Study identification

EU PAS number

EUPAS1000000093

Study ID

1000000093

Official title and acronym

A Non-interventional Post Authorisation Safety Study (PASS) of Patients with MoCD Type A Treated with NULIBRY (fosdenopterin)

DARWIN EU® study

No

Study countries

European Union
United Kingdom

Study description

The NI-PASS is to facilitate the collection of additional safety and effectiveness data on NULIBRY use, including in patients with late-onset MoCD Type A, patients 1 year of age and older, who initiate treatment without dose titration, and medication errors and administration complications in the home setting, as well as pregnancy and lactation (if reported).

Treatment with NULIBRY requires specific storage, dosing, and administration, which may increase the possibility of medication errors in the home setting. There is a need to assess the risk of medication errors in the home setting in a post-approval framework. An infusion diary will be available with the drug product for the patient/caregiver to complete and monitor dates and doses administered, adverse events (AEs), medication errors and administration complications.

Objectives:
- Primary objective is the active collection of long-term safety data;
- Secondary objective is the collection of effectiveness data of all treated patients.

Study status

Planned
Research institutions and networks

Institutions

Sciensus
United Kingdom
First published:
04/04/2024
Institution Pharmaceutical company

Contact details

Mathieu Loiseau

Primary lead investigator

Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Sentynl Therapeutics, Inc. is the owner of the product with TMC Pharma (EU) Ltd the MAH in the EU (including EEA) and the U.K. Sciensus B.V. (Netherlands) is the appointed distributor and institution for conducting the study. Sentynl Therapeutics, Inc. is financing Sciensus B.V. to conduct the study.
Regulatory

Was the study required by a regulatory body?

Yes

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

EU RMP category 2 (specific obligation of marketing authorisation)