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, longitudinal cohort study utilising data from existing SMA patient registries. The study was initially proposed to be conducted using both primary data collection and secondary data extracted from existing SMA registries. However, given the feasibility assessment results, the prospective primary
data collection was removed from the initial version of the plan, after discussion with CHMP. This study is a multi-country, non-interventional, longitudinal cohort study utilising data from existing SMA patient registries. The study was initially proposed to be conducted using both primary data collection and secondary data extracted from existing SMA registries. However, given the feasibility assessment results, the prospective primary data collection was removed from the initial version of the plan, after discussion with CHMP.

Study status

Ongoing
Research institutions and networks

Institutions

Evidera
United Kingdom
First published:
07/03/2024
InstitutionLaboratory/Research/Testing facilityNon-Pharmaceutical companyENCePP partner
Pediatric Neuromuscular Clinical Research (PNCR)

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
Updated protocol

Prot_BN43428_risdiplam_v2_12Dec2024_Redacted.pdf

English (1.37 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