Development of tools and criteria to select and prioritise candidate interventions for early-phase sarcopenia clinical trials.

Abstract ID
3142
Authors' names
C McDonald1,2; R Polyma1,2,3; , M Witham1,2
Author's provenances
1. AGE Research Group, Translational and Clinical Research Institute, Faculty of Medical Sciences, Newcastle University, Newcastle, UK; 2. NIHR Newcastle Biomedical Research Centre, Newcastle upon Tyne Hospitals NHS Foundation Trust, Cumbria, Northumberla
Abstract category
Abstract sub-category
Conditions

Abstract

Background 

Recent advances in skeletal muscle biology have identified multiple potential candidate therapeutic interventions for sarcopenia. A systematic approach is needed to prioritise the most promising interventions for early-phase clinical studies. 

Methods 

A multidisciplinary team with expertise in sarcopenia, early-phase clinical trials, and geriatric medicine sought to identify target product profile (TPP) and intervention selection tools for neuromuscular conditions. As none were identified for sarcopenia, the group then developed a sarcopenia TPP. An algorithm was created to select interventions most suitable for early-phase trials, combining "stop/go" criteria and traffic-light ratings. The tool was tested by evaluating outputs from a recent horizon scan and was adapted iteratively based on the findings. 

Results 

Key characteristics of an effective intervention for sarcopenia, as outlined by the Therapeutic Product Profile (TPP), include: - Improving strength and/or physical performance. - Safety for adults aged 65 and over. - Mild, reversible, infrequent, and/or transient side effects only. - Oral or intranasal administration twice a day or less; injectable forms once a month or less. - Minimal or no laboratory monitoring required. - No need for cold chain storage. The selection algorithm starts with two questions: “Is the drug contraindicated for older adults?” and “Is there already a Phase 2/3 clinical trial evaluating this or similar agents for sarcopenia?” The assessment considers evidence strength, mechanism of action, effective dose, safety in older adults, administration route and frequency, side effects, patent status, and availability. Examples of interventions include Alverine citrate (deprioritized as it is contraindicated in older adults), melatonin (prioritised for its safety and plausible mechanism), and angiotensin-receptor blockers (deprioritized due to previous Phase II testing). 

Conclusions 

The TPP and intervention selection tool show promise in enabling systematic evaluation of candidate sarcopenia interventions. They will now be used to select and prioritise interventions for future trials.