The study will include two groups of patients with low and intermediate 1 risk myelodysplastic syndrome. One group consists of patients who experience an hematological response while on deferasirox therapy while the other group consists of patients who are non-responders
Conditions
Sponsors
Novartis Pharma NV
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Written informed consent obtained prior to any other study procedure, 2. Males or females = 18 years of age, 3. MDS according to WHO criteria lasting = 14 weeks at the time of screening, 4. IPSS score =65 years) yes F.1.3.1 Number of subjects for this age range 20 ;Inclusion criteria: 1. Written informed consent obtained prior to any other study procedure, 2. Males or females = 18 years of age, 3. MDS according to WHO criteria lasting = 14 weeks at the time of screening, 4. IPSS score =65 years) yes F.1.3.1 Number of subjects for this age range 20 ;Inclusion criteria: 1. Written informed consent obtained prior to any other study procedure, 2. Males or females = 18 years of age, 3. MDS according to WHO criteria lasting = 14 weeks at the time of screening, 4. IPSS score =65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: 1. Known concomitant presence of anemia due to iron, B12 or folate deficiencies, auto-immune or hereditary hemolysis, gastro-intestinal bleeding, or medication induced anemia at the time of screening, 2. Known infection with viral hepatitis B (HBV) or hepatitis C (HCV) defined as the presence in blood of HBV antigens in absence of HB antibodies, or presence of HCV antibodies at the time of screening, 3. Known positivity to human immunodeficiency virus (HIV) measured by enzyme-linked immunosorbent assay (ELISA) or western blot at the time of screening, 4. Patient participating in another clinical trial or receiving an investigational drug, within 1 month prior to study inclusion 5. History of other malignancy within the last five years, with the exception of basal skin carcinoma or cervical carcinoma in situ or completely resected colonic polyps carcinoma in situ. 6. Concomitant treatment with other drugs known or suspected to elicit hematological response (azacitidine, erythroid stimulating agents, granulocyte colony stimulating factors, lenalidomide, thalidomide, valproate, ATG, cyclosporine, arsenic trioxide). When patients are still receiving red blood cell transfusions, patients are still eligible for study inclusion as long as they meet the IWG criteria of 2006 7. Female patients who are pregnant or breast feeding. ;Exclusion criteria: 1. Known concomitant presence of anemia due to iron, B12 or folate deficiencies, auto-immune or hereditary hemolysis, gastro-intestinal bleeding, or medication induced anemia at the time of screening, 2. Known infection with viral hepatitis B (HBV) or hepatitis C (HCV) defined as the presence in blood of HBV antigens in absence of HB antibodies, or presence of HCV antibodies at the time of screening, 3. Known positivity to human immunodeficiency virus (HIV) measured by enzyme-linked immunosorbent assay (ELISA) or western blot at the time of screening, 4. Patient participating in another clinical trial or receiving an investigational drug, within 1 month prior to study inclusion 5. History of other malignancy within the last five years, with the exception of basal skin carcinoma or cervical carcinoma in situ or completely resected colonic polyps carcinoma in situ. 6. Concomitant treatment with other drugs known or suspected to elicit hematological response (azacitidine, erythroid stimulating agents, granulocyte colony stimulating factors, lenalidomide, thalidomide, valproate, ATG, cyclosporine, arsenic trioxide). When patients are still receiving red blood cell transfusions, patients are still eligible for study inclusion as long as they meet the IWG criteria of 2006 7. Female patients who are pregnant or breast feeding. ;Exclusion criteria: 1. Known concomitant presence of anemia due to iron, B12 or folate deficiencies, auto-immune or hereditary hemolysis, gastro-intestinal bleeding, or medication induced anemia at the time of screening, 2. Known infection with viral hepatitis B (HBV) or hepatitis C (HCV) defined as the presence in blood of HBV antigens in absence of HB antibodies, or presence of HCV antibodies at the time of screening, 3. Known positivity to human immunodeficiency virus (HIV) measured by enzyme-linked immunosorbent assay (ELISA) or western blot at the time of screening, 4. Patient participating in another clinical trial or receiving an investigational drug, within 1 month prior to study inclusion 5. History of other malignancy within the last five years, with the exception of basal skin carcinoma or cervical carcinoma in situ or completely resected colonic polyps carcinoma in situ. 6. Concomitant treatment with other drugs known or suspected to elicit hematological response (azacitidine, erythroid stimulating agents, granulocyte colony stimulating factors, lenalidomide, thalidomide, valproate, ATG, cyclosporine, arsenic trioxide). When patients are still receiving red blood cell transfusions, patients are still eligible for study inclusion as long as they meet the IWG criteria of 2006 7. Female patients who are pregnant or breast feeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To identify differentially expressed genes in baseline bone marrow samples of low and intermediate-1 risk MDS patients with a hematologic response vs non-responder patients based on NGS of the whole transcriptome to search for a predictive gene signature.;Secondary Objective: Objective 1: To assess the mean time between the initiation of treatment of deferasirox and the emergence of hematological response in patients with low and intermediate-1 risk MDS in the responder group Objective 2: To evaluate treatment related changes of iron parameters (serum ferritin, transferrin, transferrin saturation) in responders versus non-responders Objective 3: To evaluate the effect of the differential deferasirox dosing on iron and hematological parameters in responders versus non-responders;Primary end point(s): Descriptive list of differentially expressed genes from responders versus non-responders.;Timepoint(s) of evaluation of this end point: after at least 3 months of treatment with deferasirox in patients experiencing a hematological response after at least 9 months of treatment with deferasirox in patients not experiencing an hematological response in order to exclude late responders (most responses arise between 3 and 9 months after treatment start with deferasirix);Main Objective: To identify differentially expressed genes in baseline bone marrow samples of low and intermediate-1 risk MDS patients with a hematologic response vs non-responder patients based on NGS of the whole transcriptome to search for a predictive gene signature.;Secondary Objective: Objective 1: To assess the mean time between the initiation of treatment of deferasirox and the emergence of hematological response in patients with low and intermediate-1 risk MDS in the responder group Objective 2: To evaluate treatment related changes of iron parameters (serum ferritin, transferrin, transferrin saturation) in responders versus non-responders Objective 3: To evaluate the effect of the dif | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Endpoint 1: The time to response is defined as the time (in months) between the date of deferasirox initiation and the date of the first documented hematological response only in the responder group. Endpoint 2: Changes in serum ferritin levels, serum transferrin levels, transferrin saturation levels from baseline to time of response (responder group) or time to last follow up (non-responders) Endpoint 3: Deferasirox dose is defined as the average daily dose (mg/kg/d) given to the patient from treatment initiation to the emergence of hematological response in the responder group or the time of enrollment in the study in the non-responder group.;Timepoint(s) of evaluation of this end point: Endpoint 1: at least after 3 months after treatment initiation with deferasirox Endpoint 2: * for responder patients: at least after 3 months after treatment initiation with deferasirox * for non-responder patients: at least after 9 months after treatment initiation with deferasirox Endpoint 3: * for responder patients: at least after 3 months after treatment initiation with deferasirox * for non-responder patients: at least after 9 months after treatment initiation with ;Secondary end point(s): Endpoint 1: The time to response is defined as the time (in months) between the date of deferasirox initiation and the date of the first documented hematological response only in the responder group. Endpoint 2: Changes in serum ferritin levels, serum transferrin levels, transferrin saturation levels from baseline to time of response (responder group) or time to last follow up (non-responders) Endpoint 3: Deferasirox dose is defined as the average daily dose (mg/kg/d) given to the patient from treatment initiation to the emergence of hematological response in the responder group or the time of enrollment in the study in the non-responder group.;Timepoint(s) of evaluation of this end point: Endpoint 1: at least after 3 months after treatment initiation w | — |
Countries
Belgium
Contacts
Public Contact;; ;;
Novartis Pharma nv;Novartis Pharma nv;Novartis Pharma nv
Outcome results
None listed