NeuroblastomaPhase 1/2NCT05701306
APG-115 Alone or in Combination With APG-2575 in Recurrent or Refractory Neuroblastoma or Solid Tumors
A Phase 1/2 study testing APG-115 and APG-2575 in people with Neuroblastoma and Solid Tumor, run by Ascentage Pharma Group Inc. at 3 sites in China.
Trial summary
An open, non-randomized Phase Ib-II trial of dose-escalation and cohorts expansion to evaluate the safety, pharmacokinetic profile and initial efficacy of APG-115 alone or in combination with APG-2575 in the treatment of recurrent or refractory neuroblastoma or solid tumors. Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Neuroblastoma, Solid Tumor
- What is being tested?
- APG-115 and APG-2575
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 100 participants
- Where
- 3 sitesChina
- Study dates
- 28 Feb 2023to 31 Dec 2027
- Phase
- Phase 1/2
- 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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥5 years for APG-115 monotherapy; age ≥12 years for APG-115 + APG-2575 combination.
- Relapsed or refractory neuroblastoma or solid tumors with no available or suitable standard therapy.
- Physical state score ≥ 50.
- Expected survival ≥ 3 months.
- There are target lesions (neuroblastoma) or measurable lesions (other solid tumors).
- Have adequate organ function.
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- Fresh or archived tumor tissue samples should be provided prior to treatment. If none of these specimens are available, inclusion may be made after consultation with the sponsor.
- Fertile women (≥14 years of age or having menarche) must have a negative serum pregnancy test at the time of the screening visit and must not be breastfeeding or planning to become pregnant during the study period.
- A potentially fertile male subject (who has spermatoses) or female subject (ibid.) must agree to use effective contraception during the trial period and for 3 months after the trial ends (or is prematurely discontinued).
- Informed consent must be obtained before carrying out any study procedure specified in the test. For child subjects, the consent of the subject and one of the parent/legal guardian must be obtained.
- The ability to swallow research drugs.
Who cannot join
- Received biological therapy, chemotherapy, or other investigational drugs within 21 days prior to dosing.
- Received radiotherapy, minor surgery/minimally-invasive surgery; small-molecule targeted agents, short-half-life chemotherapeutic agents, or traditional Chinese medicines with anti-tumor indications within 14 days prior to study drug administration (or within 5 half-lives, whichever is shorter).
- Patients who, according to the investigators' judgment, did not recover sufficiently after surgical treatment. Patients who underwent major surgery within 21 days before receiving the study drug for the first time.
- Adverse events due to previous antitumor therapy (except grade 2 peripheral neurotoxicity and alopecia that the investigators judged to be of no safety risk) have not recovered (severity higher than grade 1 according to CTCAE version 5.0).
- Patients with active brain tumors or brain metastases.
- Active gastrointestinal diseases (e.g. Crohn's disease, ulcerative colitis, or short bowel syndrome) or other malabsorption syndromes that may affect drug absorption.
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- A known hemorrhagic predisposition/disease, such as a history of non-chemotherapy-induced thrombocytopenic bleeding within 1 year before first receiving the study drug; Have active immune thrombocytopenic purpura (ITP), active autoimmune hemolytic anemia (AIHA), or a history of platelet transfusion failure (within 1 year before first receiving the study drug); Severe gastrointestinal bleeding occurred within 3 months.
- Clinically significant cardiovascular disease, cardiomyopathy, myocardial infarction or history within 6 months prior to administration.
- Symptomatic active fungal, bacterial, and/or viral infections requiring systemic treatment.
- Unexplained fever > 38.5℃ within 2 weeks prior to initial administration (subjects with tumor-related fever, as determined by the investigator, could be enrolled).
- Received MDM2 inhibitors or BCL-2 inhibitors.
- Any other circumstances or conditions that the investigator considers the patient inappropriate for participation in the study.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Yan Yu contact
China3 sites, 3 recruiting
- Recruiting
Sun Yat-sen University Cancer Center
Guangzhou, Guangdong
Yizhuo Zhang, Ph.D.
Yizhuo Zhang, Ph.D.
- Recruiting
Tianjin Medical University Cancer Institute & Hospital
Tianjin, Tianjin Municipality, 300060
Qiang Zhao, Ph.D.
Qiang Zhao, Ph.D.
- Recruiting
Tongji Hospital, Huazhong University of Science and Technology (HUST)
Wuhan, Hubei, 430030
Aiguo Liu, Ph.D.
Aiguo Liu, Ph.D.
About this study
Official title: A Phase Ib-ⅡClinical Study of APG-115 Alone or in Combination With APG-2575 in Recurrent or Refractory Neuroblastoma or Solid Tumors
An open, non-randomized Phase Ib-II trial of dose-escalation and cohorts expansion to evaluate the safety, pharmacokinetic profile and initial efficacy of APG-115 alone or in combination with APG-2575 in the treatment of recurrent or refractory neuroblastoma or solid tumors.
Read the detailed description
Part 1: Dose escalation and expansion of APG-115 monotherapy in pediatric neuroblastoma or solid tumors to determine MTD and recommended phase 2 dose, RP2D.
Part 2: Dose escalation of APG-2575 to determine the MTD and RP2D combined with APG-115 at the dose level determined in part 1 in pediatric neuroblastoma or solid tumor, and extend the RP2D level of the combination therapy.
- Conditions
- Neuroblastoma, Solid Tumor
- Being tested
- APG-115, APG-2575
- Keywords
- Neuroblastoma, Solid Tumor, APG-115
- Medical subject headings
- Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
APG-115Drug
Orally once every other day(QOD) for 2 weeks and suspended for 1 week, 21 days as a cycle.
APG-2575Drug
Orally once a day (QD) for 21 days, 21 days as a cycle.
| Study group | Role | What they receive |
|---|---|---|
| APG-115 monotherapy in part1 | Experimental | Multiple dose cohorts, to determine the RP2D of APG-115. Receives: Drug: APG-115 |
| APG-115 combined with APG-2575 in part2 | Experimental | Multiple dose cohorts of APG-2575, to determine the RP2D of APG-2575 combined with APG-115. Receives: Drug: APG-115; Drug: APG-2575 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose Limiting Toxicity (Phase I)
DLT will be defined based on the rate of drug-related grade 3-5 adverse events experienced within the first 3 weeks of study treatment. These will be assessed via CTCAE version 5.0.
Measured: Up to 21 days
Treatment-Emergent Adverse Events per NCI-CTCAE version 5.0
Number of patients with adverse event; Number of patients with abnormal vital signs, abnorma physical examination, laboratory abnormalities, and abnormal 12-lead ECG in monotherapy of APG-115 and combined with APG-2575.
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jan 2023
- Study start28 Feb 2023
- Primary completion1 Jun 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeuroblastomaStudies of the same condition, open and under way.
- More trials run by Ascentage Pharma Group Inc.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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ascentage Pharma Group Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Suzhou Yasheng Pharmaceutical Co., Ltd.
- Responsible party
- Sponsor
- Investigators
- Yizhuo Zhang, Principal investigator, Sun Yat-Sen University Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05701306
- Sponsor's protocol ID
- APG115XC103
- Status last verified
- Aug 2026
- First submitted
- 18 Jan 2023
- Last updated
- 21 Aug 2026
- 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 21 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ib-ⅡClinical Study of APG-115 Alone or in Combination With APG-2575 in Recurrent or Refractory Neuroblastoma or Solid Tumors
- ClinicalTrials.gov ID
- NCT05701306
- Acronym / sponsor ID
- APG115XC103
- Conditions
- Neuroblastoma, Solid Tumor
- Keywords
- Neuroblastoma, Solid Tumor, APG-115
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Ascentage Pharma Group Inc.Industry (pharma, biotech, devices)
- Collaborators
- Suzhou Yasheng Pharmaceutical Co., Ltd.
- Study start
- 28 Feb 2023 (actual)
- Primary completion
- 1 Jun 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 27 Jan 2023 (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.
An open, non-randomized Phase Ib-II trial of dose-escalation and cohorts expansion to evaluate the safety, pharmacokinetic profile and initial efficacy of APG-115 alone or in combination with APG-2575 in the treatment of recurrent or refractory neuroblastoma or solid tumors.
Detailed description
Part 1: Dose escalation and expansion of APG-115 monotherapy in pediatric neuroblastoma or solid tumors to determine MTD and recommended phase 2 dose, RP2D.
Part 2: Dose escalation of APG-2575 to determine the MTD and RP2D combined with APG-115 at the dose level determined in part 1 in pediatric neuroblastoma or solid tumor, and extend the RP2D level of the combination therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| APG-115 monotherapy in part1 | Experimental | Drug: APG-115Multiple dose cohorts, to determine the RP2D of APG-115. |
| APG-115 combined with APG-2575 in part2 | Experimental | Drug: APG-115, Drug: APG-2575Multiple dose cohorts of APG-2575, to determine the RP2D of APG-2575 combined with APG-115. |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Age ≥5 years for APG-115 monotherapy; age ≥12 years for APG-115 + APG-2575 combination.
- Relapsed or refractory neuroblastoma or solid tumors with no available or suitable standard therapy.
- Physical state score ≥ 50.
- Expected survival ≥ 3 months.
- There are target lesions (neuroblastoma) or measurable lesions (other solid tumors).
Key exclusion criteria (12 listed)
- Received biological therapy, chemotherapy, or other investigational drugs within 21 days prior to dosing.
- Received radiotherapy, minor surgery/minimally-invasive surgery; small-molecule targeted agents, short-half-life chemotherapeutic agents, or traditional Chinese medicines with anti-tumor indications within 14 days prior to study drug administration (or within 5 half-lives, whichever is shorter).
- Patients who, according to the investigators' judgment, did not recover sufficiently after surgical treatment. Patients who underwent major surgery within 21 days before receiving the study drug for the first time.
- Adverse events due to previous antitumor therapy (except grade 2 peripheral neurotoxicity and alopecia that the investigators judged to be of no safety risk) have not recovered (severity higher than grade 1 according to CTCAE version 5.0).
- Patients with active brain tumors or brain metastases.
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 |
|---|---|---|
| APG-115Orally once every other day(QOD) for 2 weeks and suspended for 1 week, 21 days as a cycle. | Drug | APG-115 combined with APG-2575 in part2, APG-115 monotherapy in part1 |
| APG-2575Orally once a day (QD) for 21 days, 21 days as a cycle. | Drug | APG-115 combined with APG-2575 in part2 |
Primary outcome
Dose Limiting Toxicity (Phase I)
Time frame: Up to 21 days
DLT will be defined based on the rate of drug-related grade 3-5 adverse events experienced within the first 3 weeks of study treatment. These will be assessed via CTCAE version 5.0.
Treatment-Emergent Adverse Events per NCI-CTCAE version 5.0
Time frame: Up to 12 months
Number of patients with adverse event; Number of patients with abnormal vital signs, abnorma physical examination, laboratory abnormalities, and abnormal 12-lead ECG in monotherapy of APG-115 and combined with APG-2575.
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 21 Aug 2026.
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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.
Yan Yu contact
Yizhuo Zhang principal investigator
Sun Yat-Sen University Cancer Center
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing APG-115 and APG-2575 in people with Neuroblastoma and Solid Tumor, run by Ascentage Pharma Group Inc. at 3 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 11 inclusion and 12 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 21 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 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 combined 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.
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 Yifan Zhai, MD,PHD (+86-20-28068501, Yzhai@ascentage.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose Limiting Toxicity (Phase I) (time frame: Up to 21 days). The study lists 2 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 21 Aug 2026.