Metabolic Profiling of Neuromuscular Diseases (MetabNMD) – subproject SMA

Study identification

• EU PAS number:
  EUPAS32033
• Study ID:
  44920
• Official title and acronym:
  Metabolic Profiling of Neuromuscular Diseases (MetabNMD) – subproject SMA
• DARWIN EU® study:
  No
• Study countries:
  Germany
• Study description:
  This multi-center, prospective, controlled, non-randomized, non-interventional, open, unblinded study is aimed at identifying non-invasive diagnostic and prognostic biomarker profiles for neuromuscular diseases, providing a novel method for screening, predicting disease severity and dynamic monitoring under pharmacotherapy. This will ultimately allow pre-symptomatic initiation of therapies, inform about the accurate time point of treatment initiation and provide tools for individual dosing adjustments.
• Study status:
  Planned

Research institutions and networks

Institutions

Heidelberg University Hospital

Hamburg Germany, Essen Germany, Gießen Germany, München Germany

Study timelines

Date when funding contract was signed

Planned: 30/11/2019

Study start date

Planned: 01/05/2020

Data analysis start date

Planned: 30/11/2020

Date of final study report

Planned: 31/05/2022

Sources of funding

Pharmaceutical company and other private sector 

Other

More details on funding

Biogen GmBH, own resources

Regulatory

• Was the study required by a regulatory body?:
  No
• Is the study required by a Risk Management Plan (RMP)?:
  Not applicable