Registry of Philadelphia-Negative Myeloproliferative Neoplasms

NCT ID: NCT02380378

Last Updated: 2019-04-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Total Enrollment

500 participants

Study Classification

OBSERVATIONAL

Study Start Date

2015-03-31

Study Completion Date

2025-04-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This study was developed to document current diagnosis and treatment patterns, clinical outcomes, and health care resource use associated with Philadelphia-Negative Myeloproliferative Neoplasms, in the different risk classifications for each disease.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Philadelphia-negative (Ph-negative) Myeloproliferative Neoplasms (MPN) include polycythemia vera (PV), essential thrombocythemia (TE), and primary myelofibrosis (MF). Ph-negative MPN are chronic myeloid neoplasms that appear after a malignant transformation of a hematopoietic stem cell. There are many clinical manifestations of these diseases, which include polyglobulia, thrombocytosis, leukocytosis, cytopenias, extramedullary hematopoiesis (e.g.: splenomegaly), increased thrombotic risk and the risk of transformation to acute myeloid leukemia (AML). Life expectancy varies from 5-6 years for patients with MF to more than 15 years for patients with PV and ET.

There are few therapeutic options for patients with Ph-negative MPN. In general, the treatment of these diseases is symptomatic and targeted to the relief of symptoms and control of hematological setting using oral chemotherapy agents such as hydroxyurea. Patients with MF receive palliative treatment, targeted to the relief of splenomegaly and cytopenias. Currently, there are no approved drugs for the treatment of MF.

In Latin America, there are no epidemiological studies with a great number of Ph-negative MPN patients describing the natural history of the disease, patient progress, clinical characteristics, standards of care and disease burden. The development of an up-to-date data registry of Brazilian patients with Ph-negative MPN allows the comprehension of the epidemiology of this disease, with determination of survival, thrombosis incidence, transformation to AML, use of disease treatment-related resources, as well as the impact of different therapeutic strategies in these parameters. Therefore, the objective of this study is to establish a registry of patients with Ph-negative MPN in order to document the disease diagnosis and current standards of care, clinical endpoints, and the use of treatment-related resources, according to risk classification in each MPN.

Data will be retrospectively and prospectively collected through an electronic form available at an Internet safe website. Each center participating in the study will have a principal investigator who will be in charge of the accuracy and quality of the data collected. Each principal investigator and staff will receive a login and a password to access the database and enroll patients in the registry. An employee from each site will be in charge of collecting the data and inserting them in the electronic registry. Patients shall be contacted in order to sign an informed consent form. Patient data will be updated every six months. There is no limit for the number of patients to be enrolled in the registry, either retrospectively or prospectively. The duration of this study is five years. Data collection will continue until all sites decide to interrupt the enrollment of new patients.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hematologic Diseases

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Myeloproliferative Neoplasms

Patients diagnosed with Myeloproliferative Neoplasms based on WHO 2008 criteria.

No interventions assigned to this group

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* diagnosis of (according to WHO 2008 criteria) Polycythemia Vera, Essential Thrombocythemia, Primary Myelofibrosis or Post-Polycythemia Vera/Post-Essential Thrombocythemia Myelofibrosis, Unclassifiable Myeloproliferative Neoplasm, Chronic Neutrophilic Leukemia, Chronic Eosinophilic Leukemia, Systemic Mastocytosis
* diagnosis made since 2000
* patients who are being followed-up or not
* signed informed consent form (for alive patients included in the prospective step of the study).

Exclusion Criteria

* diagnosis of Chronic Myelogenous Leukemia
* refusal to sign the informed consent form (for patients who are alive and will de included in the prospective step of the study.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Hospital Santa Marcelina

OTHER

Sponsor Role collaborator

Instituto Nacional de Cancer, Brazil

OTHER_GOV

Sponsor Role collaborator

Federal University of Minas Gerais

OTHER

Sponsor Role collaborator

Irmandade da Santa Casa de Misericordia de Sao Paulo

OTHER

Sponsor Role collaborator

Federal University of São Paulo

OTHER

Sponsor Role collaborator

Hospital Israelita Albert Einstein

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Fabio Pires de Souza Santos

Fabio Pires de Souza Santos

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Fabio P Santos

Role: PRINCIPAL_INVESTIGATOR

Instituto Israelita de Ensino e Pesquisa Albert Einstein´s (IIEP)

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Hospital Israelita Albert Einstein

São Paulo, São Paulo, Brazil

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Brazil

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Fabio P Santos

Role: CONTACT

Tarcila S Datoguia

Role: CONTACT

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Fabio P S Santos, MD

Role: primary

+551121511128

Tarcila Datoguia, MD

Role: backup

+551121511128

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

RMPN2015

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Registry of Patients With MPNs in Taiwan
NCT03618485 ACTIVE_NOT_RECRUITING
A New Blood Score for Myelofibrosis Staging
NCT06177366 NOT_YET_RECRUITING