Blood and Lymph Conditions

All Conditions

Heart and Blood Diseases

Rare Diseases

Myeloproliferative Disorders

Primary Myelofibrosis

Thrombocytosis

Thrombocythemia, Essential

Bone Marrow Diseases

Hematologic Diseases

Blood Platelet Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Chronic Myeloproliferative Disorders

Essential Thrombocythemia

All Drugs and Chemicals

Vaccines

Monatide (IMS 3015)

200 ug CALRLong36 peptide in water mixed with 500ul montanide

CALRLong36 peptide

Jacob H Grauslund, MD

A phase-I-first in man study in patients with calreticulin(CALR)-mutant MPN by vaccinating with exon 9 mutated peptide with the adjuvant Montanide ISA-51 to monitor safety and toxicity and the immunological response to vaccination.

The Philadelphia-chromosome negative chronic myeloproliferative neoplasms (MPN) are acquired cancer diseases, that arise due to mutations in the hematopoietic stem cells in the bone marrow. Median age at diagnosis is approximately 65 years of age and approximately 400 Danes are diagnosed with MPN annually. The MPN comprise essential thrombocythemia (ET), polycythemia vera (PV) and primary myelofibrosis (PMF). In December 2013 two independent research groups reported on the occurrence of somatic mutations in exon 9 of the calreticulin gene in patients with ET and PMF.

The overall rationale for a vaccine with CALR-mutant epitopes is that it will initiate a CALR-mutant specific immune response, which will "release the brakes" on the CALR-mutant specific immune response.

10 patients treated with standard therapy are needed for the trial and each patient will receive 15 vaccinations over the course of one year.

CALR Exon 9 Mutant Peptide Vaccine to Patients With CALR-mutant Myeloproliferative Neoplasms

A Phase-1-first in Man Study in Patients With CALR-mutant Myeloproliferative Neoplasms by Vaccinating With CALR Exon 9 Mutant Peptide

Next Generation Sequencing pre- and post-treatment

Revised response criteria by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) and European LeukemiaNet (ELN) consensus report

Adverse events are graded 1-5 according to the criteria

T-cell cytokine release towards target antigens

Inge Marie Svane

Professor, MD, PhD

Symptoms and General Pathology

Neoplasms

Immune System Diseases

Syndrome

Leukemia

Leukemia, Myeloid

Leukemia, Myeloid, Acute

Preleukemia

Myelodysplastic Syndromes

Precursor Cell Lymphoblastic Leukemia-Lymphoma

Leukemia, Lymphoid

Neoplasm Metastasis

Lymphoma

Neoplasms by Histologic Type

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Myeloid Leukemia

Acute Myeloid Leukemia

Acute Non Lymphoblastic Leukemia

Acute Lymphoblastic Leukemia

Lymphoblastic Lymphoma

Acute Graft Versus Host Disease

Lymphosarcoma

Standard and routine care.

For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

For storage,limited volumes of blood or bone marrow aspirate will be added to usual sampling and stored.

Biobanking

Hervé DOMBRET, Pr

Jérôme Lambert, Pr

Thorsten Braun, Pr

André Baruchel, Pr

Hervé Dombret, Pr

If for years the treatment strategy of leukemia and related disorders (LRDs, including acute leukemias and predisposition syndromes) has been based solely on whether the patient could receive or not intensive chemotherapy and transplantation, the advent of new targeted or less targeted drugs has led to the development of a growing number of new therapeutic approaches, very often offered to specific patient/disease subsets, justifying the generic term of 'precision medicine'.

As an international leukemia center of excellence, THEMA, the French National Center for Precision Medicine in Leukemia (selected as IHUB-2 by the French National Agency for Research), is a care, research, transfer and education initiative located at the Saint-Louis Research Institute (IRSL) in Paris and devoted to precision medicine in leukemia in a real-life environment.

The present non-interventional study (eTHEMA) is a pillar of the whole THEMA project. As a prerequisite for precision medicine, this program focuses on individual data collection, aiming to collect high-quality data not only in patients treated into prospective clinical trials, but in every THEMA patient with a special interest in outpatients' care and research.

The primary objective of this non-interventional study is to describe the baseline characteristics planned treatments and outcomes of patients newly diagnosed with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS), or myeloproliferative neoplasm (MPN)-related myelofibrosis, when managed and treated according to standard diagnosis and care practices.

The Patient Cohort of the National Center for Precision Medicine in Leukemia

Treatment-related toxicities will be Evaluated by the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0

Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.

Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire.Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.

Quality-of-life will be assessed using the EORTC-QLQ-C30 v3 questionnaire. Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.

Assistance Publique - Hôpitaux de Paris

Fuad Huamán Garaicoa, MD

The goal of this observational study is to evaluate the epidemiological and clinical-pathological features of Philadelphia-negative myeloproliferative neoplasms through data from medical records

The investigators are proposing a study that provides descriptive understanding of patients with Philadelphia-negative myeloproliferative neoplasms in Ecuador. The primary objective of this pilot study is to lay the foundations for the development of a multicenter registry of Philadelphia-negative myeloproliferative neoplasms, which will provide high-quality real data and serve as a basis for the application to future projects and elaboration of public policies in the medium and long term.

Epidemiological and Clinical-pathological Factors of Philadelphia-negative Myeloproliferative Neoplasms in Ecuador

Participants were followed-up for 8 years. This is the number of participants who have had Philadelphia-negative Myeloproliferative Neoplasms in Ecuador during the time of observation.

Calculation of the prevalence of participants who have had Philadelphia-negative Myeloproliferative Neoplasms in Ecuador during the time of observation.

The Dynamic International Prognostic Scoring System (DIPSS) was proposed and validated by Passamonti and is used as a prognostic model to predict survival in participants with Primary Myeloid Fibrosis a Philadelphia-negative myeloproliferative neoplasm.

Possible scores range from 0 to 6, with higher scores indicating higher mortality risk.

Percent of participants with Primary Myeloid Fibrosis who were alive at 96 months of follow-up

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
CALR Exon 9 Mutant Peptide Vaccine to Patients With CALR-mutant Myeloproliferative Neoplasms	NCT03566446	Myeloproliferat... Essential Throm... Myelofibrosis	CALRLong36 pept...	18 Years -	Herlev Hospital
The Patient Cohort of the National Center for Precision Medicine in Leukemia	NCT05326919	Acute Myeloid L... Acute Lymphobla... High-risk Myelo... Secondary Myelo... Myeloproliferat...	Biobanking	-	Assistance Publique - Hôpitaux de Paris
CALR Exon 9 Mutant Peptide Vaccine to Patients With CALR-mutant Myeloproliferative Neoplasms	NCT03566446	Myeloproliferat... Essential Throm... Myelofibrosis	CALRLong36 pept...	18 Years -	Herlev Hospital
Epidemiological and Clinical-pathological Factors of Philadelphia-negative Myeloproliferative Neoplasms in Ecuador	NCT05825326	Myeloproliferat... Myeloproliferat... Myeloproliferat... Myeloproliferat... Myeloproliferat...		18 Years -	Sociedad de Lucha Contra el Cáncer del Ecuador
CALR Exon 9 Mutant Peptide Vaccine to Patients With CALR-mutant Myeloproliferative Neoplasms	NCT03566446	Myeloproliferat... Essential Throm... Myelofibrosis	CALRLong36 pept...	18 Years -	Herlev Hospital
Epidemiological and Clinical-pathological Factors of Philadelphia-negative Myeloproliferative Neoplasms in Ecuador	NCT05825326	Myeloproliferat... Myeloproliferat... Myeloproliferat... Myeloproliferat... Myeloproliferat...		18 Years -	Sociedad de Lucha Contra el Cáncer del Ecuador

Spots Global Cancer Trial Database for myeloproliferative neoplasm, unclassifiable