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

ID#: NCT05025488

Age: 18 years - 66+

Gender: All

Healthy Subjects: No

Study Phase: Phase 1

Recruitment Status: Recruiting

Start Date: April 04, 2023

End Date: January 01, 2025

Contact Information:
Marina Kremyanskaya, MD, PhD
(212) 241-4106
Mikaela Dougherty
212-241-8839
Summary: The primary objective of this study is to assess the safety and tolerability of administrating mutated-CALR peptide Vaccine to patients with MPN. The researchers plan to enroll 10 patients over a 12 month period. Maximum length of participation in 80 weeks. Patients will be asked to complete questionnaires, bone marrow biopsies, research lab collection, and standard of care lab draw. This research will be taking place only at The Mount Sinai Hospital, specifically at the Ruttenberg Treatment Center.
Eligibility: Inclusion Criteria

- Subjects must be ≥18 years of age at the time of signing the informed consent form.

- Confirmed diagnosis of chronic phase MPN: high risk ET (HU failure/intolerance), low-intermediate 1 (DIPSS 0-1) PMF

- 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.

- INR<2 if off of anticoagulation. Patients on anticoagulation therapy with an INR>2 may be enrolled at the discretion of the investigator if have not had any episodes of severe hemorrhage.

- 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 before 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 child bearing potential even if 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 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 Ruxolitinib or Fedratinib use

- Current use of hydroxyurea

- Current use of INF (use of anagrelide is permitted)

- Treatment with other experimental drugs

- Any significant psychiatric/medical condition per investigators judgment