Brief Summary
The purpose of the Connect® Myeloid disease registry is to provide unique insights into treatment decisions and treatment patterns as they relate to clinical outcomes of patients with myeloid diseases in routine clinical practice. This disease registry will also evaluate molecular and cellular markers that may provide further prognostic classification which may or may not be predictive of therapy and clinical outcomes.
Brief Title
Connect® Myeloid Disease Registry
Detailed Description
This Disease Registry will collect data on patient characteristics, treatment patterns and clinical outcomes. The objective is to describe how patients with myeloid diseases are treated; and to build a knowledge base regarding the effectiveness and safety of first line and subsequent treatment regimens in both community and academic settings. Enrolled patients will receive treatment and evaluations for their disease according to the standard of care and routine clinical practice at each study site. All treatments that patients receive for their disease will be recorded, including initial treatment and any subsequent therapy. Data on treatment outcomes, including response rates as measured by the treating physician, evidence of progression, survival, and patient-reported outcomes will be collected quarterly on the electronic CRF.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Primary Myelofibrosis
Myelodysplastic Syndromes
Leukemia, Myeloid, Acute
Eligibility Criteria
Inclusion Criteria:
* Patients must be able to provide written informed consent form (ICF)
* Must be willing and able to complete baseline and follow-up HRQoL instruments, for which patients must be proficient in either English or Spanish
* AML patients must be at least 55 years of age at the time of informed consent.
* MF, ICUS, and MDS patients must be at least 18 years of age at the time of informed consent.
Newly diagnosed Idiopathic Cytopenias of Undetermined Significance (ICUS), Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML) patients:
* Newly diagnosed primary or secondary disease. To be considered "newly diagnosed", a patient's confirmed diagnosis must be made no more than 60 days prior to the date of consent signature. (An additional 5-day window \[i.e., up to 65 days prior to the date of ICF signature\] may be allowed in special circumstance upon sponsor approval)
* Cohort assignment confirmed by central eligibility review. Cohort assignment must also be confirmed by the site.
Myelofibrosis (MF) patients:
* Patients who initiated their first active systemic treatment for MF and/or MF-related cytopenias within 90 days prior to the date of consent signature. This cohort allows the enrollment of subjects with a diagnosis of Myelodysplastic/Myeloproliferative overlap syndromes (MDS/MPN overlap syndrome).
* Cohort assignment is confirmed by the site. Central eligibility review is not required.
Treated Lower-Risk Myelodysplastic Syndromes (LR-MDS) patients:
* Patients who have initiated first active treatment regimen containing at least one non-ESA therapy, within 90 days prior to ICF
* Cohort assignment is confirmed by site. Central eligibility review is not required.
Luspatercept treated patients:
* Patient must have been at least 18 years of age at the start of luspatercept.
* Among LR-MDS patients, patient must have initiated luspatercept on or after September 1, 2023, must be ESA-naïve and luspatercept must be the first active treatment (as monotherapy or part of a treatment regimen) for their disease.
* Among all other myeloid malignancies, there is no date restriction for initiation of luspatercept .Patient may have received prior treatment for their disease.
* Patient must have at least 3 months of follow-up from start of luspatercept treatment at the participating site.
Exclusion Criteria:
* Suspected or proven acute promyelocytic leukemia (APL) (FAB M3 or WHO 2008) based on morphology, immunophenotype, molecular assay or karyotype
* Currently enrolled in any interventional clinical trial where the patient is being treated with an investigational product that cannot be identified.
* Idiopathic Cytopenias of Undetermined Significance (ICUS), Myelodysplastic Syndromes (MDS) patients who received or are receiving active (disease modifying) therapy for the treatment of MDS prior to the date of informed consent.
* Acute Myeloid Leukemia (AML) patients who initiated active (disease modifying treatment for AML more than 2 weeks prior to the date of consent.
* Myelofibrosis (MF) and Myelodysplastic/Myeloproliferative (MDS/MPN) overlap syndrome patients with suspected juvenile myelomonocytic leukemia (JMML).
Luspatercept treated patients:
* Patient must not be currently or previously enrolled in the Connect Myeloid Registry.
* Patient must not have received luspatercept as part of a clinical trial.
* Patients must be able to provide written informed consent form (ICF)
* Must be willing and able to complete baseline and follow-up HRQoL instruments, for which patients must be proficient in either English or Spanish
* AML patients must be at least 55 years of age at the time of informed consent.
* MF, ICUS, and MDS patients must be at least 18 years of age at the time of informed consent.
Newly diagnosed Idiopathic Cytopenias of Undetermined Significance (ICUS), Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML) patients:
* Newly diagnosed primary or secondary disease. To be considered "newly diagnosed", a patient's confirmed diagnosis must be made no more than 60 days prior to the date of consent signature. (An additional 5-day window \[i.e., up to 65 days prior to the date of ICF signature\] may be allowed in special circumstance upon sponsor approval)
* Cohort assignment confirmed by central eligibility review. Cohort assignment must also be confirmed by the site.
Myelofibrosis (MF) patients:
* Patients who initiated their first active systemic treatment for MF and/or MF-related cytopenias within 90 days prior to the date of consent signature. This cohort allows the enrollment of subjects with a diagnosis of Myelodysplastic/Myeloproliferative overlap syndromes (MDS/MPN overlap syndrome).
* Cohort assignment is confirmed by the site. Central eligibility review is not required.
Treated Lower-Risk Myelodysplastic Syndromes (LR-MDS) patients:
* Patients who have initiated first active treatment regimen containing at least one non-ESA therapy, within 90 days prior to ICF
* Cohort assignment is confirmed by site. Central eligibility review is not required.
Luspatercept treated patients:
* Patient must have been at least 18 years of age at the start of luspatercept.
* Among LR-MDS patients, patient must have initiated luspatercept on or after September 1, 2023, must be ESA-naïve and luspatercept must be the first active treatment (as monotherapy or part of a treatment regimen) for their disease.
* Among all other myeloid malignancies, there is no date restriction for initiation of luspatercept .Patient may have received prior treatment for their disease.
* Patient must have at least 3 months of follow-up from start of luspatercept treatment at the participating site.
Exclusion Criteria:
* Suspected or proven acute promyelocytic leukemia (APL) (FAB M3 or WHO 2008) based on morphology, immunophenotype, molecular assay or karyotype
* Currently enrolled in any interventional clinical trial where the patient is being treated with an investigational product that cannot be identified.
* Idiopathic Cytopenias of Undetermined Significance (ICUS), Myelodysplastic Syndromes (MDS) patients who received or are receiving active (disease modifying) therapy for the treatment of MDS prior to the date of informed consent.
* Acute Myeloid Leukemia (AML) patients who initiated active (disease modifying treatment for AML more than 2 weeks prior to the date of consent.
* Myelofibrosis (MF) and Myelodysplastic/Myeloproliferative (MDS/MPN) overlap syndrome patients with suspected juvenile myelomonocytic leukemia (JMML).
Luspatercept treated patients:
* Patient must not be currently or previously enrolled in the Connect Myeloid Registry.
* Patient must not have received luspatercept as part of a clinical trial.
Inclusion Criteria
Inclusion Criteria:
* Patients must be able to provide written informed consent form (ICF)
* Must be willing and able to complete baseline and follow-up HRQoL instruments, for which patients must be proficient in either English or Spanish
* AML patients must be at least 55 years of age at the time of informed consent.
* MF, ICUS, and MDS patients must be at least 18 years of age at the time of informed consent.
Newly diagnosed Idiopathic Cytopenias of Undetermined Significance (ICUS), Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML) patients:
* Newly diagnosed primary or secondary disease. To be considered "newly diagnosed", a patient's confirmed diagnosis must be made no more than 60 days prior to the date of consent signature. (An additional 5-day window \[i.e., up to 65 days prior to the date of ICF signature\] may be allowed in special circumstance upon sponsor approval)
* Cohort assignment confirmed by central eligibility review. Cohort assignment must also be confirmed by the site.
Myelofibrosis (MF) patients:
* Patients who initiated their first active systemic treatment for MF and/or MF-related cytopenias within 90 days prior to the date of consent signature. This cohort allows the enrollment of subjects with a diagnosis of Myelodysplastic/Myeloproliferative overlap syndromes (MDS/MPN overlap syndrome).
* Cohort assignment is confirmed by the site. Central eligibility review is not required.
Treated Lower-Risk Myelodysplastic Syndromes (LR-MDS) patients:
* Patients who have initiated first active treatment regimen containing at least one non-ESA therapy, within 90 days prior to ICF
* Cohort assignment is confirmed by site. Central eligibility review is not required.
Luspatercept treated patients:
* Patient must have been at least 18 years of age at the start of luspatercept.
* Among LR-MDS patients, patient must have initiated luspatercept on or after September 1, 2023, must be ESA-naïve and luspatercept must be the first active treatment (as monotherapy or part of a treatment regimen) for their disease.
* Among all other myeloid malignancies, there is no date restriction for initiation of luspatercept .Patient may have received prior treatment for their disease.
* Patient must have at least 3 months of follow-up from start of luspatercept treatment at the participating site.
* Patients must be able to provide written informed consent form (ICF)
* Must be willing and able to complete baseline and follow-up HRQoL instruments, for which patients must be proficient in either English or Spanish
* AML patients must be at least 55 years of age at the time of informed consent.
* MF, ICUS, and MDS patients must be at least 18 years of age at the time of informed consent.
Newly diagnosed Idiopathic Cytopenias of Undetermined Significance (ICUS), Myelodysplastic Syndromes (MDS), Acute Myeloid Leukemia (AML) patients:
* Newly diagnosed primary or secondary disease. To be considered "newly diagnosed", a patient's confirmed diagnosis must be made no more than 60 days prior to the date of consent signature. (An additional 5-day window \[i.e., up to 65 days prior to the date of ICF signature\] may be allowed in special circumstance upon sponsor approval)
* Cohort assignment confirmed by central eligibility review. Cohort assignment must also be confirmed by the site.
Myelofibrosis (MF) patients:
* Patients who initiated their first active systemic treatment for MF and/or MF-related cytopenias within 90 days prior to the date of consent signature. This cohort allows the enrollment of subjects with a diagnosis of Myelodysplastic/Myeloproliferative overlap syndromes (MDS/MPN overlap syndrome).
* Cohort assignment is confirmed by the site. Central eligibility review is not required.
Treated Lower-Risk Myelodysplastic Syndromes (LR-MDS) patients:
* Patients who have initiated first active treatment regimen containing at least one non-ESA therapy, within 90 days prior to ICF
* Cohort assignment is confirmed by site. Central eligibility review is not required.
Luspatercept treated patients:
* Patient must have been at least 18 years of age at the start of luspatercept.
* Among LR-MDS patients, patient must have initiated luspatercept on or after September 1, 2023, must be ESA-naïve and luspatercept must be the first active treatment (as monotherapy or part of a treatment regimen) for their disease.
* Among all other myeloid malignancies, there is no date restriction for initiation of luspatercept .Patient may have received prior treatment for their disease.
* Patient must have at least 3 months of follow-up from start of luspatercept treatment at the participating site.
Gender
All
Gender Based
false
Keywords
Myelodysplastic syndromes
MDS
Acute myeloid leukemia
AML
Registry
Connect®
ICUS
Idiopathic Cytopenias of Undetermined Significance
Myelofibrosis
MF
Myelodysplastic/Myeloproliferative overlap syndromes
MDS/MPN overlap syndromes
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Minimum Age
18 Years
NCT Id
NCT01688011
Org Class
Industry
Org Full Name
Celgene
Org Study Id
AZA-MDS-006
Overall Status
Terminated
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
Connect® Myeloid: The Myelofibrosis (MF), Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) Disease Registry
Primary Outcomes
Outcome Description
Describe demographics, baseline characteristics and clinical outcomes of the patients with LR or HR MDS, ICUS, and AML.
Outcome Measure
Patient Demographics- MDS/AML/ICUS Cohorts
Outcome Time Frame
Up to 8 years
Outcome Description
Describe current and evolving patterns for diagnosis, treatment sequencing, routine clinical practice patterns and clinical outcome measures in patients with LR or HR MDS, ICUS, and AML
Outcome Measure
Diagnostic and Treatment Patterns- MDS/AML/ICUS Cohorts
Outcome Time Frame
Up to 8 years
Outcome Description
Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events.
Outcome Measure
Safety and Effectiveness- MDS/AML/ICUS Cohorts
Outcome Time Frame
Up to 8 years
Outcome Description
Describe demographics, baseline characteristics, patient recorded outcomes, and clinical outcomes of patients enrolled to the MF cohort
Outcome Measure
Patient Demographics- MF Cohort
Outcome Time Frame
Up to 5 years
Outcome Description
Describe current and evolving patterns for diagnosis, treatment sequencing, routine clinical practice patterns and clinical outcome measures in patients enrolled in the MF Cohort
Outcome Measure
Diagnostic and Treatment Patterns- MF Cohort
Outcome Time Frame
Up to 5 years
Outcome Description
Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events.
Outcome Measure
Safety and Effectiveness- MF Cohort
Outcome Time Frame
Up to 5 years
Outcome Description
Describe clinical response to treatment, transfusion information, ECOG performance status and deaths.
Outcome Measure
Treatment effectiveness - LTC
Outcome Time Frame
Minimum of 3-months post index date
Outcome Description
Describe the myeloid malignancy treatment patterns and clinical outcomes before and after initiating luspatercept treatment
Outcome Measure
Treatment patterns and clinical outcomes - LTC Cohort
Outcome Time Frame
Minimum of 3-months post index date
Outcome Description
Describe changes in hemoglobin and transfusion independence status.
Outcome Measure
Transfusion information - LTC
Outcome Time Frame
Minimum of 3-months post index date
Outcome Description
Luspatercept treatment duration
Outcome Measure
Treatment duration - LTC Cohort
Outcome Time Frame
Minimum of 3-months post index date
Secondary Ids
Secondary Id
Connect® MDS/AML Registry
Secondary Outcomes
Outcome Description
Summarize patient reported outcomes (including e.g., Health-Related Quality of Life (HRQOL)) and economic outcomes, and their association with patient characteristics, treatment regimens, and clinical outcomes
Outcome Time Frame
Up to 8 years
Outcome Measure
Patient Reported Outcome
Outcome Description
Perform molecular and cellular correlative studies on blood/bone marrow and oral epithelial cell samples.
Outcome Time Frame
Up to 8 years
Outcome Measure
Correlative Studies
Outcome Description
Describe demographics, baseline characteristics and clinical outcomes of the patients treated with luspatercept
Outcome Time Frame
Baseline
Outcome Measure
Patient demographics and clinical characteristics - LTC
Outcome Description
Describe reasons for luspatercept treatment discontinuation
Outcome Time Frame
Minimum of 3-months post index date
Outcome Measure
Reason for treatment discontinuation - LTC
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Study Population
Approximately 2,300 adult patients diagnosed with myeloid disease will be enrolled in Connect® Myeloid Registry from 200 sites (both community \[70-80%\] and academic \[20-30%\] facilities) across the US that are representative of where patients with myeloid diseases are diagnosed and treated.
Std Ages
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
18
Investigators
Investigator Type
Principal Investigator
Investigator Name
Amit Verma
Investigator Email
amit.verma@einsteinmed.org
Investigator Phone
718-405-8505 / 718-904-2900
Categories Mesh Debug
Blood & Bone Marrow Cancers --- MYELODYSPLASTIC SYNDROMES
Blood & Bone Marrow Cancers --- LEUKEMIA, MYELOID, ACUTE
Blood & Bone Marrow Cancers --- MYELOPROLIFERATIVE DISORDERS
Blood & Bone Marrow Cancers --- BONE MARROW DISEASES
Blood Disorders --- HEMATOLOGIC DISEASES
Blood & Bone Marrow Cancers --- HEMATOLOGIC DISEASES
Blood & Bone Marrow Cancers --- LEUKEMIA, MYELOID
Cancer --- LEUKEMIA
Blood & Bone Marrow Cancers --- LEUKEMIA
Cancer --- NEOPLASMS
MeSH Terms
PRIMARY MYELOFIBROSIS
MYELODYSPLASTIC SYNDROMES
LEUKEMIA, MYELOID, ACUTE
MYELOPROLIFERATIVE DISORDERS
BONE MARROW DISEASES
HEMATOLOGIC DISEASES
HEMIC AND LYMPHATIC DISEASES
LEUKEMIA, MYELOID
LEUKEMIA
NEOPLASMS BY HISTOLOGIC TYPE
NEOPLASMS
LUSPATERCEPT