A Study of VAC85135, a Neoantigen Vaccine Regimen, Concurrently Administered With Ipilimumab for the Treatment of Myeloproliferative Neoplasms

NCT05444530 | Completed Phase 1 FDA Drug

• 4 other identifiers: CR109149VAC85135MPN10012021-006033-202022-501913-30-00
• Study Type: interventional
• Target: 14
• Locations: 2 countries
• Sites: 7

Brief Summary

The purpose of this study is to evaluate the safety of VAC85135 administered with ipilimumab for the treatment of myeloproliferative neoplasms (MPNs).

Conditions

Myeloproliferative Neoplasms

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Dose-limiting Toxicity (DLT)

  Number of participants with a DLT will be reported. The DLTs are specific adverse events and are defined as any of the following: high grade non-hematologic toxicity, or hematologic toxicity. Toxicities will be graded for severity according to the National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 5.0.

  Baseline (Day 1) up to Day 78

• Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Number of participants with AEs will be reported. An AE is any untoward medical occurrence in a clinical study participant administered a pharmaceutical product. AEs will be graded as Grade 1: Mild- asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Grade 2: Moderate- minimal, local or noninvasive intervention indicated; limiting age-appropriate instrumental activities of daily living (ADL); Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care activities of daily living; Grade 4- Life-threatening consequences- urgent intervention indicated; Grade 5: Death related to AE.

  Up to 79 weeks

Secondary Outcomes (8)

• Number of Participants With Antigen-specific T-cell response

  Up to end of treatment (EOT) (Up to 64 weeks)

• Number of Participants With Overall Response per Revised Response Criteria by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) Consensus Report

  Up to 79 weeks

• Number of Participants Disease Response at Weeks 24, 48 and End of Treatment (EOT) per Modified IWG-MRT Criteria

  Weeks 24, 48 and EOT (64 weeks)

• Number of Participants With Peripheral Blood Mutant Calreticulin (mutCALR) and Janus Kinase 2 With V617F Mutation (JAK2V617F) Allele Burden

  Up to end of treatment (EOT) (Up to 64 weeks)

• Number of Participants With Transfusion Burden

  Up to end of treatment (EOT) (Up to 64 weeks)

Study Arms (2)

Dose Escalation

EXPERIMENTAL

Participants with essential thrombocythemia (ET) and myelofibrosis (MF) will receive VAC85135 target dose intramuscular (IM) injection in the safety lead-in cohort (Cohort 0). Participants in subsequent cohorts will receive VAC85135 target dose IM injection along with ipilimumab intravenous (IV) infusion. Ipilimumab dose may be escalated based on dose limiting toxicity (DLT) observations.

Biological: VAC85135; Drug: Ipilimumab

Dose Expansion

EXPERIMENTAL

Participants with polycythemia vera (PV) or post-polycythemia vera myelofibrosis, ET and MF will receive VAC85135 target dose IM injection with ipilimumab IV infusion at the dose(s) determined by study evaluation team (SET).

Biological: VAC85135; Drug: Ipilimumab

Interventions

VAC85135 BIOLOGICAL

Participants will receive VAC85135 as IM injection.

Dose Escalation; Dose Expansion

Ipilimumab DRUG

Participants will receive Ipilimumab as IV infusion.

Dose Escalation; Dose Expansion

Eligibility Criteria

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

You may qualify if:

• Be positive for a CALR (calreticulin) mutation: Type 1 or Type 2; Type 1-like, or Type 2-like may be considered with Sponsor approval; or positive for the JAK2V617F (Janus kinase 2 with valine 617 to phenylalanine mutation) mutation with HLA-A02:01 (human leukocyte antigens) per medical history or local testing
• Have an Eastern Cooperative Oncology Group (ECOG) performance status grade of 0 or 1 or 2
• Have the following hematologic laboratory values: Leukocytes greater than or equal to (\>=) 1.5\*10\^9 per liter, Neutrophils \>=1.0\*10\^9 per liter, Platelets \>=20\*10\^9 per liter, Hemoglobin greater than (\>) 7 gram per deciliter (g/dL)
• Have the following chemistry laboratory values: Alanine aminotransferase (ALT): less than or equal to (\<=) 3\*upper limit of normal (ULN), Aspartate aminotransferase (AST): \<=3\*ULN, Total bilirubin: \<=1.5\*ULN, and glomerular filtration rate \>=40 milliliter per minute (mL/min)
• A female participant of childbearing potential must agree to all the following during the study and for 6 months after the last dose of study treatment: use a barrier method of contraception, use a highly effective preferably user-independent method of contraception, not to donate eggs (ova, oocytes) or freeze for future use for the purposes of assisted reproduction, not plan to become pregnant, not to breast-feed
• A male participant must agree to all the following during the study and for 90 days after the last dose of study treatment: wear a condom when engaging in any activity that allows for passage of ejaculate to another person, not to father a child, not to donate sperm or freeze for future use for the purpose of reproduction

You may not qualify if:

• History of any significant medical condition per investigators judgment (example: severe asthma/chronic obstructive pulmonary disease (COPD), poorly regulated heart condition, insulin dependent diabetes mellitus)
• Serious known clinically relevant allergies or earlier anaphylactic reactions
• Currently pregnant or breastfeeding
• Prior treatment with any Janus kinase 1/2 (JAK1/2) inhibitor
• Known sensitivity or contraindications to the use of Ipilimumab per local prescribing information

Sponsors & Collaborators

• Janssen Research & Development, LLC: lead
• Bristol-Myers Squibb: collaborator

Study Sites (7)

City of Hope

Duarte, California, 91010, United States

Location

Moffitt Cancer Center

Tampa, Florida, 33612, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Guy's and St Thomas' Hospital

London, SE1 9RT, United Kingdom

Location

The Christie NHS Foundation Trust Christie Hospital

Manchester, M20 4BX, United Kingdom

Location

Churchill Hospital

Oxford, OX3 7LE, United Kingdom

Location

MeSH Terms

Conditions

Myeloproliferative Disorders

Interventions

Ipilimumab

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Janssen Research & Development, LLC Clinical Trial

  Janssen Research & Development, LLC

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 30, 2022
• First Posted: July 6, 2022
• Study Start: July 21, 2022
• Primary Completion: June 24, 2025
• Study Completion: June 24, 2025
• Last Updated: July 20, 2025

Record last verified: 2025-07

Data Sharing

• IPD Sharing: Will share

Locations

US (4); GB (3)