Clinical trial NCT05852418: Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.
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.
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS Registry study
SMA Registry study
ALS medication and care registry study
SMA medication and care registry study
NfL
Neurofilament light chain
ALSFRS-R
ALS-App
ALS
APST Research
Biomarker
Tofersen
Nusinersen
Risdiplam
Lead sponsor: Ambulanzpartner Soziotechnologie APST GmbH
Sponsor class: INDUSTRY
Collaborators: name: Charite University, Berlin, Germany
First posted: 2023-05-10
Enrollment: 25000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: The cohort includes patients with ALS, SMA and other chronic neurological conditions who received care with assistive devices, remedies, medications, or nursing interventions.

Brief 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.

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

-

Primary outcomes:
measure: The systematic recording of neurological treatment and its evaluation by patients via specific questionnaires
timeFrame: 10 years
Secondary outcomes:
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Christoph Münch
city: Bochum
country: Germany
facility: Neurologische Klinik und Poliklinik, Universitätsklinikum Bergmannsheil
city: Bonn
country: Germany
facility: Klinik für Neurodegenrative Erkrankungen und Gerontopsychiatrie, ALS-Ambulanz und Huntington Ambulanz, Universitätsklinikum
city: Dresden
country: Germany
facility: Klinik und Poliklinik für Neurologie, Medizinische Fakultät Carl Gustav Carus, Technischen Universität Dresden
city: Essen
country: Germany
facility: Neurologische Klinik, ALS-Ambulanz, Alfried Krupp Krankenhaus Rüttenscheid, Essen
city: Göttingen
country: Germany
facility: Klinik für Neurologie, Universitätsmedizin, Georg-August-Universität Göttingen
city: Hannover
country: Germany
facility: ALS- und Muskelambulanz, Medizinische Hochschule Hannover
city: Heidelberg
country: Germany
facility: Neurologie und Poliklinik, Universitätsklinikum
city: Jena
country: Germany
facility: Ambulanz für Neuromuskuläre und Motoneuron-Erkrankungen, Universitätsklinikum Jena
city: Leipzig
country: Germany
facility: Klinik und Poliklinik für Neurologie
city: Lübeck
country: Germany
facility: Klinik für Neuroogie-Präzisionsneurologie, Universitätsklinikum Schleswig-Holstein, Universität zu Lübeck
city: Mannheim
country: Germany
facility: Neurologisches Klinik, Ambulanz für Motoneuronerkrankungen und ALS, Diakonissenkrankenhaus
city: Mannheim
country: Germany
facility: Universitätklinikum
city: München
country: Germany
facility: Klinik und Poliklinik für Neurologie, Klinikum rechts der Isar der Technischen Universität München
city: Münster
country: Germany
facility: Klinik für Schlafmedizin und Neuromuskuläre Erkrankungen, Westfälische Willhelms-Universität Münster
city: Steinfurt
country: Germany
facility: Klinik für Innere Medizin, Neurologie, UKM Marienhospital Steinfurt
Study officials:
affiliation: Ambulanzpartner Soziotechnologie APST GmbH
name: Thomas Meyer, Prof. Dr.
role: STUDY_DIRECTOR
References: