A European, non-interventional, multicentre, registry-based post-authorisation safety study to evaluate the long-term safety of Loargys treatment in arginase 1 deficiency patients in standard clinical care

Study identification

• EU PAS number:
  EUPAS1000000555
• Study ID:
  1000000555
• Official title and acronym:
  A European, non-interventional, multicentre, registry-based post-authorisation safety study to evaluate the long-term safety of Loargys treatment in arginase 1 deficiency patients in standard clinical care
• DARWIN EU® study:
  No
• Study countries:
  Austria

  France
• Study description:
  This is a non-interventional, non-comparative, multi-centre, prospective, registry-based PASS, which will be conducted in collaboration with the E-IMD. It will be based on registry data using observational methods to collect uniform data prospectively in patients with ARG1 D to monitor the long-term safety of Loargys following granting of the EU marketing authorisation.
• Study status:
  Planned

Research institutions and networks

Institutions

Immedica Pharma AB

Sweden

European registry and network for Intoxication type Metabolic Diseases (E-IMD)

Heidelberg University Hospital

Study timelines

Date when funding contract was signed

Planned:30/07/2025

Study start date

Planned:03/11/2025

Data analysis start date

Planned:15/12/2025

Date of interim report, if expected

Planned:02/02/2026

Date of final study report

Planned:04/02/2036

Sources of funding

Pharmaceutical company and other private sector 

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)
• Regulatory procedure number:
  EMA/PASS/0000258458