A Prospective, Single-arm Clinical Study of Liposomal Mitoxantrone Combination Regimen in the Treatment of Relapsed and Refractory Solid Tumors in Children, Adolescents and Young Adults

Phase 2

Not yet recruiting

• Conditions: Solid Tumors; Children; Adolescents
• Interventions: Drug: Mitoxantrone liposome; Drug: Capecitabine; Drug: Anlotinib

• Registration Number: NCT06761417
• Lead Sponsor: Cancer Institute and Hospital, Chinese Academy of Medical Sciences

Brief Summary

The aim of this clinical study is to determine the efficacy and safety of the liposomal mitoxantrone combination regimen in treating relapsed and refractory solid tumors among children, adolescents, and young adults. The key questions it intends to address are:

Can the liposomal mitoxantrone combination regimen improve the objective response rate (ORR) compared to historical data? What are the adverse events associated with this combination regimen? Researchers will administer the liposomal mitoxantrone combination regimen (including capecitabine and anlotinib with specific dosing regimens) to the participants and closely monitor their conditions.

Participants will:

Receive the treatment regimen for 4 - 6 cycles, with each cycle lasting 21 days Undergo regular checkups and tests during the treatment period and follow-up period as per the protocol.

Have their tumor status, blood parameters, and other relevant indicators measured to evaluate the treatment effect and safety.

Detailed Description

This is a prospective, single-arm, multicenter clinical study named "A Prospective, Single-arm, Multicenter Clinical Study of Liposomal Mitoxantrone Combination Regimen in the Treatment of Relapsed and Refractory Solid Tumors in Children, Adolescents and Young Adults". The study aims to assess the safety and effectiveness of the liposomal mitoxantrone combination regimen in treating relapsed and refractory solid tumors among children, adolescents, and young adults.

The research anticipates enrolling 49 patients meeting specific criteria. The inclusion criteria require patients to have a clear understanding of the study, sign the informed consent form voluntarily, fall within the age range of 6 - 24 years with an expected survival time exceeding 3 months, have histopathologically confirmed relapsed and refractory solid tumors of certain subtypes (such as bone and soft tissue tumors, neuroblastoma, etc.), possess measurable tumor lesions detectable by CT, enhanced CT, PET/CT or MRI, and meet specific Karnofsky and ECOG scores as well as requirements for bone marrow, liver, and kidney functions. Exclusion criteria cover those with hypersensitivity to study drugs, uncontrolled other malignancies, pregnant or lactating women, and those judged unsuitable by investigators.

The treatment regimen consists of capecitabine at a dose of 1000 mg/m² from day 1 to 14, twice daily, and anlotinib with the dose adjusted according to body weight for 14 days, with a 21-day cycle repeated 4 - 6 times. Patients achieving a partial response or better will undergo local treatment like surgery or radiotherapy.

The primary endpoint of the study is the objective response rate (ORR), while secondary endpoints include the proportion of patients eligible for local treatment after treatment, disease control rate (DCR), progression-free survival (PFS), overall survival (OS), and adverse events evaluated by NCI CTCAE v5.0. This study holds great significance for exploring new treatment options for relapsed and refractory solid tumors in the target population.

Study Timeline

• Study First Submitted: 2024-12-29
• Status Verified: 2025-01
• Study First Submitted QC: 2025-01-05
• Study First Posted: 2025-01-07
• Last Update Submitted: 2025-01-07
• Last Update Posted: 2025-01-08
• Study Start: 2025-02-01
• Primary Completion: 2027-04-01
• Study Completion: 2028-02-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 49

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Liposomal Mitoxantrone Combination Regimen	Mitoxantrone liposome	Mitoxantrone liposome+Capecitabine+Anlotinib
Liposomal Mitoxantrone Combination Regimen	Capecitabine	Mitoxantrone liposome+Capecitabine+Anlotinib
Liposomal Mitoxantrone Combination Regimen	Anlotinib	Mitoxantrone liposome+Capecitabine+Anlotinib

Primary Outcome Measures

Name	Time	Method
Rate of Objective response(ORR)	Up to 6 cycles of chemotherapy (each cycle is 21 days)	Rate of Objective response (ORR) after 4 or 6 cycles of Liposomal Mitoxantrone Combination Regimen, including complete response (CR) and partial response (PR)

Secondary Outcome Measures

Name	Time	Method
Rate of Disease control (DCR)	Up to 6 cycles of Liposomal Mitoxantrone Combination Regimen (each cycle is 21 days)	Refers to the percentage of patients with confirmed complete response, partial response, and disease stability in patients with evaluable efficacy
Progression-free survival (PFS)	From date of enrollment unit the date of first documented progression or date of death from any cause, whichever came first, assessed up to 12 months.	Means from the date of enrollment to the date of first disease progression or death from any cause, whichever comes first. If the subject has no disease progression during the trial period, PFS is defined as the last date until the subject's last confirmed progression-free survival.
Overall survival (OS)	From date of enrollment unit the date of first documented date of death from any cause, assessed up to 24 months.	Overall survival (OS) was defined from the date of enrollment to the date of death from any cause.
Proportion of patients eligible for local treatment after treatment	Up to 6 cycles of chemotherapy (each cycle is 21 days)	Proportion of patients who meet the conditions for local treatment after treatment. mathematically, it can be expressed as: Proportion = (Number of patients eligible for local treatment / Total number of patients in the study) × 100%.
Safty	After signing the informed consent form, the safety monitoring began and continued until the end of the follow-up (follow-up period of 1 year).	Number of subjects with each adverse event. Safety Evaluation Criteria: The National Cancer Institute Adverse Event Common Terminology Criteria (NCICTCAE V.5.0) were used to judge the adverse events and severity during the study.

Trial Locations

Locations (1)

Cancer Hospital, Chinese Academy of Medical Sciences; 🇨🇳 Beijing, China