Study identification

PURI

https://redirect.ema.europa.eu/resource/49676

EU PAS number

EUPAS47679

Study ID

49676

Official title and acronym

Risdiplam Single-Arm Pregnancy Safety Study

DARWIN EU® study

No

Study countries

Germany
Italy
United States

Study description

This single-arm pregnancy safety study will collect primary data from risdiplam-exposed pregnant women and their healthcare professionals (HCPs), as well as their infant’s HCP. Patients who have been exposed to risdiplam during 14 days prior to their last menstrual period (LMP) or at any time during pregnancy will be eligible for participation. Inclusion of exposure prior to LMP is based on pharmacokinetic characteristics of risdiplam. Dosing and treatment duration of risdiplam as part of this non-interventional study is at discretion of physician in accordance with local clinical practice and local labeling. Total duration of participation is up to 21 months (e.g. 9 months of pregnancy and 12 months of infant follow-up), and study duration is expected to be maximum of 10 years. Shorter duration may be adopted based on cases reported and in agreement with Health Authorities. Participants may enroll prospectively or retrospectively (e.g.after pregnancy outcome has occurred).

Study status

Ongoing
Research institutions and networks

Institutions

Fondazione I.R.C.C.S. Istituto Carlo Besta Milan, Italy, Universitätsklinikum Essen (AöR), Klinik für Neurologie Essen, Germany, Helen DeVos Children's Hospital at Spectrum Health Grand Rapids, USA

Contact details

Marianne Gerber

Primary lead investigator
Study timelines

Date when funding contract was signed

Actual:

Study start date

Planned:
Actual:

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 BN42833 risdiplam v3, Published Output-1_Redacted

English (1.54 MB - PDF)View document
Regulatory

Was the study required by a regulatory body?

Yes

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

EU RMP category 3 (required)