Clinical Trials

The QOL-ONE Association develops and manages Multicenter National and International Clinical Trials to evaluate new treatments for patients with hematologic diseases. The clinical objectives of the studies include efficacy – in terms of response to treatment, survival and patient’s subjective measure (quality of life and symptoms -Patient Reported Outcomes) – and safety (assessment of adverse events associated with treatments). 

QOL-ONE also performs observational and translational studies. Further, it develops new instruments for the evaluation of patient-reported outcomes in hematological patients.

The trials and projects involve hematologists, patients and advocacy groups throughout the world.

QOL-ONE is technically supported by Dielnet s.r.l.  (Contract Reasearch Organization)

ONGOING CLINICAL TRIALS

QoLESS AZA-AMLE: “A Randomized Study to Evaluate the Efficacy of 5-Azacitidine for Post-Remission Therapy of Acute Myeloid Leukemia in Elderly Patients.”

EudraCT Number: 2010-019710-24

https://www.clinicaltrialsregister.eu/ctr-search/trial/2010-019710-24/IT#A

Italian, randomized, prospective, open-label, multicenter, phase III study. The aim of the study is to evaluate the effects of the therapy with the injectable azacitidine for the maintenance of complete remission in elderly patients with acute myeloid leukemia. This disease is characterized by a scarce response to treatments and early relapses inpatients in remission, leading to a short survival.

Patients in complete remission following the conventional induction and consolidation chemotherapy treatment, are randomized for  5-Azacitidine (experimental arm) or best supportive care (control arm).

Objective of the study are to evaluate the difference in disease-free survival (DFS), in the overall survival, in adverse events and in the in changes in QoL scores from diagnosis at 2 and 5 years between 5-Azacitidine and best supportive care arms.

Participating centers:

Italy

EQol MDS  “Eltrombopag for the treatment of thrombocytopenia due to low- and intermediate risk myelodysplastic syndromes.”

Eudract number 2010-022890-33

https://www.clinicaltrialsregister.eu/ctr-search/search?query=EQOL-MDS

International multicenter, single-blind, randomized, placebo-controlled, study.

In patients myelodysplastic syndrome (MDS) with thrombocytopenia and low- or intermediate-1 IPSS risk with severe piastrinopenia there is an increased hemorrhagic risk.

In Europe The only available treatment for severe thrombocytopenia, in the presence of bleeding, is platelet (PLT) transfusions, and in few cases the allogeneic bone marrow transplantation, while in the USA it is possible to offer azacitidine treatment.

Objectives of the study are to assess the platelet response rate in the proportion of patients in the experimental arm with eltrombopag versus the patients in the placebo controlled arm. In addition will be assessed the safety and tolerability in terms of frequency and severity of adverse events, quality of life and overall survival in the two groups.

To see the preliminary result of the study, please see the following article:

Eltrombopag versus placebo for low-risk myelodysplastic syndromes with thrombocytopenia (EQoL-MDS): phase 1 results of a single-blind, randomised, controlled, phase 2 superiority trial. Lancet Haematol. 2017 Mar;4(3):e127-e136. doi: 10.1016/S2352-3026(17)30012-1. Epub 2017 Feb 3.

https://www.ncbi.nlm.nih.gov/pubmed/28162984

Participating centers:

Italy

Germany

QOL-ONE Trans-1 “Accuracy of alternative TP53 somatic mutational and expression analyses for the prognostication of myelodysplastic syndrome”

National, multicenter, non-interventional, prospective, observational trial.

The prognosis of patients with isolated del5q MDS is and partly dependent on the mutation of the TP53 gene in the neoplastic clone. An accurate laboratory methodology for the prognosis of these patients is useful for the choice of the treatment, especially in patients with MDS with severe prognosis del5q.

The primary objective of the study is to evaluate the concordance between TP53 expression in IHC and in qPCR realtime relative method on BM blood sample, in terms of proportion of patients with concordance. Will be also evaluate the frequency of TP53 somatic mutations in the MDS sample by SNP single assay; the prognostic value on disease progression of the mutational status on BM blood sample compared to the expression in IHC and in qPCR realtime relative method on BM blood sample; the prognostic value on overall survival of the mutational status on BM blood sample compared to the expression in IHC and in qPCR realtime relative method on BM blood sample

Participating centers:

Italy