A GLOBAL PROSPECTIVE OBSERVATIONAL REGISTRY OF PATIENTS WITH POMPE DISEASE

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/107300
• EU PAS number:
  EUPAS107299
• Study ID:
  107300
• Official title and acronym:
  A GLOBAL PROSPECTIVE OBSERVATIONAL REGISTRY OF PATIENTS WITH POMPE DISEASE
• DARWIN EU® study:
  No
• Study countries:
  Argentina

  Australia

  Austria

  Belgium

  Bosnia and Herzegovina

  Brazil

  Canada

  Chile

  Czechia

  Denmark

  Estonia

  France

  Germany

  Greece

  Hungary

  Israel

  Italy

  Japan

  Korea, Republic of

  Netherlands

  New Zealand

  Poland

  Portugal

  Serbia

  Slovakia

  Slovenia

  Spain

  Sweden

  Taiwan

  Thailand

  United Kingdom

  United States
• Study description:
  The goal of this registry is to assess clinical outcomes in patients with Pompe disease, including patients with late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD), regardless of current or previous therapy.
• Study status:
  Planned

Research institutions and networks

Institutions

Amicus Therapeutics

Study timelines

Date when funding contract was signed

Planned: 01/10/2023

Study start date

Planned: 01/10/2023

Date of final study report

Planned: 30/12/2033

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Amicus Therapeutics Inc

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)

Other study registration identification numbers and links

POM-005