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

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

Study timelines

Date when funding contract was signed

Planned: 02/02/2024

Actual: 02/02/2024

Study start date

Planned: 01/06/2024

Date of final study report

Planned: 01/06/2025

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)