatin; Drug: Cyclophosphamide; Drug: Methotrexate; Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR)
The proportion of evaluable participants who achieve a complete response (CR) or partial response (PR) according to the Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria, as assessed by an Independent Review Committee (IRC). ORR is calculated as the number of participants with CR or PR divided by the number of participants evaluable for tumor response.
Measured: Within 4 weeks after completion of induction chemotherapy; for participants proceeding immediately to autologous stem cell transplantation (ASCT) or other consolidation therapy, before initiation of consolidation therapy.
Secondary outcomes (4)
Overall Survival (OS) Rate
Overall survival is defined as the time from study enrollment to death from any cause. Survival status will be determined from medical records and follow-up data.
Measured: 1, 2, 3, and 5 years after enrollment
Progression-Free Survival (PFS)
Progression-free survival is defined as the time from study enrollment to disease progression or death from any cause, whichever occurs first. Disease progression will be assessed according to RAPNO criteria using clinical, imaging, and follow-up data, with assessment by the Independent Review Committee.
Measured: 3 and 5 years after enrollment
Incidence of Adverse Events (AEs)
The incidence and severity of adverse events will be assessed in all participants according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), version 5.0. Adverse events will be monitored throughout treatment and follow-up.
Measured: From the first dose of study treatment through treatment and follow-up, up to 5 years
Incidence of Serious Adverse Events (SAEs)
The incidence of serious adverse events will be evaluated throughout treatment and follow-up. Serious adverse events include events resulting in death, being life-threatening, requiring or prolonging hospitalization, causing persistent or significant disability, or other medically important events.
Measured: From the first dose of study treatment through treatment and follow-up, up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Oct 2026
- Study startOct 2026 (estimated)
- Primary completionOct 2030 (estimated)
- Study completionOct 2034 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for MedulloblastomaStudies of the same condition, open and under way.
- More trials run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityThe 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
- 78 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Yang Li, MD, PhD, Principal investigator, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07856069
- Sponsor's protocol ID
- SYSKY-2026-375-01
- Status last verified
- Sep 2026
- First submitted
- 27 Sep 2026
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Individual participant data will not be shared because no data-sharing plan has been established for this study.
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Single-Arm, Open-Label, Multicenter Clinical Study to Evaluate the Efficacy and Safety of Arsenic Trioxide Combined With Chemotherapy in Patients With Relapsed SHH-Activated and/or TP53-Mutant Medulloblastoma
- ClinicalTrials.gov ID
- NCT07856069
- Acronym / sponsor ID
- SYSKY-2026-375-01
- Conditions
- Medulloblastoma
- Keywords
- SHH-Activated Medulloblastoma, TP53 Mutation, TP53-Mutant Medulloblastoma, Relapsed Medulloblastoma, Refractory Medulloblastoma, Arsenic Trioxide, Pediatric Medulloblastoma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 78 participants (estimated)
- Lead sponsor
- Sun Yat-Sen Memorial Hospital of Sun Yat-Sen UniversityUniversity, hospital or other
- Study start
- Oct 2026 (estimated)
- Primary completion
- Oct 2030 (estimated)
- Study completion
- Oct 2034 (estimated)
- First posted
- 2 Oct 2026 (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 prospective, open-label, single-arm, multicenter study will evaluate the efficacy and safety of arsenic trioxide (ATO) combined with chemotherapy in pediatric patients with relapsed or refractory SHH-activated medulloblastoma and/or TP53-mutant medulloblastoma. Eligible participants will be 18 months to 18 years of age.
Participants will receive six cycles of induction chemotherapy containing ATO in combination with etoposide and either cisplatin or cyclophosphamide. The primary outcome is the objective response rate (ORR), defined as the proportion of participants achieving a complete response or partial response, assessed according to the Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria by an independent review committee.
Secondary outcomes include overall survival, progression-free survival, and treatment-related safety, including adverse events and serious adverse events. A total of 78 participants are planned for enrollment.
Detailed description
Medulloblastoma is a malignant pediatric central nervous system tumor. Patients with relapsed or refractory disease, particularly those with SHH-activated tumors and/or TP53 mutations, have limited treatment options and generally poor outcomes. Arsenic trioxide (ATO) has demonstrated antitumor activity through mechanisms involving inhibition of the SHH signaling pathway and restoration of the function of selected mutant TP53 proteins.
This is a prospective, open-label, single-arm, multicenter clinical study designed to investigate the efficacy and safety of ATO combined with chemotherapy in pediatric patients with relapsed or refractory SHH-activated medulloblastoma and/or TP53-mutant medulloblastoma.
Induction treatment consists of six 21-day cycles. Cycles 1, 3, and 5 include ATO, etoposide, and cisplatin, while cycles 2, 4, and 6 include ATO, etoposide, and cyclophosphamide. Intrathecal methotrexate with dexamethasone may also be administered according to the study protocol. Following induction therapy, subsequent consolidation or maintenance treatment may be selected according to the participant's clinical condition and treatment plan.
Tumor response will be assessed according to RAPNO criteria. The primary efficacy endpoint is objective response rate after completion of induction therapy. Overall survival, progression-free survival, adverse events, and serious adverse events will also be evaluated during treatment and follow-up.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Arsenic Trioxide Combined With Chemotherapy | Experimental | Drug: Arsenic Trioxide (ATO), Drug: ETOPOSIDE - Study, Drug: Cisplatin, Drug: Cyclophosphamide, Drug: Methotrexate, Drug: DexamethasoneParticipants will receive six 21-day cycles of induction chemotherapy. Cycles 1, 3, and 5 consist of arsenic trioxide (ATO), etoposide, and cisplatin. Cycles 2, 4, and 6 consist of ATO, etoposide, and cyclophosphamide. Intrathecal methotrexate with dexamethasone may be administered according to the protocol. |
Who may qualify
- Ages
- 18 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Histologically confirmed SHH-activated medulloblastoma meeting at least one of the following conditions: Tumor recurrence demonstrated by imaging or cerebrospinal fluid examination after standard treatment consisting of surgery, radiotherapy, and chemotherapy; or Refractory medulloblastoma, defined as an increase in tumor size of more than 25% or the appearance of new lesions after two cycles of standard first-line chemotherapy.
- Medulloblastoma with a TP53 mutation identified by genetic testing of tumor tissue, including at least one of the following: A TP53 mutation present at initial diagnosis; A newly detected TP53 mutation during subsequent treatment in a patient with relapsed medulloblastoma; or A newly detected TP53 mutation during subsequent treatment in a patient with refractory medulloblastoma.
- Age ≥18 months and ≤18 years.
- Written informed consent obtained from the participant's legal guardian. Participants must meet either Inclusion Criterion 1 or Inclusion Criterion 2, in addition to Inclusion Criteria 3 and 4.
Key exclusion criteria (4 listed)
- History of another malignancy for which the participant received chemotherapy and abdominal radiotherapy.
- Severe progressive or persistent heart failure, defined as New York Heart Association (NYHA) class III or IV heart failure or a left ventricular ejection fraction (LVEF) <50%.
- Severe progressive or persistent renal failure, defined as a glomerular filtration rate (GFR) <30 mL/min/1.73 m² or serum creatinine >5 mg/dL (442 μmol/L).
- Severe hepatic dysfunction, defined as aspartate aminotransferase (AST) or alanine aminotransferase (ALT) ≥5 times the age-appropriate upper limit of normal (ULN), or total serum bilirubin ≥3 times the age-appropriate ULN.
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 |
|---|---|---|
| Arsenic Trioxide (ATO)Arsenic trioxide 0.18 mg/kg/day is administered by intravenous infusion over more than 8 hours on Days 1-10 of each 21-day induction cycle. | Drug | Arsenic Trioxide Combined With Chemotherapy |
| ETOPOSIDE - StudyEtoposide 100 mg/m²/day is administered by intravenous infusion over more than 3 hours on Days 3-5 of each induction cycle. | Drug | Arsenic Trioxide Combined With Chemotherapy |
| CisplatinCisplatin 90 mg/m² is administered by intravenous infusion over 6 hours on Day 3 of induction Cycles 1, 3, and 5. | Drug | Arsenic Trioxide Combined With Chemotherapy |
| CyclophosphamideCyclophosphamide 800 mg/m² is administered by intravenous infusion over 3 hours on Day 3 of induction Cycles 2, 4, and 6. | Drug | Arsenic Trioxide Combined With Chemotherapy |
| MethotrexateIntrathecal methotrexate is administered on Day 5 according to age-based dosing specified in the protocol. | Drug | Arsenic Trioxide Combined With Chemotherapy |
| DexamethasoneIntrathecal dexamethasone is administered with methotrexate on Day 5 according to age-based dosing specified in the protocol. | Drug | Arsenic Trioxide Combined With Chemotherapy |
Primary outcome
Objective Response Rate (ORR)
Time frame: Within 4 weeks after completion of induction chemotherapy; for participants proceeding immediately to autologous stem cell transplantation (ASCT) or other consolidation therapy, before initiation of consolidation therapy.
The proportion of evaluable participants who achieve a complete response (CR) or partial response (PR) according to the Response Assessment in Pediatric Neuro-Oncology (RAPNO) criteria, as assessed by an Independent Review Committee (IRC). ORR is calculated as the number of participants with CR or PR divided by the number of participants evaluable for tumor response.
4 secondary outcomes
Overall Survival (OS) Rate
Time frame: 1, 2, 3, and 5 years after enrollment
Progression-Free Survival (PFS)
Time frame: 3 and 5 years after enrollment
Incidence of Adverse Events (AEs)
Time frame: From the first dose of study treatment through treatment and follow-up, up to 5 years
Incidence of Serious Adverse Events (SAEs)
Time frame: From the first dose of study treatment through treatment and follow-up, up to 5 years
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 2 Oct 2026.
- Individual participant dataSharing planned: No. Individual participant data will not be shared because no data-sharing plan has been established for this study.
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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.
Yang Li, MD, PhD principal investigator
Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
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 Arsenic Trioxide (ATO), ETOPOSIDE - Study and Cisplatin in people with Medulloblastoma, run by Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University 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 4 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: opening soon, as of the sponsor's last update on 2 Oct 2026. 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?
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?
Contact Yang Li, MD, PhD (+86 13570209818, drliyang@126.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: Within 4 weeks after completion of induction chemotherapy; for participants proceeding immediately to autologous stem cell transplantation (ASCT) or other consolidation therapy, before initiation of consolidation therapy.). 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 2 Oct 2026.