Multiple myeloma (MM) remains incurable, with patients frequently progressing through multiple lines of therapy. In relapsed/refractory cases (RRMM), treatment options narrow significantly—especially in later lines. In a global Phase 3 trial evaluated a novel regimen against standard care (Vd) in patients with RRMM.
This case study explores how the sponsor and its partners successfully executed a complex, multinational trial involving >400 patients across 150 sites in 18 countries. Despite the challenges of global coordination, regulatory variability, and a high-risk patient population, the study achieved full enrollment and delivered pivotal data that supported regulatory approval and expanded treatment options for patients with RRMM.
|
Therapeutic Area |
Oncology |
|
Indication |
Relapsed/Refractory Multiple Myeloma |
|
Study Phase |
Phase 3 |
|
Patient Segment |
Second line and beyond (2L–4L+), Stage I–III |
|
Number of Patients |
400+ randomized |
|
Number of Sites |
150 sites |
|
Countries |
18 countries across North America, Europe, Asia, and Australia |
|
Design |
Randomized, controlled, open-label, active comparator study |
Executing a randomized, comparator-controlled trial across 150 sites in 18 countries required precision planning and operational agility. The sponsor partnered with regional CROs and deployed a hybrid site management model to accelerate activation and maintain enrollment momentum.
To meet these challenges, the sponsor partnered with global CROs and deployed a hybrid site management model, leveraging regional expertise to accelerate activation and enrollment.
The study launched in May 2017 and completed primary data collection in February 2020. Over the course of nearly three years, the study achieved several critical milestones:
|
Milestone |
Date |
|
Study Start |
May 2017 |
|
First Site Activated |
July 2017 |
|
Enrollment Completed |
February 2019 |
|
Primary Completion Date |
February 2020 |
|
Total Patients Enrolled |
402 |
|
Total Sites Activated |
150 |
|
Countries Activated |
18 |
Precision for Medicine managed 120 of the 150 sites, primarily in Europe and Australia, while the sponsor oversaw North American operations.
A dual oversight model allowed regional CROs to tailor contracting, regulatory submissions, and site engagement to local requirements. This approach reduced startup timelines and supported sustained enrollment across geographies.
Dynamic dashboards tracked screening, randomization, and dropout rates. These insights enabled the sponsor to shift resources toward high-performing regions and deprioritize underperforming sites—maintaining enrollment velocity and geographic balance.
To accommodate diverse treatment paradigms, the protocol allowed flexibility in prior lines of therapy and disease staging. This broadened eligibility and improved enrollment rates, especially in regions with differing standards of care.
With multiple CROs and sponsor teams involved, expectation management was critical. Weekly cross-functional meetings, shared trackers, and clear escalation pathways ensured transparency and timely issue resolution.
Early planning and local expertise were essential to navigate country-specific ethics and health authority requirements. Harmonized templates and centralized oversight ensured consistency across submissions.
Despite the scale, data integrity remained high. Centralized biostatistics and data management teams oversaw query resolution, form freezing, and interim analysis preparation—delivering clean datasets on schedule.
This Phase 3 program surfaced actionable insights for sponsors managing large-scale oncology trials in relapsed/refractory populations:
This trial demonstrated the feasibility of executing a complex, multinational Phase 3 study in a high-risk oncology population. The data supported regulatory approval and expanded treatment options for patients with RRMM. Operational learnings from this program continue to inform future trial designs—especially in relapsed/refractory settings where speed, flexibility, and precision are essential.
At Precision for Medicine, we specialize in navigating complexity—from biomarker-driven enrollment to global site activation and complex data workflows. Our integrated teams bring deep therapeutic expertise and operational agility to every engagement.