Mutant CALR-peptide Based Vaccine in Patients With Mutated CALR Myeloproliferative Neoplasm

Launched by MARINA KREMYANSKAYA · Aug 24, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a new vaccine designed for patients with a specific type of blood cancer called myeloproliferative neoplasm (MPN), which includes conditions like myelofibrosis and essential thrombocythemia. The main goal is to see if this vaccine is safe and well-tolerated by patients who have a mutation in a gene known as CALR. Researchers are looking to enroll 10 participants over a year, and involvement in the study can last up to 80 weeks. All study activities will take place at The Mount Sinai Hospital.

To be eligible for this trial, participants must be at least 18 years old and have a confirmed diagnosis of MPN with a CALR mutation. They should also have stable organ function and meet specific health criteria. Participants can expect to complete questionnaires, undergo bone marrow biopsies, and have regular blood tests during the study. It’s important for potential participants to understand that certain medical conditions and treatments may disqualify them from joining, so discussing eligibility with a healthcare provider is crucial. Overall, this study aims to explore a promising new treatment option for patients with MPN.

Gender

ALL

Eligibility 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.