NCT05852418

Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.

Registry Study of the Provision of Assistive Devices, Medicines, and Other Healthcare Measures in an Inter-cohort Observation of Patients With ALS , SMA and Other Neurological Diseases.

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 25,000 (estimated)
• Sponsor: Ambulanzpartner Soziotechnologie APST GmbH · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This registry study aims to collect data on the provision of assistive devices, medicines, and other healthcare measures, such as ventilation therapy and nutrition support, in patients with Amyotrophic lateral sclerosis (ALS), Spinal muscular atrophy (SMA) and other neurological disorders. The data collected should describe the clinical practice, meaning real-world evidence and patient-reported outcomes.

Detailed description

This registry study aims to collect data on the provision of assistive devices, medicines, and other healthcare measures, such as ventilation therapy and nutrition support, in patients with ALS, SMA and other neurological disorders. The data collected should describe the clinical practice, meaning real-world evidence and patient-reported outcomes. The collected data include clinical characteristics, medical history, assessment scales, such as ALS functional rating scale-revised (ALSFRS-R), prognosis indicators, such as ALS progression rate, biomarker data, such as serum Neurofilament light chain (sNfL), genetic data, such as the mutation status of SOD1, FUS, c9orf72, TARDBP in ALS, SMN1 in SMA patients, innovative drugs such as Tofersen, Nusinersen, Risdiplam, as well as symptomatic drugs, patient-reported outcome data being captured using questionnaires and established clinical scales on medication expectation and treatment satisfaction.

Conditions

• Amyotrophic Lateral Sclerosis

Timeline

• Start date: 2015-10-30
• Primary completion: 2028-12-31
• Completion: 2029-06-01
• First posted: 2023-05-10
• Last updated: 2025-01-29

Locations

16 sites across 1 country: Germany