Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Amyotrophic Lateral Sclerosis. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Germany
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
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.
Overview
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.
Primary outcome measures
- The systematic recording of neurological treatment and its evaluation by patients via specific questionnaires [Time frame: 10 years]
Eligibility criteria
Inclusion Criteria: Diagnosis of a chronic neurological disease -
Exclusion Criteria: Lack of cooperation and unwillingness to store and share medical data collected in the registry study
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Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
Germany · 16 centers
- Christoph Münch — Berlin
- Neurologische Klinik und Poliklinik, Universitätsklinikum Bergmannsheil — Bochum
- Klinik für Neurodegenrative Erkrankungen und Gerontopsychiatrie, ALS-Ambulanz und Huntingt — Bonn
- Klinik und Poliklinik für Neurologie, Medizinische Fakultät Carl Gustav Carus, Technischen — Dresden
- Neurologische Klinik, ALS-Ambulanz, Alfried Krupp Krankenhaus Rüttenscheid, Essen — Essen
- Klinik für Neurologie, Universitätsmedizin, Georg-August-Universität Göttingen — Göttingen
- ALS- und Muskelambulanz, Medizinische Hochschule Hannover — Hanover
- Neurologie und Poliklinik, Universitätsklinikum — Heidelberg
- … and 8 more centers
Identifiers
NCT: NCT05852418 · EA1/219/15