Real-World Clinical Outcomes in Patients With Relapsed/Refractory Multiple Myeloma

Sponsor

Regeneron Pharmaceuticals (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05673967

Collaborator

(none)

330

Enrollment

15.6

Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

Primary Objective:

To describe the distribution of treatment regimens and objective response rate (ORR) in a Benchmark Cohort of real-world patients with relapsed/refractory multiple myeloma (RRMM) who initiate treatment after meeting the following criteria: (1) have either (a) at least three prior lines (3L) and are triple-class exposed (TCE), or (b) are triple-class refractory (TCR), and (2) meet similar inclusion/exclusion criteria to patients in phase 2 cohort 2 of the R5458-ONC-1826 trial.

Secondary Objectives:

To describe additional outcomes (duration of response [DOR], progression-free survival [PFS], overall survival [OS], and time to next treatment [TTNT]) in the same Benchmark Cohort population described in the primary objective.
To describe distribution of treatment regimens, ORR, DOR, PFS, OS, and to compare ORR, PFS, OS, and TTNT in an Analysis Cohort consisting of real-world patients derived from the Benchmark Cohort described above who are weighted using inverse probability of treatment weighting to align with the characteristics of patients in phase 2 cohort 2 of the R5458-ONC-1826 trial. Comparative analyses of PFS and OS will be performed conditional on sufficient maturity of survival data in the R5458-ONC-1826 trial at the time of analysis.

Condition or Disease	Intervention/Treatment	Phase
Relapsed and Refractory Multiple Myeloma (RRMM)	Other: Non-Interventional

Study Design

Study Type:

Observational

Anticipated Enrollment :

330 participants

Observational Model:

Cohort

Time Perspective:

Retrospective

Official Title:

A Study to Characterize Treatment Patterns and Real-World Outcomes in Heavily Pretreated Patients With Relapsed and Refractory Multiple Myeloma (RRMM) and Similar Clinical Characteristics to Patients in the Phase 2 Cohort 2 of the R5458-ONC-1826 Trial

Anticipated Study Start Date :

Jan 9, 2023

Anticipated Primary Completion Date :

Nov 6, 2023

Anticipated Study Completion Date :

Apr 28, 2024

Arms and Interventions

Arm	Intervention/Treatment
Cohort 1 Real-world (RW) patients with RRMM who have either at least three prior lines of therapy (LOT) and are triple-class exposed (3L+/TCE), or are triple-class refractory (TCR), meet similar inclusion/exclusion criteria used to establish phase 2 cohort 2 of the R5458-ONC-1826 trial, and are initiating currently available therapies.	Other: Non-Interventional No study treatment will be administered on this study.

Arm

Intervention/Treatment

Cohort 1

Real-world (RW) patients with RRMM who have either at least three prior lines of therapy (LOT) and are triple-class exposed (3L+/TCE), or are triple-class refractory (TCR), meet similar inclusion/exclusion criteria used to establish phase 2 cohort 2 of the R5458-ONC-1826 trial, and are initiating currently available therapies.

Other: Non-Interventional

No study treatment will be administered on this study.

Outcome Measures

Primary Outcome Measures

Proportion of patients exposed to each type of regimen by line of therapy (LOT) [Up to 6 years]
Distribution of treatment regimens
Proportion of patients with objective response rate (ORR) [Up to 6 years]
Defined as stringent complete response (sCR), complete response (CR), very good partial response (VGPR), or partial response (PR)

Secondary Outcome Measures

Duration of response (DOR) [Up to 6 years]
Defined as time from the date of the first documented response (best overall response of sCR, CR, VGPR, PR) until the first date of progressive disease (PD) by International Myeloma Working Group (IMWG) or death due to any cause, whichever occurs first.
Progression-free survival (PFS) [Up to 6 years]
Defined as time from the start of study treatment until the first date of PD by IMWG, or death due to any cause, whichever occurs first.
Overall survival (OS) [Up to 6 years]
OS is measured from the start of study treatment until death due to any cause.
Time to next treatment (TTNT) [Up to 6 years]
Defined as time from the start of study treatment until the initiation of the subsequent LOT.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Eastern Cooperative Oncology Group (ECOG) performance status ≤1 at baseline
Confirmed diagnosis of active MM by IMWG diagnostic criteria
Have myeloma that is response-evaluable with measurable disease by M-protein in serum or urine as specified in the IMWG response criteria.
Triple-class exposed or refractory

Exclusion Criteria:

Diagnosis of plasma cell leukemia, primary systemic light-chain amyloidosis (excluding myeloma-associated amyloidosis), Waldenström macroglobulinemia, or polyneuropathy, organomegaly, endocrinopathy, monoclonal protein, and skin (POEMS) changes syndrome
Known MM brain lesions or meningeal involvement
History of neurodegenerative condition, central nervous system (CNS) movement disorder, or seizure
Cardiac ejection fraction <40% by echocardiogram or multi-gated acquisition scan (MUGA) (or a diagnosis of congestive heart failure, cardiomyopathy, or valvular heart disease as a potential proxy)
Continuous systemic corticosteroid treatment with more than 10 mg per day of prednisone or anti-inflammatory equivalent
Live or live attenuated vaccines
Treated with B-cell maturation antigen (BCMA)-directed immunotherapies (BCMA antibody-drug conjugates are not excluded).