Vaccine targeting mutated CALR for patients with myeloproliferative neoplasms
A Phase I Open Label Peptide Based Vaccine in Patients With Myeloproliferative Neoplasm Harboring CALR Mutations
This study is testing a new vaccine aimed at helping patients with myeloproliferative neoplasms, like myelofibrosis and essential thrombocythemia, by targeting a specific mutated protein to see if it can improve their immune response and health.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Icahn School of Medicine at Mount Sinai Academic / other |
| Drugs / interventions | immunotherapy |
| Locations | 1 site (New York, New York) |
| Trial ID | NCT05025488 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the safety and tolerability of a peptide-based vaccine designed to target the mutated CALR protein in patients with myeloproliferative neoplasms (MPN), specifically myelofibrosis and essential thrombocythemia. Researchers will enroll 10 patients over a 12-month period, with participation lasting up to 80 weeks. Participants will undergo various assessments, including questionnaires, bone marrow biopsies, and standard lab tests, to monitor their response to the vaccine. The goal is to enhance T cell immunity against CALR+ malignant cells, potentially leading to improved clinical outcomes.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 and older with a confirmed diagnosis of chronic phase MPN and a verified mutation in CALR exon 9.
Not a fit: Patients without CALR mutations or those with advanced stages of MPN may not benefit from this vaccine.
Why it matters
Potential benefit: If successful, this vaccine could provide a targeted immunotherapy option that may improve outcomes for patients with CALR-mutated MPN.
How similar studies have performed: While this approach is novel, similar immunotherapy strategies targeting specific mutations have shown promise in other cancer types.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Subjects must be ≥18 years of age at the time of signing the informed consent form. * Confirmed diagnosis of chronic phase MPN: * Previously treated or relapsed/refectory high risk ET * Low to intermediate 1 risk (DIPSS 0-1) PMF or ET-MF * Verified mutation in CALR exon 9 * PS ≤ 2 * Adequate organ function: * Absolute neutrophil count ≥ 1000/mm3, * Platelet count ≥ 50,000/mm3, * Creatinine ≤ 2.5 mg/dL, * Total bilirubin ≤ 2 mg/dL, (except in patients with Gilbert Syndrome who can have total bilirubin \< 3.0 mg/dL) * Transaminases \< 3 times above the upper limits of the institutional normal. * Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of at least 50 mIU/mL within 10 - 14 days prior to starting study medication and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control, one highly effective method and one additional effective method AT THE SAME TIME, at least 4 weeks prior to first dose of vaccine. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a condom during sexual contact with a female of childbearing potential even if they have had a successful vasectomy. * Ability to understand and the willingness to sign a written informed consent. * Ability to adhere to the study visit schedule and all protocol requirements. * Subjects receiving cytoreductive therapy with hydroxyurea must be on a stable dose for at least 8 weeks prior to week 1. Exclusion Criteria: * Other invasive malignancy in the past 3 years except non-melanoma skin cancer, localized cured prostate cancer and early stage breast cancer on HRT. * Active autoimmune disease. * Uncontrolled serious infection. * Known immunodeficiency. * Pregnant and breastfeeding women. * Not willing to use contraception. * Current use of immunosuppressive medications including steroids. * Current JAK inhibitor use. * Current use of IFN (use of anagrelide is permitted). * Treatment with other experimental drugs within 30 days of week 1. * Treatment with any MPN directed therapy unless otherwise noted within 5 half-lives of week 1. * Any significant psychiatric/medical condition per investigators judgment.
Where this trial is running
New York, New York
- Icahn School of Medicine at Mount Sinai — New York, New York, United States (Recruiting)
Study contacts
- Principal investigator: Marina Kremyanskaya, MD, PhD — Icahn School of Medicine at Mount Sinai
- Study coordinator: Marina Kremyanskaya, MD, PhD
- Email: marina.kremyanskaya@mssm.edu
- Phone: (212) 241-4106
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.