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Connect® MDS/AML Disease Registry

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ClinicalTrials.gov Identifier: NCT01688011
Recruitment Status : Recruiting
First Posted : September 19, 2012
Last Update Posted : October 25, 2018
Sponsor:
Information provided by (Responsible Party):
Celgene

Brief Summary:
The purpose of the Connect® MDS/AML Disease Registry is to provide unique insights into treatment regimens and sequencing of these regimens as they relate to clinical outcomes of patients with newly diagnosed MDS, ICUS or AML in routine clinical practice and evaluate molecular and cellular markers that may provide further prognostic classification and/or might be predictive of therapy outcomes.

Condition or disease
Myelodysplastic Syndromes Acute Myeloid Leukemia

Detailed Description:
This Disease Registry will collect data on patient characteristics, treatment patterns and clinical outcomes. The objective is to describe how newly diagnosed MDS, ICUS or AML patients are treated; and to build a knowledge base regarding the effectiveness and safety of front-line and subsequent treatment regimens in both community and academic settings. Enrolled patients will receive treatment and evaluations for MDS, ICUS or AML according to the standard of care and routine clinical practice at each study site. All treatments that patients receive for MDS, ICUS or AML 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.

Study Type : Observational
Estimated Enrollment : 1500 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Connect® MDS/AML: The Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) Disease Registry
Study Start Date : December 12, 2013
Estimated Primary Completion Date : December 12, 2020
Estimated Study Completion Date : December 12, 2028


Group/Cohort
Lower-Risk Myelodysplastic Syndromes (LR MDS)
Newly diagnosed lower risk MDS patients as determined by International Prognostic Scoring System (IPSS).
Higher-Risk Myelodysplastic Syndromes (HR MDS)
Newly diagnosed higher risk MDS patients as determined by International Prognostic Scoring System (IPSS).
Acute Myeloid Leukemia (AML)
Newly diagnosed AML patients (≥55 years old, excluding patients with acute promyelocytic leukemia (APL).
Idiopathic Cytopenia of Undetermined Significance (ICUS)
Newly diagnosed ICUS patients as determined by clinical criteria defined by Valent et al. (Valent P, Horny H-P, Bennett JM et al. 2007. Definitions and standards in the diagnosis and treatment of the myelodysplastic syndromes: Consensus statements and report from a working conference. Leukemia Research. 31: 727-736. And further updated Valent P, Bennett JM et al. 2017. Proposed minimal diagnostic criteria for myelodysplastic syndromes (MDS) and potential pre-MDS conditions. Oncotarget. 8(43): 73483-73500.)



Primary Outcome Measures :
  1. Patient Demographics [ Time Frame: Up to 8 years ]
    Describe patient demographics and clinical outcomes of patients with LR or HR MDS, ICUS, and AML

  2. Diagnostic and Treatment Patterns [ Time Frame: Up to 8 years ]
    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

  3. Safety and Effectiveness [ Time Frame: Up to 8 years ]
    Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events.


Secondary Outcome Measures :
  1. Patient Reported Outcome [ Time Frame: Up to 8 years ]
    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

  2. Correlative Studies [ Time Frame: Up to 8 years ]
    Perform molecular and cellular correlative studies on blood/bone marrow and oral epithelial cell samples.


Biospecimen Retention:   Samples With DNA
Perform molecular and cellular correlative studies on blood/bone marrow and oral epithelial cell samples.


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Approximately 1,500 patients across approximately 150 sites throughout the US will be enrolled in the Connect® MDS and AML Registry. Sites will include both community-based and academic centers that are representative of where patients with MDS and AML are diagnosed and treated. To best capture the distribution of sites with regard to the settings of where MDS and AML patients are typically treated in routine practice, approximately 70-80% of the sites will be community hematology/ oncology clinics and approximately 20-30% will be academic-based institutions.
Criteria

Inclusion Criteria:

  • Patients must be able to provide written informed consent
  • Newly diagnosed (confirmed diagnosis within 60 days prior to date of informed consent signature), primary or secondary Myelodysplastic Syndromes (MDS), or Acute Myeloid Leukemia (AML), or Idiopathic Cytopenia of Undetermined Significance (ICUS)
  • Disease diagnosis confirmed by Central Eligibility Review
  • AML patients must be at least 55 years of age at the time of informed consent signature
  • MDS/ICUS patients must be at least 18 years of age at the time of informed consent signature
  • Patients must be willing and able to complete enrollment and follow-up HRQoL instruments, for which patients must be proficient in either English or Spanish

Exclusion Criteria:

  • Suspected or proven acute promyelocytic leukemia (APL) (FAB M3 or WHO 2008) based on morphology, immunophenotype, molecular assay or karyotype
  • MDS, AML or ICUS cohort assignment by Central Eligibility Review is not confirmed by site
  • For MDS and ICUS patients: receiving active (disease modifying) treatment** prior to ICF date (Supportive care such as transfusions, antibiotics, iron chelators, EPO, growth factors (G-CSF/GM-CSF) is allowed)
  • For AML patients: receiving active (disease modifying) treatment** that had been initiated for more than 2 weeks (14 days) prior to ICF date (Supportive care, such as, transfusions, antibiotics, iron chelators, EPO, growth factors (G-CSF/GM-CSF), tumor lysis prophylaxis allowed)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01688011


Contacts
Contact: Melissa Nifenecker 908-219-0809 connectmdsaml-registry@celgene.com

  Show 219 Study Locations
Sponsors and Collaborators
Celgene
Investigators
Study Director: Chrystal Louis, MD Celgene Corporation

Publications:
Responsible Party: Celgene
ClinicalTrials.gov Identifier: NCT01688011     History of Changes
Other Study ID Numbers: Connect® MDS/AML Registry
First Posted: September 19, 2012    Key Record Dates
Last Update Posted: October 25, 2018
Last Verified: October 2018

Keywords provided by Celgene:
Myelodysplastic Syndromes
MDS
Acute Myeloid Leukemia
AML
Registry
Connect®

Additional relevant MeSH terms:
Syndrome
Leukemia
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Preleukemia
Disease
Pathologic Processes
Neoplasms by Histologic Type
Neoplasms
Bone Marrow Diseases
Hematologic Diseases
Precancerous Conditions