Ignite Creation Date:
2025-12-25 @ 5:05 AM
Ignite Modification Date:
2026-02-27 @ 11:20 AM
Study NCT ID:
NCT05852418
Status:
RECRUITING
Last Update Posted:
2025-01-29
First Post:
2023-04-28
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.
Sponsor:
Ambulanzpartner Soziotechnologie APST GmbH
Organization:
Study Overview
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.
Status:
RECRUITING
Status Verified Date:
2025-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: