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

ConditionsMyelofibrosisEssential ThrombocythemiaMPN
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18+
SponsorMarina Kremyanskaya

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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

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

Disqualifiers

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.

Trial design

Design model

Single group

Treatments tested in this trial

  • Peptide-based vaccine

    Drug

    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

    Drug

    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.

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: CALR mutatedExperimental treatment 2 interventions

Trial outcomes

Primary outcomes

1

Number of Participants with Dose Limiting Toxicity (DLT)

The Dose Limiting Toxicity (DLT) rate, defined as the proportion of patients with at least 1 grade 3 or higher AE considered to be at least possibly related to the treatment with Poly ICLC and CALR vaccines.

Time frame
32 weeks

Secondary outcomes

1

Number of Adverse Events

The type, incidence, severity, seriousness, and relatedness of adverse events (AEs) per NCI CTCAE v5.0.

Time frame
Week 32
2

Number of laboratory abnormalities

Number of observations, severity, and relatedness of clinical laboratory tests (hematology, biochemistry)

Time frame
Baseline through Week 32
3

Change in Immune Milieu Composite

Changes in the immune milieu (which is a composite of expression of cytokines, presence of antibodies, alterations in number and phenotype of immune cells and induction of vaccine-specific T cell response) due to the vaccines as compared to baseline values.

Time frame
Baseline through Weeks 55 or 80
4

Change in CALR VAF

The % change in driver mutation burden (CALR VAF) as compared to baseline

Time frame
Baseline through Weeks 55 or 80

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Marina Kremyanskaya

Lead sponsor

Icahn School of Medicine at Mount Sinai

Sponsor institution

Source ClinicalTrials.gov
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