PREVALENCE STUDY OF PNH CLONES IN PATIENTS WITH NEOPLASIES

Multicenter prevalence study of the PNH clone (paroxysmal hemoglobinuria nocturnal) in SMP Ph-. This multicenter, prospective study aims to evaluate the presence of an PNH clone in patients with a confirmed diagnosis of myeloproliferative neoplasia Phcon or without mutations in the 3 main genes involved in this disease (JAK2, MPL, and Cal-R),but showing signs of ongoing hemolysis or particular clinical conditions.

To this end, a multicolor flow cytometric test will be used to evaluate the presence of deficient GPI molecules in granulocytic, monocytic and other cells erythrocyte (flow cytometric test, based on the use of the FLAER reagent in peripheral blood samples).

The study will be conducted at clinical hematology centers in the wider area of the Romagna and at other Italian hematology clinical centers, where and analyzed the peripheral blood samples and clinical data to be included in the study.

The participating centers will carry out the flow cytometric diagnostic test at i own reference laboratories, while the biological material for subsequent studies genetic-molecular type (next generation sequencing) will be analyzed centrally at the Biosciences laboratory of the IRST IRCCS only for cases testing positive for the presence of the PNH clone. Clinical information will be collected for each patient enrolled in the study necessary for the classification of the case and all the laboratory data necessary for achievement of the objectives of the study. The main objective of the study is to evaluate the prevalence of PNH clones in patients with a confirmed diagnosis of myeloproliferative neoplasm Ph- with or without mutations affecting the 3 main genes involved in this disease (JAK2, MPL, e Cal-R), but showing signs of ongoing hemolysis or particular clinical conditions.

Secondary objectives of the study are:

- correlate the characteristics of the PNH clone with the clinical characteristics and laboratory of myeloproliferative neoplasms Ph- (the presence of phenomena thrombotics, the disease state, the DIPSS prognostic score index, and the state mutational).

· characterize the genomic architecture of the cases using NGS technology positive results

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Key information

Conditions

Myeloproliferative Neoplasm Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases Myeloproliferative Disorders

Age range

18 year–100 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Ausl Della Romagna

Ravenna, 48121, Italy

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Diagnosis of one of the following Ph-negative myeloproliferative neoplasms:

polycythemia vera, essential thrombocythemia, idiopathic myelofibrosis, according to i WHO 2016 criteria

Women or men, age ≥ 18 years.
Willingness and ability to give written informed consent and adhere to study procedures.
Availability of the result of the following molecular tests:

JAK2-V617F mutation,
JAK2 exon 12 in PV (if JAK2 negative) Cal-R, and MPL in MFI and TE (if JAK2 negative)
Obvious signs of hemolysis (defined as LDH≥1.5 x ULN, reduced haptoglobin, reticulocytosis) when not related to the underlying disease or significantly changed compared to baseline values OR Detection of a thrombotic event (subsequent to diagnosis) OR Appearance or worsening of anemia OR Symptom change (appearance or worsening of signs or symptoms) in a stable disease context.

Treatment and study plan

Primary outcomes

the prevalence of PNH clones

Time frame: 2022

evaluate the prevalence of PNH clones in patients with a confirmed diagnosis of myeloproliferative neoplasm Ph- with or without mutations affecting the 3 main genes involved in this disease (JAK2, MPL, e Cal-R), but showing signs of ongoing hemolysis or particular clinical conditions.

Sponsors and collaborators

Lead sponsor

AUSL Romagna Rimini

Other

Registry information

Official study title

PREVALENCE STUDY OF PNH CLONES IN PATIENTS WITH NEOPLASIES MYELOPROLIFERATIVE PHILADELPHIA-NEGATIVE

Important dates

Study start: 2017
Primary completion: 2021
Study completion: 2022
First posted: Dec 7, 2023
Registry last updated: Dec 7, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed