IRCCS Candiolo - Sindromi mielodisplastiche

MDS del5q low-intermediate IPSS R

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Linea terapeutica: Refractory or intollerant to lenalidomide

Coorti: Single ARM Phase 2

Terapia: Luspatercept

Inclusion:

Documented diagnosis of MDS with del5q according to 2018 WHO classification
IPSS-R classification (Greenberg, 2012) of very low, low, or intermediate risk disease, and: < 5% blasts in bone marrow and Peripheral blood WBC count <13,000/μL
Refractory or intolerant to, or ineligible for, prior ESA treatment
Refractory or intolerant to, or ineligible for, prior lenalidomide treatment

Exclusion:

Previously treated with either luspatercept (ACE-536) or sotatercept (ACE-011)
Secondary MDS, ie, MDS that is known to have arisen as the result of chemical injury or treatment with chemotherapy and/or radiation for other diseases.
Known clinically significant anemia due to iron, vitamin B12, or folate deficiencies, or autoimmune or hereditary hemolytic anemia, or gastrointestinal bleeding
Prior allogeneic or autologous stem cell transplant

MDS low-intermediate IPSS R with anemia TD or non TD

Linea terapeutica: or not candidate to prior ESA treatment /luspatercept/lenalidomide

Coorti: Single ARM Phase 2

Terapia: RVU120

Inclusion:

Very low, low or intermediate risk disease MDS with up to 3.5 points according to International
Prognostic Scoring System Score Revised (IPSS-R) classification (to be confirmed during screening assessment).
Patients with del(5q) and max. one further abnormality (excluding monosomy 7, del(7q), TP53mut) are eligible.
No available option of an approved MDS therapy according to decision of the treating physician and based on the following:
Patients must be ESA exposed (and refractory or intolerant) or ESA naïve and serum erythropoietin level >200 U/L
AND/OR
Luspatercept exposed (and refractory or intolerant) or luspatercept naïve and not eligible for treatment (e.g. not approved)
AND/OR
Lenalidomide exposed (and refractory or intolerant) or lenalidomide naïve and not eligible for treatment (e.g. due to non-presence of del(5q))

Exclusion:

Patient does not accept bone marrow sampling during screening and after the treatment.
Prior treatment with azacitidine (injectable or oral) or decitabine.
Iron chelation therapy

MDS low-intermediate IPSS R with transfusion dependent anemia

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Linea terapeutica: Refractory or intolerant to prior ESA treatment

Coorti: Sperimental Arm Terapia: Elritercept (KER-050)

Coorti: Control Arm Terapia: Placebo

Inclusion:

Refractory or intolerant to prior ESA treatment (discontinued ≥ 8 weeks before randomization), or unlikely to respond to ESA treatment
Less than 5% blasts in an evaluable bone marrow aspirate collected at Screening, read by an independent central reader

Exclusion:

Del(5q) MDS or secondary MDS.
Anemia due to any other known cause (e.g., thalassemia, hemolytic anemia, bleeding events, or deficiency of iron, B12, and/or folate).
Receipt of RBC transfusion for any reason(s) other than underlying MDS within 16 weeks before randomization.
Prior lifetime use of TGF-β–modulating therapy, including luspatercept

higher-risk chronic myelomonocytic leukemia (CMML)

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Linea terapeutica: I linea

Terapia: azacitidine (AZA) combined with venetoclax (VEN) and tagraxofusp (TAG)

Coorti: Single ARM Phase 2

Inclusion:

CPSS risk intermediate-2 or high at study entry
No prior HMA or VEN treatment (acceptable prior treatment with ESA or HY, with a >15 and > 3 days washout, respectively)
ECOG PS 0-2

Exclusion:
previously received HSCT