Clinical trials for advancing innovative interventions for neurodegenerative diseases

Expected Outcome:

This topic aims at supporting activities that are enabling or contributing to one or several expected impacts of destination “Tackling diseases and reducing disease burden”. To that end, proposals under this topic should aim to deliver results that are directed at, tailored towards and contributing to all the following expected outcomes:

• The scientific and clinical communities make effective use of state-of-the-art knowledge, data, technologies, tools, methods, best practices, and trainings to underpin and complement the development of innovative interventions aimed at more effective treatments for neurodegenerative diseases.

• The scientific and clinical communities benefit from the exchange of data, knowledge and best practices, thereby strengthening their collaboration in the EU, the Associated Countries and beyond.

• The scientific and clinical communities make wide use of relevant databases and/or integrate them with existing infrastructures for storage and sharing of collected data according to FAIR[1] principles, thereby encouraging further use of the data.

• Policymakers, funders, scientific and clinical communities, patient organisations, regulators, and other relevant bodies are informed of the research advances made and the requirements for a widespread implementation of the innovative therapeutic interventions and complementary approaches.

• Patients and caregivers are constructively engaged with the research, ensuring that their needs are catered for, with the aim of tangibly benefitting from the interventions.

Scope:

Neurodegenerative diseases are a high burden for patients, caregivers, health systems and society. Given the limitations with current therapeutic solutions, including that they primarily address symptoms rather than underlying causes and can have serious side effects, together with the increasing prevalence of neurodegenerative diseases in an aging population, there is a huge need to develop more innovative, safer and more effective therapeutic solutions for these diseases. To further enhance their safety and effectiveness, the therapeutic solution based on an active substance should be combined/complemented with another multidisciplinary approach (e.g. lifestyle changes, cognitive training, rehabilitation therapies). Together this innovative intervention should lead to an improved quality of life and reduce the societal impact of these diseases.

Rare neurodegenerative diseases are excluded[2].

Proposals should address most of the following aspects:

• Perform rigorous early-stage[3] clinical trials into the safety and efficacy of the innovative interventions and their mode of administration, ensuring adequate cohorts/sample sizes with adequate representation of the patient population, including in terms of age, sex and ethnicity.

• Through the clinical trials and to the extent possible of additional studies, gain further insight into the potentially novel mechanism(s) of action of the innovative therapies and complementary approaches. This could entail analyses of imaging (e.g. MRI, ultrasound, nuclear imaging), as well as physiological, molecular, biochemical or omics signatures revealing potential perturbations prior to the intervention and recovery/improvement thereafter, and it could lead to the development of surrogate endpoints. This insight should open the path to more personalised interventions and approaches.

• Use and/or develop technologies, including digital ones (e.g. (generative) Artificial Intelligence - AI[4], wearable technologies) to help implement and monitor the long-term efficacy of the intervention(s), as well as manage the disorder and/or monitor their progression (e.g. with unobtrusive technologies suitable for patient monitoring at home and in real-world conditions), whilst also ensuring they are bias-free, inclusive, and ethically sound.

• Utilise existing data, biobanks, registries and/or cohorts, together with the generation of new data that should be managed in line with the FAIR principles.

• Engage all relevant stakeholders (especially patients and patients’ representatives for the disease, caregivers, clinicians, counsellors, regulators, etc.) to design end-user optimised interventions, applying gender-sensitive and intersectional approaches.

• Advance research by leveraging already existing and emerging state-of-the-art research infrastructures (e.g. EuroBioImaging[5], European Genomic Data Infrastructure[6], ECRIN[7], EATRIS[8], EBRAINS[9], BBMRI[10], etc.), as well as results stemming from EU-supported research projects, where applicable[11].

• Engage with national public health authorities and regulators to ensure a robust development pathway and further uptake of the intervention.

The participation of start-ups, micro, small and medium-sized enterprises (SMEs)[12] is encouraged with the aim of strengthening their scientific and technological foundations, enhancing their innovation potential, and exploring possibilities for commercial exploitation.

Funded projects should liaise with the co-funded European Partnership for Brain Health[13], covered by topic HORIZON-HLTH-2025-02-DISEASE-01: “European Partnership for Brain Health”.

The topic requires the effective contribution of social sciences and humanities (SSH) disciplines and the involvement of SSH experts, institutions as well as the inclusion of relevant SSH expertise, in order to produce meaningful and significant effects enhancing the societal impact of the related research activities.

All projects funded under this topic are strongly encouraged to participate in networking and joint activities, as appropriate. Therefore, proposals should include a budget for the attendance to regular joint meetings and may consider covering the costs of any other potential joint activities without the prerequisite to detail concrete joint activities at this stage. The details of these joint activities will be defined during the grant agreement preparation phase.

Applicants should provide details of their clinical studies[14] in the dedicated annex using the template provided in the submission system. As proposals under this topic are expected to include clinical studies, the use of the template is strongly encouraged.

[1] See definition of FAIR data in the introduction to this Work Programme part.

[2] Rare diseases, as defined by the European Union Regulation on Orphan Medicinal Products (1999), being a disease that affects not more than 1 person per 2000 in the European population (https://www.orpha.net/).

[3] For pharmacological-based interventions: phase 1 and/or phase 2 clinical trials.

[4] Generative AI is a type of AI technology that can generate various forms of new content such as text, images, sounds, and even code, such as for programming or gene sequencing (https://ec.europa.eu/newsroom/dae/redirection/document/101621).

[5] https://www.eurobioimaging.eu

[6] https://gdi.onemilliongenomes.eu

[7] https://ecrin.org

[8] https://eatris.eu

[9] https://www.ebrains.eu

[10] https://www.bbmri-eric.eu

[11] Consult databases e.g. CORDIS (https://cordis.europa.eu) & the JPND Research Database (https://neurodegenerationresearch.eu/search-our-database).

[12] https://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32003H0361

[13] https://cordis.europa.eu/project/id/101259512, https://www.brainhealth-partnership.eu

[14] Please note that the definition of clinical studies (see introduction to this Work Programme part) is broad and it is recommended that you review it thoroughly before submitting your application.