Mutant CALR-peptide Based Vaccine in Patients With Mutated CALR Myeloproliferative Neoplasm
- Conditions
- Interventions
- Registration Number
- NCT05025488
- Lead Sponsor
- Marina Kremyanskaya
- 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 ...
- Detailed Description
Current MPN treatments are geared towards symptom palliation and not on changing the natural course of the disease. Mutations in calreticulin gene (CALR) is the second most common driver mutation in ET and MF patients (30%). All CALR mutations identified to date in MPN patients result in the formation of an altered protein with an identical 36-amino acid seq...
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 10
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description CALR mutated Poly ICLC peptide-based vaccine in patients with myeloproliferative neoplasm (myelofibrosis and essential thrombocythemia) with CALR mutations CALR mutated Peptide-based vaccine peptide-based vaccine in patients with myeloproliferative neoplasm (myelofibrosis and essential thrombocythemia) with CALR mutations
- Primary Outcome Measures
Name Time Method Number of Participants with Dose Limiting Toxicity (DLT) 32 weeks 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.
- Secondary Outcome Measures
Name Time Method Change in Immune Milieu Composite Baseline through Weeks 55 or 80 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.
Proportion of participants who normalize their platelet number Week 32 and weeks 55 or 80 The proportion of patients who normalize their platelet number and/or achieve platelets less than 600 if started with platelet above 600.
Myelofibrosis Symptom Assessment Form (MF-SAFv4.0) Week 32 and weeks 55 or 80 The proportion of patients who achieve improvement in quality of life as assessed by the by the Myelofibrosis Symptom Assessment Form. Each of the items are scored 0 to 10, with total score from 0 to 100, with higher score indicating more symptoms.
Number of laboratory abnormalities Baseline through Week 32 Number of observations, severity, and relatedness of clinical laboratory tests (hematology, biochemistry)
Proportion of participants achieving response Baseline and Week 32 The proportion of patients achieving response or improvement in their disease status by ELN/IWG criteria for the categories: Complete Response; Partial Response; Clinical Improvement and Stable Disease
Change in CALR VAF Baseline through Weeks 55 or 80 The % change in driver mutation burden (CALR VAF) as compared to baseline
Number of Adverse Events Week 32 The type, incidence, severity, seriousness, and relatedness of adverse events (AEs) per NCI CTCAE v5.0.
Trial Locations
- Locations (1)
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States