Overview:
Early clinical trial designs—especially first-in-human (FIH) studies—are increasingly tailored to the unique needs of specific therapeutic areas. In neurodegenerative diseases, there is a growing shift toward flexible and patient-centric development strategies, often combining multiple objectives within fewer protocols and aiming to collect early patient data. In some cases, initiating trials directly in the target population is required due to ethical or tolerability considerations.
During this webinar hosted by ASCPT we will explore key considerations and share examples from studies in neurodegenerative disease therapeutics, focusing on:
1. Essential “Inflexible” elements: Early phase clinical trial objectives and target populations
- Defining appropriate populations for assessment
- Balancing efficiency, cost-effectiveness, and data utility
2. The “dedicated patient cohort” and the patient perspective
- Understanding patient motivations and barriers to participation
- Adjusting trial designs to improve recruitment and data quality
3. The expanding universe of biomarkers
- The scope and methodologies of biomarkers
- Choosing the right biomarker at the right time
- Selected regulatory expectations and practical considerations for implementation
Speakers:
Paula Kopschina Feltes, Ph.D.
Paula is a Senior Manager, Solutions Architect at ICON. Before joining ICON, Paula was a researcher in brain PET imaging at the University Medical Center Groningen (UMCG) in The Netherlands, holding a double-PhD in the field. She has worked on many investigator-initiated research as well as partnerships with the industry in clinical trials for major pharma companies. Within ICON, Paula performs feasibility assessments for early phase clinical trials and acts as a subject matter expert in Nuclear Medicine, where she provides guidance and consultancy for customers on optimal trial design, clinical conduct and scientific advice.
Matthias Mohse, Dipl.-Psych
Matthias is a Director of Clinical Pharmacology at ICON. He provides drug development guidance to customers, including trial design optimisation, dose selection, advice regarding safety, PK and PD, eligibility criteria, risk mitigation, and practical aspects of the study conduct.Matthias has a strong neuroscience background, extensive CNS research methodology experience, and a passion for experimental disease models and their utilias,tion for proof of mechanism.
Target audience:
The target audience for this webinar includes clinical researchers, drug development professionals, and scientists involved in early phase clinical trials focused on neurodegenerative diseases. It is particularly intended to raise interest and stimulate innovation in patient engagement strategies, study design optimisation, and biomarker selection in first-in-human and early clinical development settings.
The session will be especially relevant for:
- Clinical pharmacologists and trial designers working on neurodegenerative disease therapeutics
- Medical scientists and researchers specializing in CNS disorders and biomarker development
- Patient engagement and advocacy professionals seeking to improve recruitment and retention in early trials
- Regulatory affairs specialists interested in strategic biomarker implementation for trial approval
- Pharmaceutical and biotech industry professionals involved in early clinical development and feasibility assessments
- Clinicians and translational researchers focusing on patient-centric trial methodologies and outcomes