Trial Outcomes & Findings for Phase II Study of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma (NCT NCT03503864)

NCT ID: NCT03503864

Last Updated: 2025-05-21

Results Overview

The percentage of patients who had complete remission（CR）or partial remission（PR）after induction chemotherapy combined ATO. Per Response Evaluation Criteria In Solid Tumors Criteria (WHO criteria) for target lesions and assessed by MRI or CT: CR: All lesions completely disappear, maintained for at least 4 weeks. PR: Estimated tumor size reduction by more than 50%, maintained for at least 4 weeks.

• Recruitment status: ACTIVE_NOT_RECRUITING
• Study phase: PHASE2
• Target enrollment: 80 participants
• Primary outcome timeframe: Four weeks after ATO-combined induction chemotherapy
• Results posted on: 2025-05-21

Participant Flow

Until July 31, 2023, a total of 80 children from the paediatrics departments of 8 hospitals, were recreated in this study.

6 patients changed therapeutic regimen，7 patient withdrew consent.

Participant milestones

Measure	ATO-combined Chemotherapy Patients receive combined induction chemotherapy with arsenic trioxide. Arsenic Trioxide: Arsenic trioxide（ATO） is administered 0.18mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.
Overall Study STARTED	80
Overall Study COMPLETED	67
Overall Study NOT COMPLETED	13

Reasons for withdrawal

Measure	ATO-combined Chemotherapy Patients receive combined induction chemotherapy with arsenic trioxide. Arsenic Trioxide: Arsenic trioxide（ATO） is administered 0.18mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.
Overall Study Protocol Violation	6
Overall Study Withdrawal by Subject	7

Baseline Characteristics

Phase II Study of Combined Chemotherapy With Arsenic Trioxide in Stage 4/M Neuroblastoma

Baseline characteristics by cohort

Measure	ATO-combined Chemotherapy n=67 Participants Patients receive combined induction chemotherapy with arsenic trioxide. Arsenic Trioxide: Arsenic trioxide（ATO） is administered 0.16mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.
Age, Continuous	3.5 years n=5 Participants
Sex: Female, Male Female	27 Participants n=5 Participants
Sex: Female, Male Male	40 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	67 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment China	67 participants n=5 Participants

PRIMARY outcome

Timeframe: Four weeks after ATO-combined induction chemotherapy

The percentage of patients who had complete remission（CR）or partial remission（PR）after induction chemotherapy combined ATO. Per Response Evaluation Criteria In Solid Tumors Criteria (WHO criteria) for target lesions and assessed by MRI or CT: CR: All lesions completely disappear, maintained for at least 4 weeks. PR: Estimated tumor size reduction by more than 50%, maintained for at least 4 weeks.

Outcome measures

Measure	ATO-combined Chemotherapy n=67 Participants Patients receive combined induction chemotherapy with arsenic trioxide. Arsenic Trioxide: Arsenic trioxide（ATO） is administered 0.18mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.	Vomiting Grade 3/4 adverse events	Transaminitis Grade 3/4 adverse events	Cardiovascular Toxicity Grade 3/4 adverse events	Infection Grade 3/4 adverse events
Objective Response Rate	62 Participants	—	—	—	—

SECONDARY outcome

Timeframe: 3 years.

Population: 1 patient lost to follow-up for OS.

The proportion of patients who are alive from the date of randomization to 3 years, regardless of the cause of death.

Outcome measures

Measure	ATO-combined Chemotherapy n=66 Participants Patients receive combined induction chemotherapy with arsenic trioxide. Arsenic Trioxide: Arsenic trioxide（ATO） is administered 0.18mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.	Vomiting Grade 3/4 adverse events	Transaminitis Grade 3/4 adverse events	Cardiovascular Toxicity Grade 3/4 adverse events	Infection Grade 3/4 adverse events
Overall Survival Rate	68.0 percentage of participent Interval 53.8 to 78.6	—	—	—	—

SECONDARY outcome

Timeframe: 3 years.

Population: 6 patients lost to follow-up for PFS.

The proportion of patients who have not experienced progression or death from any cause, whichever occurs first, within a maximum of 3 years from the date of enrollment.

Treatment response was evaluated according to the WHO criteria for the efficacy of solid tumours, which was classified as complete response (CR), partial response (PR), stable disease (SD) and progressive disease (PD). PD was characterized as more than 25% increase in one or more lesions or appearance of new lesions.

Outcome measures

Measure	ATO-combined Chemotherapy n=61 Participants Patients receive combined induction chemotherapy with arsenic trioxide. Arsenic Trioxide: Arsenic trioxide（ATO） is administered 0.18mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.	Vomiting Grade 3/4 adverse events	Transaminitis Grade 3/4 adverse events	Cardiovascular Toxicity Grade 3/4 adverse events	Infection Grade 3/4 adverse events
Progression Free Survival Rate	47.7 percentage of participent Interval 32.5 to 61.4	—	—	—	—

SECONDARY outcome

Timeframe: From date of ATO-combined chemotherapy until the date of first documented adverse event, and follow up for 3 years.

Adverse events are monitored and graded by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE)

Outcome measures

Measure	ATO-combined Chemotherapy n=67 Participants Patients receive combined induction chemotherapy with arsenic trioxide. Arsenic Trioxide: Arsenic trioxide（ATO） is administered 0.18mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.	Vomiting n=67 Participants Grade 3/4 adverse events	Transaminitis n=67 Participants Grade 3/4 adverse events	Cardiovascular Toxicity n=67 Participants Grade 3/4 adverse events	Infection n=67 Participants Grade 3/4 adverse events
Number of Participants With Adverse Events	60 Participants	33 Participants	4 Participants	1 Participants	20 Participants

Adverse Events

ATO-combined Chemotherapy

Serious events: 0 serious events

Other events: 67 other events

Deaths: 22 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Measure	ATO-combined Chemotherapy n=67 participants at risk Patients receive combined induction chemotherapy with arsenic trioxide. Arsenic Trioxide: Arsenic trioxide（ATO） is administered 0.16mg/kg per day over eight hours IV daily for ten days. Patients will receive ATO alone on days 1-2 and combined with conventional induction chemotherapy on days 3-10. Nine cycles at most of ATO-combined chemotherapy were applied in the whole scheme.
Blood and lymphatic system disorders Myelosuppression	100.0% 67/67 • Adverse Events monitored up to 3 years.
Gastrointestinal disorders Vomiting	80.6% 54/67 • Adverse Events monitored up to 3 years.
Infections and infestations Infection	34.3% 23/67 • Adverse Events monitored up to 3 years.
Hepatobiliary disorders Transaminitis	34.3% 23/67 • Adverse Events monitored up to 3 years.
Cardiac disorders Cardiovascular toxicity	14.9% 10/67 • Adverse Events monitored up to 3 years.

Additional Information

Dr Yang Li

Sun Yat-sen Memorial Hospital

Phone: 02081332045

Email: drliyang@126.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place