Case In Point
Rescuing a First-in-Human Trial for a Liver Cell Transplant Therapy
About Client
A biotechnology sponsor developing a first-in-human therapy for end-stage liver disease required an experienced clinical research partner to rescue and operationalize a Phase 2a clinical trial.
The investigational therapy uses processed donor liver cells transplanted into abdominal lymph nodes, where they develop into functioning ectopic liver tissue, with the goal of serving as a bridge to liver transplantation for patients with limited treatment options.
Challenge
Following transition from the previous CRO, the study required immediate operational stabilization. Critical clinical trial infrastructure—including clinical data management systems, study oversight processes, and cross-functional coordination—needed to be implemented while maintaining regulatory compliance and minimizing delays.
Additional challenges included:
- Coordinating enrollment around unpredictable donor organ availability and tissue processing timelines
- Supporting site transition activities and reassessing site feasibility
- Managing the complexities of first-in-human safety oversight in patients with end-stage liver disease
- Maintaining continuous study readiness despite factors outside the control of the sponsor and CRO
Solution
Everest assembled an integrated cross-functional team to assume full operational ownership of the study and establish a stable foundation for execution.
Clinical operations and project management teams coordinated site activities, enrollment readiness, sponsor communications, and ongoing operational oversight throughout the study.
To accelerate study startup, Everest designed, built, validated, and deployed the electronic clinical database within three months using a phased implementation strategy, significantly improving operational readiness after study transition.
The team maintained continuous operational readiness throughout the study, enabling enrollment whenever donor organs became available while ensuring compliance with evolving FDA IND safety reporting requirements.
Everest provided comprehensive support across:
- Clinical operations and project management
- Clinical data management and database development
- Biostatistics and statistical programming
- Pharmacovigilance and safety database management
- DSMB coordination and safety reporting
- Medical writing support
- Regulatory and ethics management, including central IRB submissions and ongoing compliance
Results
Key Outcomes Included:
- Successfully stabilized operations following CRO transition
- Built, validated, and deployed the clinical database within three months
- Supported enrollment, treatment, and follow-up for all Cohort 1 participants
- Coordinated statistical analyses and comprehensive safety reporting for DSMB review
- Enabled successful completion of Cohort 1 with a favorable safety profile
- Supported DSMB authorization to proceed with Cohort 2 enrollment
- Maintained uninterrupted regulatory and ethics compliance throughout the study
- Sustained operational readiness despite donor organ availability constraints and other logistical complexities
