Adaptive Design for Phase II/III Platform Trial of Lassa Fever Therapeutics

Area of Science:

• Infectious Diseases
• Clinical Trials
• Epidemiology

Background:

• The current treatment recommendation for Lassa fever relies on ribavirin, but this is supported by limited clinical evidence.
• Lassa fever remains a significant public health concern in West Africa, with endemic transmission and epidemic potential.
• There is a critical need for improved therapeutic strategies and robust clinical trial methodologies tailored to Lassa fever's epidemiological characteristics.

Purpose of the Study:

• To propose a novel adaptive clinical trial design for evaluating Lassa fever therapeutics.
• To address the challenges in establishing robust clinical trial methods for Lassa fever research.
• To facilitate the efficient assessment of existing and novel treatment candidates.

Main Methods:

• A multicenter, randomized controlled platform trial design is proposed.
• The trial will employ an adaptive phase II/III framework.
• A superiority framework with an equal allocation ratio and interim analysis for endpoint selection and sample size estimation will be utilized.

Main Results:

• The proposed trial design offers a structured approach to overcome current limitations in Lassa fever clinical research.
• It incorporates adaptive elements to enhance efficiency and statistical power.
• The methodology addresses key challenges in primary endpoint selection and sample size determination.

Conclusions:

• The proposed adaptive platform trial provides a viable and efficient framework for rigorously evaluating Lassa fever treatments.
• This approach is crucial for advancing therapeutic options against Lassa fever.
• Implementing such a trial will accelerate the identification of effective interventions for this devastating disease.