ACT4ALS-MND is the national clinical research network dedicated to amyotrophic lateral sclerosis (ALS) and motor neuron diseases. Designated a F-CRIN network of excellence in 2022, it brings together the 22 expert centers of the FilSLAN network and serves as a single point of entry for developing and conducting innovative clinical trials in France and internationally. It strengthens academic and industrial collaborations to improve the management of these rare and progressive diseases.
ACT4ALS-MND, the national clinical research network on Amyotrophic Lateral Sclerosis (ALS) and Motor Neuron Diseases (MND)
Established in March 2020 under the leadership of the national health network FilSLAN, ACT4ALS-MND is the French network dedicated to clinical research on ALS and motor neuron diseases. FilSLAN coordinates the care pathway, training, information, and organization of expert centers in the field of ALS and motor neuron diseases. ACT4ALS-MND is part of this organization, contributing expertise specifically focused on clinical research and the development of therapeutic trials.
The network brings together the 22 French centers of expertise in this field and provides academic and industrial sponsors with a national network of experienced centers that possess recognized clinical and scientific expertise and access to well-characterized patient cohorts.
ACT4ALS-MND aims to facilitate, accelerate, and ensure the development of clinical research on ALS and MND, both in France and internationally.
The network supports projects at various stages of their development:
- study design and feasibility
- identification and mobilization of research centers
- patient recruitment
- Methodology and development of innovative protocols
- Identification and Use of Biomarkers
- Regulatory and Contractual Support
- Monitoring of patient enrollment and study conduct
ACT4ALS-MND thus fosters collaboration among clinicians, researchers, academic and industry sponsors, patient organizations, and institutional partners.
Organization
ACT4ALS-MND is supported by a national organization that enables the rapid mobilization of the expertise needed for each project.
- A coordination committee: it defines the network’s strategic direction, analyzes project feasibility, and ensures their scientific consistency.
- Operational coordination : it handles the day-to-day management of projects, coordination between project sponsors and research centers, regulatory monitoring, and tracking calls for proposals.
- Thematic expertise: expert groups can be mobilized as needed for projects, particularly in the areas of trial methodology, biomarkers, databases, and information for patients and healthcare professionals.
Thanks to its network of 22 expert centers, ACT4ALS-MND provides nationwide coverage and facilitates patient access to clinical trials.
Scientific Expertise
ACT4ALS-MND offers academic and industrial sponsors personalized support, from project design through implementation.
Scientific and Translational Expertise
- Expertise in the pathophysiological and genetic mechanisms of ALS and MNDs
- Identification and validation of diagnostic, prognostic, and monitoring biomarkers
- Development of new therapeutic approaches and translational research
- Access to databases, registries, and biobanks of characterized patients
Clinical Research Expertise
- Feasibility: rapid assessment of scientific, technical, and organizational feasibility within the network
- Design and methodology: Support in the design of clinical trials, particularly early-phase trials and innovative designs
- Study Implementation: Identification and Enrollment of Investigator Sites
- Recruitment: optimizing enrollment strategies and actively monitoring recruitment
- Regulatory and Contractual Support: Facilitating Administrative Procedures and the Contract Process
- Study Monitoring: support for research teams and rapid identification of challenges encountered in the field
Completed Projects and Notable Results
- Abstract: A national multicenter study aimed at identifying clinical, biological, genetic, histological, electrophysiological, and imaging factors predictive of ALS progression and endophenotypes in order to better stratify patients for future therapeutic trials and their management.
- Impact: Establishment of a characterized reference cohort and a major advance in the discovery of prognostic biomarkers.
- Publications on ClinicalTrials.gov: NCT02360891
- Abstract: International Phase II trial evaluating the safety and efficacy of a glucocorticoid receptor modulator in patients with ALS.
- Impact: Successful mobilization of French expert centers within the network for rapid enrollment in an international industrial trial.
- Press release from the company
- Abstract: Longitudinal evaluation of the natural history and open-label extension to assess BIIB067 in clinically presymptomatic adults with a confirmed SOD1 mutation
- Study Details: NCT04856982
- Abstract: A Study to Evaluate the Tolerability and Efficacy of AMX0035 Compared to Placebo
- Study Details NCT05021536
- Abstract: A multicenter, randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of edaravone in patients with ALS
- Publications on ClinicalTrials.gov: NCT05178810
- Abstract: To evaluate the efficacy, safety, tolerability, and pharmacokinetics of multiple intravenous infusions of NX210c
- Study Details NCT06365216
- Abstract: A Phase 2, placebo-controlled study of VHB937 for the treatment of amyotrophic lateral sclerosis;
- Study Details NCT06643481
- Abstract: A study to evaluate the efficacy, safety, tolerability, the pharmacokinetics, and the effects of biomarkers of PTC857 in adult subjects with ALS
- Study Details NCT05349721
- Abstract: Safety and tolerability evaluation, including analysis of the gut microbiota, of an oral microbiotherapy for amyotrophic lateral sclerosis
- Study details NCT05889572
- Abstract: A study to evaluate the safety and efficacy of IFB-088 as an add-on to 100 mg of riluzole compared to placebo as an add-on to 100 mg of riluzole in patients with bulbar ALS
- Study details NCT05508074
- Abstract: Diagnosis of ALS Using a Saliva Sample: an approach using non-coding RNA analysis
- Study Details NCT05928416
- Abstract: Phase IIA study to evaluate the safety, tolerability, efficacy, pharmacokinetics, and immunogenicity of ARGX-119 in patients with ALS
- Study Details NCT06441682
Current Projects
- Objective: To evaluate an innovative biologic therapy (IVB-001) based on the platelet secretome administered via intrathecal injection in patients newly diagnosed.
- Innovation: A world first in translational research aimed at slowing neurodegeneration. This project is part of a translational research effort aimed at developing new strategies capable of slowing neurodegeneration in ALS.
- Learn more about the RHU project SECRET-GIFT
Contact the network
Are you interested in developing a clinical research project on ALS or motor neuron diseases?
ACT4ALS-MND is your gateway to the clinical, scientific, and organizational expertise of the French network. Whether you are an academic or industry sponsor or a research partner, our team can assist you in assessing your project’s feasibility, identifying research centers, and developing a strategy tailored to your objectives.
To learn more about the ACT4ALS-MND network:
- Official website: https://act4als.org
- F-CRIN platform: https://fcrin.org
- Email contact: act4als.mnd@icm-institute.org
Network Partners
- Rare Diseases Health Network (FilSLAN)
- 22 French expert centers (CRMR and CRC)
- F-CRIN
- Brain Institute (ICM)
- Coordinating University Hospitals
- ARSLA Association
- TRICALS European Network
- ECRIN Infrastructure
