A Study to Evaluate Treatment Patterns and Effectiveness of Luspatercept

NCT06971185 | Active Not Recruiting FDA Drug

• 1 other identifier: CA056-1124
• Study Type: observational
• Target: 430
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to understand the treatment patterns and clinical outcomes of myelodysplastic syndromes patients treated with luspatercept or erythropoiesis-stimulating agents

Conditions

Myelodysplastic Syndromes (MDS)

Keywords

Myelodysplastic syndromes (MDS)

Outcome Measures

Primary Outcomes (8)

• Participant baseline demographics

  Baseline

• Luspatercept dose at treatment initiation

  Cohort 1 and 3 only

  Baseline

• Luspatercept dose at treatment discontinuation

  Cohort 1 and 3 only

  Up to 50 months

• Luspatercept dose change or escalation or reduction

  Cohort 1 and 3 only

  Up to 50 months

• Time from luspatercept initiation to the first occurrence of dose escalation

  Cohort 1 and 3 only

  Up to 50 months

• Proportion of participants that discontinued treatment

  Up to 50 months

• Time from treatment initiation to treatment discontinuation

  Up to 50 months

• Time from luspatercept/erythropoiesis stimulating agents treatment initiation to initiation of a new treatment for myelodysplastic syndromes

  Up to 50 months

Study Arms (3)

Cohort 1

Participants treated with first-line (1L) luspatercept treatment

Drug: Luspatercept

Cohort 2

Participants treated with first-line (1L) erythropoiesis stimulating agents

Drug: Erythropoiesis-stimulating agent (ESA)

Cohort 3

Participants treated with second-line (2L) luspatercept treatment

Drug: Luspatercept

Interventions

Luspatercept DRUG

As per product label

Cohort 1; Cohort 3

Erythropoiesis-stimulating agent (ESA) DRUG

As per product label

Cohort 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will include adult patients identified from the Flatiron Health electronic health record database between January 1, 2011 and August 31, 2024 that have been diagnosed with myelodysplastic syndromes receiving either (a) first-line (1L) luspatercept, (b) 1L erythropoiesis stimulating agents, or (c) second-line luspatercept treatment.

You may qualify if:

• Included in the Flatiron Health Broad Research Network, with 2 or more visits after January 1, 2011
• Has evidence of diagnosis with myelodysplastic syndromes (MDS) after Jan 1, 2020, as identified by a natural language processing (NLP)-based machine-learning (ML) model
• Has evidence of diagnosis with MDS as identified via structured International Classification of Diseases (ICD) codes:
• International Classification of Diseases, Tenth Revision, Clinical Modification (ICD-10-CM): D46.x
• International Classification of Diseases, Ninth Revision, Clinical Modification (ICD-9-CM): 238.7x
• Age ≥ 18 years at MDS diagnosis
• Has either ring sideroblasts positive or negative status, as confirmed by bone marrow aspirate lab results or clinician notes
• Has at least one confirmed structured activity more than 8 weeks prior to the index date
• First-line (1L) luspatercept cohort
• Has evidence of receipt of luspatercept as identified via structured data as evidenced by non-cancelled Medication Order or Medication Administration and confirmed via unstructured data
• Has evidence of treatment with luspatercept for at least 12 weeks as evidenced by non-cancelled Medication Orders or Medication Administrations
• L erythropoiesis stimulating agents (ESA) cohort:
• Has evidence of receipt of any ESA (i.e., epoetin alfa, darbepoetin alfa, epoetin beta, epoetin alfa-epbx, epoetin zeta, or epoetin beta-methoxy polyethylene glycol) for at least 12 weeks as evidenced by non-cancelled Medication Orders or Medication Administrations
• Note: this criterion is included to maximize alignment between the 1L ESA cohort and the 1L luspatercept cohort and minimize bias induced by the dosage requirement in the 1L luspatercept cohort
• Second-lin (2L) luspatercept cohort:

You may not qualify if:

• Lacking relevant unstructured documents in the Flatiron database for review by the abstraction team
• Have been exposed to any of the following MDS-related therapy prior to luspatercept initiation in the 1L and 2L settings or ESA initiation in the 1L setting: lenalidomide, azacitidine, decitabine, cedazuridine, eltrombopag, cytarabine, daunorubicin, idarubicin, filgrastim, pegfilgrastim, lipefilgrastim, sargramostim, venetoclax, or has evidence of a stem cell transplant

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (1)

Bristol Myers Squibb

Princeton, New Jersey, 08540-4715, United States

Location

Related Links

• BMS Clinical Trial Information
• FDA Safety Alerts and Recalls

MeSH Terms

Conditions

Myelodysplastic Syndromes

Interventions

luspatercept; Hematinics

Condition Hierarchy (Ancestors)

Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Hematologic Agents; Therapeutic Uses; Pharmacologic Actions; Chemical Actions and Uses

Study Officials

• Bristol Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 6, 2025
• First Posted: May 14, 2025
• Study Start: November 22, 2024
• Primary Completion: August 1, 2025
• Study Completion: August 1, 2025
• Last Updated: May 14, 2025

Record last verified: 2025-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)