Previously untreated patients with diagnosis of HCL MedDRA version: 21.0 Level: PT Classification code 10019053 Term: Hairy cell leukaemia System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Conditions
Interventions
Trade Name: MABTHERA - 1 FIALA 500 MG 50 ML
Product Name: Rituximab
Product Code: [L01X- C02]
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: RITUXIMAB
CAS Number: 1747
Sponsors
DIPARTIMENTO DI MEDICINA, UNIVERSITà DI PERUGIA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Any incoming patient with untreated HCL must be screened, and must be enrolled in the study if satisfying all of the following general eligibility criteria no. 1-8 (while not meeting any exclusion criteria – see next section): 1. Previously untreated patients with centrally reviewed diagnosis of HCL 2. Need of treatment, i.e. at least one of the following: neuthrophils 38.3°C or a sustained temperature of =38°C for >1 hour) in a neutropenic patient (=65 years) yes F.1.3.1 Number of subjects for this age range 30
Exclusion criteria
Exclusion criteria: 1.Concurrent administration of other any anti-cancer therapies. Prior splenectomy for diagnosis and/or therapy of HCL is not allowed. 2. Pregnancy or lactation. 3. Other active advanced cancer with projected life expectancy <1 year.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the trial is to show that the experimental therapy (VR) is no less effective and less toxic than the standard therapy (CDAR).;Secondary Objective: a)Time from starting treatment until resolution of cytopenias; b)Time from starting treatment until recovery of CD4+ T cells, CD8+ T cells, B cells and NK cells recovery to normal values; c)Proportion of patients clearing measurable/minimal residual disease (MRD); d)Survival free from MRD; e)Survival free from relapse; f)Survival free from disease progression; g)Survival free from a subsequent anti-leukemic treatment; h)Survival free from death; i)and j) Treatment-related toxicities by central and local assessment k)Adverse events and toxicities of any grades; l)Role of PET-CT in HCL staging and response to therapy; m)Quality of life; n)Pharmaco-economic analysis of global treatment costs; o)In patients enrolled but not randomized, prospective evaluation of the efficacy and safety of any alternative treatment strategy;Primary end point(s): There are two independent primary endpoints, one for efficacy and one for safety. Primary efficacy endpoint: rate of complete remission (CR) at ~6 months after treatment initiation in randomized patients, as centrally adjudicated by a blinded external independent committee (including bone marrow biopsy). CR requires the normalization of blood counts, absence of palpable splenomegaly, and absence of hairy cells visible in the bone marrow biopsy at hematoxylin and eosin staining. Primary safety endpoint: proportion of randomized patients experiencing =1 drug-related toxicity of maximum grade =3 according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0 that occurs within 6 months from starting treatment as reported by the investigators, and that is at least possibly related to the study drugs as centrally adjudicated by another member of the external independent committee (blinded to response to treatment but not to trea | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Survival free from a subsequent anti-leukemic treatment (calculated in patients undergoing a new treatment from the end of trial therapy); Survival free from disease progression (calculated in all patients since starting treatment); Survival free from death (calculated in all patients since starting treatment); Treatment-related toxicities (total counts and proportion of patients affected) separately grouped in grade 3, grade 4 or grade 5, by central assessment; Treatment-related toxicities (total counts and proportion of patients affected) separately grouped in grade 3, grade 4 or grade 5, by local assessment; Adverse events and toxicities of any grades (total counts and proportion of patients affected), by local assessment; Proportion of patients clearing measurable/minimal residual disease (MRD) by PCR and/or flow cytometry and/or immunohistochemistry in bone marrow and blood cell samples, as well as by PCR in the plasma (liquid biopsy); Survival free from MRD (calculated in patients clearing MRD from the time of MRD clearing); Survival free from relapse (calculated in patients obtaining a OR from the time of OR achievement); Role of PET-CT in HCL staging and response to therapy; Quality of life, as quantified through ad hoc questionnaire; Pharmaco-economic analysis of global treatment costs; Time from starting treatment until recovery of CD4+ T cells, CD8+ T cells, B cells and NK cells recovery to normal values; In patients enrolled but not randomized due to concern(s) against the standard and/or experimental treatment (including, but not limited to, active severe infection or risk thereof, including risk of severe Covid19 in unvaccinated patients; renal or hepatic impairment; severe QTc prolongation; history of an aggressive cancer type frequently associated with RAS mutations that was unlikely eradicated, lack of the BRAF-V600E mutation; etc.), prospective evaluation of the efficacy and safety of any alternative treatment strategy chose | — |
Countries
Italy
Contacts
Public ContactS. Ematologia e Immunologia clinica
Dipartimento di Medicina e Chirurgia, Università degli studi di Perugia
Outcome results
None listed