Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)

Study identification

• EU PAS number:
  EUPAS25082
• Study ID:
  49288
• Official title and acronym:
  Mucopolysaccharidosis VII Disease Monitoring Program (MPS VII DMP)
• DARWIN EU® study:
  No
• Study countries:
  Argentina

  Brazil

  France

  Germany

  Netherlands

  Portugal

  Spain

  United States
• Study description:
  The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Ultragenyx Germany

Study timelines

Date when funding contract was signed

Planned:23/01/2018

Actual:23/01/2018

Study start date

Planned:29/01/2018

Actual:29/01/2018

Date of final study report

Planned:15/05/2033

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Ultragenyx

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)

Other study registration identification numbers and links

NCT03604835