Recruiting
Phase 1

Mutant CALR Vaccine

Sponsor:

Marina Kremyanskaya

Code:

NCT05025488

Conditions

Myelofibrosis

Essential Thrombocythemia

MPN

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

Interventions

Peptide-based vaccine

Poly ICLC

Study Details

Brief 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.

Conditions

Myelofibrosis

Essential Thrombocythemia

MPN

Study ID

NCT05025488

Start date

Apr 4, 2023

Status verified date

Apr, 2025

Completion date

Mar, 2027

Anticipated

Primary completion date

Mar, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

10 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: CALR mutated

peptide-based vaccine in patients with myeloproliferative neoplasm (myelofibrosis and essential thrombocythemia) with CALR mutations

Interventions

Peptide-based vaccine

ten (10) doses of Mutant-CALR peptides with KLH as helper peptide (in the first vaccine only). Mutant-CALR vaccine will administered every 2 weeks for the first 4 doses and then every 4 weeks for additional 6 doses. Maintenance Treatment The protocol allows for a continued administration of up to four (4) additional Mutant-CALR vaccine and four (4) Poly-ICLC administrations, 12 weeks apart.

Poly ICLC

ten (10) doses of Poly-ICLC. Poly-ICLC will be given on weeks 1, 3, 5, 7, 11, 15, 19, 23, 27 and 31. each Poly-ICLC dose must be given the day after the corresponding Mut-CALR vaccination.

Primary outcome measure

  • Number of Participants with Dose Limiting Toxicity (DLT) [ Time Frame: 32 weeks ]

Central Contacts and Locations

Central contacts

Marina Kremyanskaya, MD, PhD

(212) 241-4106marina.kremyanskaya@mssm.edu

Gabriela Bello

(212) 241-0463gabriela.bello@mssm.edu

Locations

Icahn School of Medicine at Mount Sinai

Recruiting

New York, New York, United States, 10029

Contacts

Gabriela Bello, BS

212-241-0463gabriela.bello@mssm.edu

Principal Investigator:

Marina Kremyanskaya, MD, PhD

More Information

Sponsor

Marina Kremyanskaya

Last update posted

Apr 6, 2025

Last verified

Apr, 2025

Keywords

  • CALR
  • Vaccine
  • MF
  • ET
  • peptide

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Marina Kremyanskaya on 2025-04-06.