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Longitudinal Data Collection from Patients with Spinal Muscular Atrophy: The SMArtCARE Database

Organizational Data

DRKS-ID:
DRKS00012699
Recruitment Status:
Recruiting ongoing
Date of registration in DRKS:
2018-08-09
Last update in DRKS:
2023-10-16
Registration type:
Retrospective

Acronym/abbreviation of the study

SMArtCARE

URL of the study

http://www.smartcare.de

Brief summary in lay language

The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as disease-specific SMA registry. For this purpose, an online platform will be provided for SMA patients seen by health-care providers in German-speaking countries. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries. SMArtCARE is a joint initiative of neurologists, child neurologists, and patient organizations.

Brief summary in scientific language

The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as a disease-specific SMA registry. For this purpose, an online platform will be provided for SMA patients seen by health-care providers in German-speaking countries. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries. SMArtCARE collects data on motor function as well as changes in respiratory function, nutritional status and orthopedic symptoms. To evaluate changes in motor function, standardized physiotherapeutic assessments are performed. SMArtCARE is a joint initiative of neurologists, child neurologists, and patient organizations.

Health condition or problem studied

ICD10:
G12.0 - Infantile spinal muscular atrophy, type I [Werdnig-Hoffman]
Healthy volunteers:
No

Interventions, Observational Groups

Arm 1:
The aim of SMArtCARE is to collect longitudinal “real-world data” on all available SMA patients independent of their actual treatment regime as a disease-specific SMA registry. For this purpose, an online platform will be provided for SMA patients seen by health-care providers in German-speaking countries. Data are collected during routine patient visits and items for data collection are aligned with the international consensus for SMA registries. SMArtCARE collects data on motor function as well as changes in respiratory function, nutritional status and orthopedic symptoms. To evaluate changes in motor function, standardized physiotherapeutic assessments are performed.

Endpoints

Primary outcome:
The aim of SMArtCARE is to collect data from all SMA patients during routine patient visits. SMArtCARE collects data on motor function as well as changes in respiratory function, nutritional status and orthopedic symptoms. To evaluate changes in motor function, standardized physiotherapeutic assessments are performed.
Secondary outcome:
na

Study Design

Purpose:
Other
Retrospective/prospective:
Both
Study type:
Non-interventional
Longitudinal/cross-sectional:
No Entry
Study type non-interventional:
Patient Registry

Recruitment

Recruitment Status:
Recruiting ongoing
Reason if recruiting stopped or withdrawn:
No Entry

Recruitment Locations

Recruitment countries:
  • Austria
  • Germany
  • Switzerland
Number of study centers:
Multicenter study
Recruitment location(s):
  • University medical center Klinik für Neuropädiatrie und Muskelerkrankungen Freiburg im Breisgau
  • University medical center Universitätzmedizin Leipzig, Klinik und Poliklinik für Neurologie Leipzig
  • University medical center Universitätsmedizin Mannheim, Neurologische Klinik Mannheim
  • University medical center Universitätsklinikum Würzburg, Neurologische Klinik und Poliklinik Würzburg
  • University medical center Universitätsklinikum Essen, Neurologische Klinik und Poliklinik Essen
  • Medical center DRK Kliniken Berlin Westend - Klinik für Kinder- und Jugendmedizin Berlin
  • University medical center Universitätsklinikum Essen, Klinik für Kinderheilkunde - Neuropädiatrie Essen
  • University medical center Universitätsklinikum Tübingen, Kinderklinik Abteilung III Tübingen
  • University medical center Universitätsklinikum Würzburg, Kinderklinik und Poliklinik, Sozialpädiatrisches Zentrum, Neuropädiatrie Würzburg
  • University medical center Medizinische Hochschule Hannover, Klinik für Neurologie Hannover
  • Medical center Klinikum Kassel, Neuropädiatrie Kassel
  • Medical center Ordensklinikum Linz GmbH Barmherzige Schwestern - Neuropädiatrie Linz
  • University medical center Universitätsklinikum Münster - Neuropädiatrie Münster
  • Medical center Medizinische Hochschule Hannover - Neuropädiatrie Hannover
  • Medical center Klinik für Kinder- und Jugendmedizin Ruhruniversität Bochum im St. Josef Hospital Bochum
  • Medical center Universitätsklinikum Augsburg - Mutter-Kind-Zentrum Schwaben Augsburg
  • University medical center Universitätsklinikum Heidelberg Heidelberg
  • University medical center Universitätsklinikum des Saarlandes - Klinik für Allgemeine Pädiatrie und Neonatologie Homburg
  • Medical center Hans-Berger-Klinik für Neurologie Jena
  • University medical center Universitätsklinikum Halle - Neurologie Halle Saale
  • Medical center Kaiser-Franz-Josef Spital mit G.v. Preyer´sches Kinderspital Wien
  • Medical center Klinik für Neuropädiatrie Sozialpädiatrisches Zentrum Jena
  • Medical center Kantonspital St. Gallen St. Gallen
  • University medical center Charité – Universitätsmedizin Berlin Campus Virchow Klinikum Berlin
  • University medical center Universitätsklinikum Carl Gustav Carus Dresden an der Technischen Universität Dresden - Neuropädiatrie Dresden
  • Medical center Landeskrankenhaus Bregenz - Neuropädiatrie Bregenz
  • University medical center Kepler Universitätsklinikum Linz - Neurologie Linz
  • Medical center Technische Universität München Klinikum rechts der Isar - Neurolgie München
  • University medical center Universitätsklinikum Schleswig-Holstein - Neurologie Kiel
  • Medical center Med. Campus Universität Oldenburg, Klinik für neurologische Intensivmedizin und Frührehabilitation Oldenburg
  • University medical center Universitätsklinik für Pädiatrie I Department für Kinder - und Jugendheilkunde Innsbruck
  • University medical center Universitätsklinikum Hamburg-Eppendorf Zentrum für Geburtshilfe, Kinder- und Jugendmedizin Hamburg
  • University medical center Klinik für Neuropädiatrie und Muskelerkrankungen Freiburg im Breisgau
  • University medical center Universitätzmedizin Leipzig, Klinik und Poliklinik für Neurologie Leipzig
  • University medical center Universitätsmedizin Mannheim, Neurologische Klinik Mannheim
  • University medical center Universitätsklinikum Würzburg, Neurologische Klinik und Poliklinik Würzburg
  • University medical center Universitätsklinikum Essen, Neurologische Klinik und Poliklinik Essen
  • Medical center Klinikum Stuttgart Olgaspital - Neurologie Stuttgart
  • Medical center DRK Kliniken Berlin Westend - Klinik für Kinder- und Jugendmedizin Berlin
  • University medical center Universitätsklinikum Essen, Klinik für Kinderheilkunde - Neuropädiatrie Essen
  • University medical center Universitätsklinikum Tübingen, Kinderklinik Abteilung III Tübingen
  • University medical center Universitätsklinikum Würzburg, Kinderklinik und Poliklinik, Sozialpädiatrisches Zentrum, Neuropädiatrie Würzburg
  • University medical center Universitätsmedizin Göttingen - Neuropädiatrie Göttingen
  • University medical center Universitätsklinikum Erlangen - Neuropädiatrie Erlangen
  • University medical center Medizinische Hochschule Hannover, Klinik für Neurologie Hannover
  • Medical center Klinikum Kassel, Neuropädiatrie Kassel
  • Medical center Ordensklinikum Linz GmbH Barmherzige Schwestern - Neuropädiatrie Linz
  • University medical center Universitätsklinikum Münster - Neuropädiatrie Münster
  • Medical center Medizinische Hochschule Hannover - Neuropädiatrie Hannover
  • Medical center Klinik für Kinder- und Jugendmedizin Ruhruniversität Bochum im St. Josef Hospital Bochum
  • Medical center Universitätsklinikum Augsburg - Mutter-Kind-Zentrum Schwaben Augsburg
  • University medical center Universitätsklinikum Heidelberg Heidelberg
  • University medical center Universitätsklinikum des Saarlandes - Klinik für Allgemeine Pädiatrie und Neonatologie Homburg
  • Medical center Hans-Berger-Klinik für Neurologie Jena
  • University medical center Universitätsklinikum Halle - Neurologie Halle Saale
  • Medical center Kaiser-Franz-Josef Spital mit G.v. Preyer´sches Kinderspital Wien
  • Medical center Klinik für Neuropädiatrie Sozialpädiatrisches Zentrum Jena
  • Medical center Kantonspital St. Gallen St. Gallen
  • University medical center Charité – Universitätsmedizin Berlin Campus Virchow Klinikum Berlin
  • University medical center Universitätsklinikum Carl Gustav Carus Dresden an der Technischen Universität Dresden - Neuropädiatrie Dresden
  • Medical center Landeskrankenhaus Bregenz - Neuropädiatrie Bregenz
  • Medical center Klinikum Wels-Grieskirchen GmbH - Neuropädiatrie Wels
  • Medical center DKD Helios Klinik Wiesbaden - Neurologie Wiesbaden
  • Medical center Klinikum Klagenfurt am Wörthersee - Neuroädiatrie Klagenfurt
  • University medical center Universitätsklinikum Bonn - Neuropädiatrie Bonn
  • University medical center Universitätsklinikum Carl Gustav Carus Dresden an der Technischen Universität Dresden - Neurologie Dresden
  • Medical center Landesklinikum Baden-Mölding - Neuropädiatrie Mödling
  • University medical center Universitätsklinikum Gießen und Marburg GmbH Klinikum der Justus-Liebig-Universität - Neuropädiatrie Gießen
  • Medical center Asklepios Klinik Nord Hamburg - Neuropädiatrie Hamburg
  • Medical center Kliniken der Stadt Köln gGmbH Kinderkrankenhaus Köln
  • University medical center Universitätsmedizin Göttingen - Neurologie Göttingen
  • University medical center Universitätsklinikum Erlangen - Neurologie Erlangen
  • Medical center Klinikum Klagenfurt am Wörthersee - Neurologie Klagenfurt
  • Medical center Klinikum der Universität München, Friedrich-Baur-Institut München

Recruitment period and number of participants

Planned study start date:
2018-07-15
Actual study start date:
2018-07-15
Planned study completion date:
No Entry
Actual Study Completion Date:
No Entry
Target Sample Size:
2500
Final Sample Size:
No Entry

Inclusion Criteria

Sex:
All
Minimum Age:
no minimum age
Maximum Age:
no maximum age
Additional Inclusion Criteria:
• All patients with genetically confirmed 5q SMA with residence in German-speaking regions. • Informed consent provided by patient/caregiver

Exclusion Criteria

• Further types of SMA (non 5q SMA) • Participation in an AMG trial

Addresses

Primary Sponsor

Universitätsklinikum Freiburg; Klinik für Neuropädiatrie und Muskelerkrankungen
Prof. Dr. Janbernd Kirschner
Mathildenstraße 1
79106 Freiburg
Germany
Telephone:
+49 761 270 43150
Fax:
+49 761 270 44750
Contact per E-Mail
URL:
http://www.uniklinik-freiburg.de
Investigator Sponsored/Initiated Trial (IST/IIT):
Yes

Contact for Scientific Queries

Universitätsklinikum Freiburg; Klinik für Neuropädiatrie und Muskelerkrankungen
Prof. Dr. Janbernd Kirschner
Mathildenstraße 1
79106 Freiburg
Germany
Telephone:
+49 761 270 43150
Fax:
+49 761 270 44750
Contact per E-Mail
URL:
http://www.uniklinik-freiburg.de

Contact for Public Queries

Universitätsklinikum Freiburg; Klinik für Neuropädiatrie und Muskelerkrankungen
Prof. Dr. Janbernd Kirschner
Mathildenstraße 1
79106 Freiburg
Germany
Telephone:
+49 761 270 43150
Fax:
+49 761 270 44750
Contact per E-Mail
URL:
http://www.uniklinik-freiburg.de

Principal Investigator

Universitätsklinikum Freiburg; Klinik für Neuropädiatrie und Muskelerkrankungen
Prof. Dr. Janbernd Kirschner
Mathildenstraße 1
79106 Freiburg
Germany
Telephone:
+49 761 270 43150
Fax:
+49 761 270 44750
Contact per E-Mail
URL:
http://www.uniklinik-freiburg.de

Sources of Monetary or Material Support

Commercial (pharmaceutical industry, medical engineering industry, etc.)

Biogen International GmbH
Neuhofstr. 30
6304 Baar
Switzerland
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Commercial (pharmaceutical industry, medical engineering industry, etc.)

Novartis Gene Therapies
60015 Bannockburn
United States
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Commercial (pharmaceutical industry, medical engineering industry, etc.)

Roche Pharma AG
79639 Grenzach-Whylen
Germany
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Commercial (pharmaceutical industry, medical engineering industry, etc.)

Roche Austria GmbH
1211 Wien
Austria
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Commercial (pharmaceutical industry, medical engineering industry, etc.)

F. Hoffmann - La Roche Ltd
4058 Basel
Switzerland
Telephone:
No Entry
Fax:
No Entry
Contact per E-Mail
URL:
No Entry

Ethics Committee

Address Ethics Committee

Ethik-Kommission der Albert-Ludwigs-Universität Freiburg
Engelberger Str. 21
79106 Freiburg
Germany
Telephone:
+49-761-27072600
Fax:
+49-761-27072630
Contact per E-Mail
URL:
No Entry

Vote of leading Ethics Committee

Date of ethics committee application:
2017-07-31
Ethics committee number:
EK-Nr. 56/18
Vote of the Ethics Committee:
Approved
Date of the vote:
2018-06-07

Further identification numbers

Other primary registry ID:
No Entry
EudraCT Number:
No Entry
UTN (Universal Trial Number):
No Entry
EUDAMED Number:
No Entry

IPD - Individual Participant Data

Do you plan to make participant-related data (IPD) available to other researchers in an anonymized form?:
Yes
IPD Sharing Plan:
So far, no IPD sharing plan has been created for the SMARTCARE register, as registers have not been obliged to do so to date.

Study protocol and other study documents

Study protocols:
No Entry
Study abstract:
No Entry
Other study documents:
Treatment with Nusinersen – Challenges Regarding the Indication for Children with SMA Type 1 (Pechmann A et al., Journal of neuromuscular diseases, 2020)
Zur Gentherapie der Spinalen Muskelatrophie mit Onasemnogene Abeparvovec. Stellungnahme der Gesellschaft für Neuropädiatrie (Kirschner J et al., Monatsschrift Kinderheilkunde, 2020)
Evaluation of Children with SMA Type 1 Under Treatment with Nusinersen within the Expanded Access Program in Germany (Pechmann A et al., Journal of Neuromuscular Diseases, 2018)
Background literature:
No Entry
Related DRKS studies:
No Entry

Publication of study results

Planned publication:
No Entry
Publikationen/Studienergebnisse:
SMArtCARE - A platform to collect real-life outcome data of patients with spinal muscular atrophy (Pechmann A et al., Orphanet Journal of Rare Diseases, 2019)
Date of first publication of study results:
No Entry
DRKS entry published for the first time with results:
No Entry

Basic reporting

Basic Reporting / Results tables:
No Entry
Brief summary of results:
No Entry