Study identification

EU PAS number

EUPAS11275

Study ID

48265

Official title and acronym

Long-Term Observational study of Translarna Safety and Effectiveness in Usual Care (STRIDE)

DARWIN EU® study

No

Study countries

Austria
Brazil
Czechia
France
Germany
Greece
Hungary
Israel
Italy
Latvia
Portugal
Romania
Slovenia
Sweden
United Kingdom

Study description

This is a multicenter, observational study of patients receiving Translarna. No study medication will be provided as part of this observational study: the treating physician will make all treatment decisions according to his or her usual practice and will provide prescriptions as appropriate. Patients will present to the study sites for scheduled routine clinic visits with no additional visits required for data collection in the study. Enrolled patients will be followed for at least 5 years from their date of enrollment, or until patient withdrawal of consent or death, whichever occurs first. Patients who discontinue treatment with Translarna will continue to be followed for the duration of the study unless they withdraw consent to participate in the study. Data will be collected during this time period in conjunction with all routine care visits, estimated to occur at 3-6 months intervals.

Study status

Ongoing
Research institutions and networks

Institutions

Networks

TRiNDS

Contact details

Christine Keller

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Data analysis start date

Planned:
Actual:

Date of interim report, if expected

Planned:
Actual:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

PTC Therapeutics Int.
Regulatory

Was the study required by a regulatory body?

No

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

Not applicable