NCT06159816

PREVALENCE STUDY OF PNH CLONES IN PATIENTS WITH NEOPLASIES

Completed Last updated 7 December 2023

What this trial tests

trial in Myeloproliferative Neoplasm in 300 participants. Completed in 1 January 2022.

Timeline

1 November 2017

Primary endpoint
1 January 2021

1 January 2022

Quick facts

Lead sponsor	AUSL Romagna Rimini
Status	Completed
Study type	OBSERVATIONAL
Enrollment	300
Start date	1 November 2017
Primary completion	1 January 2021
Estimated completion	1 January 2022
Sites	1 location across Italy

Conditions studied

• Myeloproliferative Neoplasm — all drugs for Myeloproliferative Neoplasm →

Sponsor

AUSL Romagna Rimini — full company profile →

Who can join

Adults 18 to 100, any sex, with Myeloproliferative Neoplasm. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. PNH clones prevalence study in ph-negative myeloproliferative neoplasms: a multicenter Italian study.
   D'Addio A, Rondoni M, Salvucci M, Marconi G, et al · · 2025 · cited 1× · PMID 40844514 · DOI 10.1007/s00277-025-06556-y