Connect® MDS/AML Disease Registry

• ClinicalTrials.gov Identifier: NCT01688011
• Recruitment Status: Recruiting
• First Posted: September 19, 2012
• Last Update Posted: November 2, 2020
• Sponsor: Celgene
• Information provided by (Responsible Party): Celgene

Tracking Information

• First Submitted Date: September 14, 2012
• First Posted Date: September 19, 2012
• Last Update Posted Date: November 2, 2020
• Study Start Date: December 12, 2013
• Estimated Primary Completion Date: December 12, 2022 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: October 23, 2019)

• Patient Demographics [ Time Frame: Up to 8 years ]
  Describe demographics, baseline characteristics and clinical outcomes of the patients with LR or HR MDS, MDS risk not determined, and AML.
• 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, MDS risk not determined, and AML
• 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.

Original Primary Outcome Measures (submitted: September 14, 2012)

• Patient Demographics [ Time Frame: Up to 8 years ]
  Patient Demographics
• Diagnostic and Treatment Patterns [ Time Frame: Up to 8 years ]
  Diagnostic and Treatment Patterns

Current Secondary Outcome Measures (submitted: April 18, 2013)

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

Original Secondary Outcome Measures (submitted: September 14, 2012)

• Patient Reported Outcome Measure [ Time Frame: Up to 8 years ]
  Analyze 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
• Correlative Outcome Measure [ Time Frame: Up to 8 years ]
  Perform molecular and cellular correlative studies on collected blood/bone marrow samples.

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Connect® MDS/AML Disease Registry
• Official Title: Connect® MDS/AML: The Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML) Disease Registry
• 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 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.
• 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 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 or AML according to the standard of care and routine clinical practice at each study site. All treatments that patients receive for MDS 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
• Study Design: Observational Model: Cohort; Time Perspective: Prospective
• Target Follow-Up Duration: Not Provided

Biospecimen

Retention:   Samples With DNA

Description:

Perform molecular and cellular correlative studies on blood/bone marrow and oral epithelial cell samples.

• Sampling Method: Non-Probability Sample
• Study Population: Approximately 2,100 patients across approximately 150-200 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.

Condition

• Myelodysplastic Syndromes
• Acute Myeloid Leukemia

• Intervention: Not Provided

Study Groups/Cohorts

• 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).
• Unknown-Risk MDS
  Newly diagnosed unknown-risk MDS patients as determined by International Prognostic Scoring System (IPSS); defined as not having risk assigned due to unsuccessful cytogenetics after two bone marrow attempts.

• Publications *: Steensma DP, Abedi M, Bejar R, Cogle CR, Foucar K, Garcia-Manero G, George TI, Grinblatt D, Komrokji R, Ma X, Maciejewski J, Pollyea DA, Savona MR, Scott B, Sekeres MA, Thompson MA, Swern AS, Nifenecker M, Sugrue MM, Erba H. Connect MDS/AML: design of the myelodysplastic syndromes and acute myeloid leukemia disease registry, a prospective observational cohort study. BMC Cancer. 2016 Aug 19;16:652. doi: 10.1186/s12885-016-2710-6.

Recruitment Information

• Recruitment Status: Recruiting
• Estimated Enrollment (submitted: October 23, 2019): 2100
• Original Estimated Enrollment (submitted: September 14, 2012): 1500
• Estimated Study Completion Date: December 12, 2030
• Estimated Primary Completion Date: December 12, 2022 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• Patients must be able to provide written informed consent
• Newly diagnosed (confirmed diagnosis within 60 days prior to enrollment), primary or secondary Myelodysplastic Syndromes (MDS) or Acute Myeloid Leukemia (AML)
• Disease diagnosis confirmed by Central Eligibility Review
• MDS patients of unknown-risk must have undergone two (2) bone marrow tests with failed cytogenetics
• AML patients must be at least 55 years of age at the time of informed consent signature
• MDS 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:

• For MDS and ICUS patients: received/receiving active (disease modifying) therapy** for the treatment of MDS and ICUS prior to ICF date (Supportive care such as transfusions, antibiotics, iron chelators, EPO, growth factors (G-CSF/GM-CSF) is allowed)
• For AML patients: received/receiving active (disease modifying) therapy** for the treatment of AML. Patients who initiated treatment for AML within 2 weeks (14 days) prior to ICF date are eligible for the registry if all other eligibility criteria are met (Supportive care, such as, transfusions, antibiotics, iron chelators, EPO, growth factors (G-CSF/GM-CSF), tumor lysis prophylaxis is allowed)

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Contacts

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

• Listed Location Countries: Puerto Rico, United States

Administrative Information

• NCT Number: NCT01688011
• Other Study ID Numbers: Connect® MDS/AML Registry
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Celgene
• Study Sponsor: Celgene
• Collaborators: Not Provided

Investigators

• Study Director: Irene Sheng DeGutis, PharmD, RPh; Bristol-Myers Squibb

• PRS Account: Celgene
• Verification Date: October 2020