Analysis of patients with arginase 1 deficiency treated with Loargys in standard clinical care enrolled in a European, non-interventional, multicentre registry

Study identification

• EU PAS number:
  EUPAS1000000495
• Study ID:
  1000000495
• Official title and acronym:
  Analysis of patients with arginase 1 deficiency treated with Loargys in standard clinical care enrolled in a European, non-interventional, multicentre registry
• DARWIN EU® study:
  No
• Study countries:
  Austria

  France

  Netherlands
• Study description:
  This is a post-authorisation efficacy study (PAES) conducted in collaboration with the European Registry and Network for Intoxication Type Metabolic Diseases (E-IMD) that will analyse the ARG1-D population of the pre-existing and active registry held by the E-IMD. ; The E-IMD international, multicentric, observational registry collects health data generated during the routine care of affected patients with urea cycle disorders, including ARG1-D.
• 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/PAM/0000264956