Connect® Myeloid Disease Registry

Terminated

• Conditions: Primary Myelofibrosis; Myelodysplastic Syndromes; Leukemia, Myeloid, Acute
• Interventions: Drug: Luspatercept

• Registration Number: NCT01688011
• Lead Sponsor: Celgene

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.

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.

Study Timeline

• Study First Submitted: 2012-09-14
• Study First Submitted QC: 2012-09-14
• Study First Posted: 2012-09-19
• Study Start: 2013-12-12
• Primary Completion: 2024-10-18
• Study Completion: 2024-10-18
• Status Verified: 2025-06
• Last Update Submitted: 2025-07-01
• Last Update Posted: 2025-07-03

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 2013

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Luspatercept treated cohort (LTC)	Luspatercept	Participants that have initiated luspatercept treatment for a myeloid malignancy.

Primary Outcome Measures

Name	Time	Method
Patient Demographics- MDS/AML/ICUS Cohorts	Up to 8 years	Describe demographics, baseline characteristics and clinical outcomes of the patients with LR or HR MDS, ICUS, and AML.
Safety and Effectiveness- MF Cohort	Up to 5 years	Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events.
Treatment effectiveness - LTC	Minimum of 3-months post index date	Describe clinical response to treatment, transfusion information, ECOG performance status and deaths.
Diagnostic and Treatment Patterns- MDS/AML/ICUS Cohorts	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
Safety and Effectiveness- MDS/AML/ICUS Cohorts	Up to 8 years	Describe the survival status, clinical response to treatment, select laboratory results, occurrence of secondary primary malignancies, deaths, select adverse events.
Diagnostic and Treatment Patterns- MF Cohort	Up to 5 years	Describe current and evolving patterns for diagnosis, treatment sequencing, routine clinical practice patterns and clinical outcome measures in patients enrolled in the MF Cohort
Treatment patterns and clinical outcomes - LTC Cohort	Minimum of 3-months post index date	Describe the myeloid malignancy treatment patterns and clinical outcomes before and after initiating luspatercept treatment
Treatment duration - LTC Cohort	Minimum of 3-months post index date	Luspatercept treatment duration
Patient Demographics- MF Cohort	Up to 5 years	Describe demographics, baseline characteristics, patient recorded outcomes, and clinical outcomes of patients enrolled to the MF cohort
Transfusion information - LTC	Minimum of 3-months post index date	Describe changes in hemoglobin and transfusion independence status.

Secondary Outcome Measures

Name	Time	Method
Patient demographics and clinical characteristics - LTC	Baseline	Describe demographics, baseline characteristics and clinical outcomes of the patients treated with luspatercept
Reason for treatment discontinuation - LTC	Minimum of 3-months post index date	Describe reasons for luspatercept treatment discontinuation
Patient Reported Outcome	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	Up to 8 years	Perform molecular and cellular correlative studies on blood/bone marrow and oral epithelial cell samples.

Trial Locations

Locations (157)

DCH Health System (Lewis and Faye Manderson Cancer Center); 🇺🇸 Tuscaloosa, Alabama, United States

Arizona Oncology; 🇺🇸 Phoenix, Arizona, United States

The University of Arizona Cancer Center; 🇺🇸 Tucson, Arizona, United States

Local Institution - 1273; 🇺🇸 Tucson, Arizona, United States

Highlands Oncology Group; 🇺🇸 Fayetteville, Arkansas, United States

Clopton Clinic of Jonesboro, Inc.; 🇺🇸 Jonesboro, Arkansas, United States

NEABC- Fowler Family Center for Cancer Care; 🇺🇸 Jonesboro, Arkansas, United States

Local Institution - 1161; 🇺🇸 Jonesboro, Arkansas, United States

Comprehensive Blood & Cancer Center; 🇺🇸 Bakersfield, California, United States

Alta Bates Summit Comprehensive Cancer; 🇺🇸 Berkeley, California, United States

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