XP-005 Personalized Vaccine Alone or in Combination With Toripalimab for the Prevention of Relapse After Remission in Acute Myeloid Leukemia

NCT06980155 | Recruiting Early Phase 1 DMC

• 1 other identifier: 2024PCV002-XP005
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

The main objective of this study is to observe and evaluate the safety and tolerability of the XP-005 personalized tumor mRNA vaccine, either alone or in combination with toripalimab, for the treatment of acute myeloid leukemia patients who are in remission with minimal residual disease (MRD) positive but cannot undergo allogeneic hematopoietic stem cell transplantation.

Conditions

Acute Myeloid Leukemia

Keywords

Neoantigen; Vaccines; Acute Myeloid Leukemia

Outcome Measures

Primary Outcomes (3)

• Dose-limiting toxicity (DLT) & Maximum tolerated dose (MTD)

  Day1 to Day21

• Biologically Effective Dose (BED)

  If MTD is not reached, BED will be used.

  Day1 to Day21

• Percentage of Participants With Adverse Events (AEs)

  Percentage of Participants with Adverse Events (AEs) by Severity According to National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.

  Up to 52 weeks

Secondary Outcomes (10)

• MRD negativity rate

  Up to 52 weeks

• Degree of MRD decrease

  Up to 52 weeks

• Duration of MRD response

  Up to 52 weeks

• Recurrence-Free Survival (RFS)

  Up to 52 weeks

• Overall survival (OS)

  Up to 52 weeks

Study Arms (2)

Neoantigen tumor vaccine monotherapy arm

EXPERIMENTAL

Dose Escalation Phase vaccine: 0.4mg, 0.8mg, 1 mg Dose Expansion Phase vaccine: MTD or 1mg

Biological: Personalized neoantigen tumor vaccine

Neoantigen tumor vaccine and Toripalimab combination arm

EXPERIMENTAL

Dose Escalation Phase vaccine: 0.4mg, 0.8mg, 1 mg Dose Expansion Phase vaccine: MTD or 1 mg Toripalimab: 240mg/dose

Biological: Personalized neoantigen tumor vaccine; Biological: PD-1 inhibitor

Interventions

Personalized neoantigen tumor vaccine BIOLOGICAL

Neoantigen tumor vaccine

Neoantigen tumor vaccine and Toripalimab combination arm; Neoantigen tumor vaccine monotherapy arm

PD-1 inhibitor BIOLOGICAL

Toripalimab

Neoantigen tumor vaccine and Toripalimab combination arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subject voluntarily signs the written informed consent form and is able to comply with the scheduled visits and related procedures specified in the protocol
• Subject must be ≥ 18 years old, with no gender restrictions
• The expected survival period is ≥3 months
• Conforming to the World Health Organization (WHO) 2022 classification criteria for Acute Myeloid Leukemia (AML).
• Subject has completed induction and consolidation chemotherapy and has achieved complete remission (CR), complete remission with partial hematologic recovery (CRh), or complete remission with incomplete hematologic recovery (CRi) according to the 2022 European LeukemiaNet (ELN) criteria. The patient does not meet the criteria for or has contraindications to stem cell transplantation.
• CR is defined as: bone marrow blasts \<5%, absence of circulating blasts or blasts with Aure rods; absence of extramedullary disease, absolute neutrophil count (ANC) ≥1.0 × 10\^9/L, and platelet count ≥100 × 10\^9/L.
• CRh is defined as: ANC ≥0.5 × 10\^9/L, and platelet count ≥50 × 10\^9/L, otherwise all other CR criteria met CRi is defined as: All CR criteria except for ANC \<1.0 × 10\^9/L or platelet count \<100 × 10\^9/L. If both CRh and CRi are considered, CRi only includes patients who do not meet the criteria for CRh.
• MRD positivity (①when using MFC, MRD is considered positive if the proportion of immunophenotypically abnormal cells among CD45+ cells is ≥0.01%; ② when using qPCR, MRD is considered positive if the NPM1 \<3log10 reduction in BM.
• NPM1 mutation classification as Type A, D, G, H, B, and J
• The peripheral blood HLA typing is HLA-A02:01
• The Eastern Cooperative Oncology Group Performance Status (ECOG PS) score is 0-2
• Subject must be willing to provide existing valid diagnostic evidence prior to treatment or undergo bone marrow aspiration and biopsy, and must also be willing to undergo bone marrow aspiration and biopsy after receiving treatment

You may not qualify if:

• Copy number variants (CNVs) or loss of heterozygosity (LOH) in HLA-related genes or chromosomal regions were detected by genetic sequencing
• Received chemotherapy, hormonal therapy, traditional Chinese medicine with anti-tumor indications, or other anti-tumor treatments within 4 weeks before the first dose (for mitomycin and nitrosoureas, within 6 weeks after the last dose), or within 5 half-lives of immunotherapy or molecular targeted therapy
• Received tumor vaccines, cellular therapy, or planned to receive other vaccines within 4 weeks before the first dose
• Subject who has undergone major surgery other than diagnostic or biopsy procedures within 4 weeks before the first dose, or who are expected to undergo major surgery during the study period
• Uncontrolled central nervous system (CNS) lymphoma
• Patients with extramedullary disease, or those deemed unsuitable for enrollment by the investigator
• Eligible for allogeneic bone marrow or allogeneic stem cell transplantation at the time of Screening
• Subject has previously undergone allogeneic hematopoietic stem cell transplantation or organ transplantation, or who is planned to undergo organ transplantation during this study
• Within 7 days before treatment, laboratory tests show:
• AST (SGOT) / ALT (SGPT) \> 3 ULN
• Total bilirubin \> 2 ULN
• eGFR \< 45 mL/min
• SpO2 \< 95% without supplemental oxygen
• DIC (Disseminated Intravascular Coagulation)
• Active malignancy

Sponsors & Collaborators

• Shanghai Jiao Tong University School of Medicine: lead
• Shanghai Xinpu BioTechnology Company Limited: collaborator

Study Sites (1)

Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Shanghai, China

RECRUITING

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Immune Checkpoint Inhibitors

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Molecular Mechanisms of Pharmacological Action; Pharmacologic Actions; Chemical Actions and Uses; Antineoplastic Agents, Immunological; Antineoplastic Agents; Therapeutic Uses

Central Study Contacts

Zhen Jin

CONTACT

15821962620; jane-king@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of hematology

Study Record Dates

• First Submitted: May 12, 2025
• First Posted: May 20, 2025
• Study Start: May 30, 2025
• Primary Completion (Estimated): January 30, 2027
• Study Completion (Estimated): August 30, 2028
• Last Updated: May 20, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)