Study identification

PURI

https://redirect.ema.europa.eu/resource/48479

EU PAS number

EUPAS47916

Study ID

48479

Official title and acronym

A Prospective, Observational, Post-Authorisation Efficacy Study to Assess Long-term Effectiveness of Risdiplam in Patients with Genetically Confirmed 5q SMA

DARWIN EU® study

No

Study countries

Austria
Germany
Sweden
Switzerland

Study description

This study is a multi-country, non-interventional, prospective, longitudinal cohort study that will use a hybrid method of 2 sources of data collection. The data will be extracted from existing Spinal Muscular Atrophy (SMA) patient registries as Secondary data use (SDU) and collected de novo from sites in countries not covered by the SDU under Primary data collection (PDC). Study countries will be primarily in Europe, although non-European countries (e.g. those in North America or Australasia) may be included to achieve the target sample size.

Study status

Ongoing
Research institutions and networks

Institutions

Evidera
United Kingdom
First published:
07/03/2024
InstitutionLaboratory/Research/Testing facilityNon-Pharmaceutical companyENCePP partner
Translational Research in Europe for the Assessment and Treatment of Neuromuscular Disorders (TREAT-NMD) Europe, SMArtCARE Germany, Neuromuscular Diseases in SwedenSjukdomar i Sverige (NMiS) Sweden

Contact details

Nahila Justo

Primary lead investigator
Study timelines

Date when funding contract was signed

Actual:

Study start date

Planned:
Actual:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Hoffmann-La Roche
Study protocol
Initial protocol

Prot BN43428 risdiplam v1, Published Output-1_Redacted

English (1.94 MB - PDF)View document
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

Not applicable