Long-term efficacy and safety follow-up of MLD patients treated with atidarsagene autotemcel (LongTERM-MLD)

Study identification

• EU PAS number:
  EUPAS48374
• Study ID:
  48375
• Official title and acronym:
  Long-term efficacy and safety follow-up of MLD patients treated with atidarsagene autotemcel (LongTERM-MLD)
• DARWIN EU® study:
  No
• Study countries:
  France

  Germany

  Italy

  Netherlands

  Sweden

  United Kingdom
• Study description:
  The aim of this study is to ensure that efficacy and safety of patients treated with Libmeldy in the Clinical Development Program (CDP) and in post-authorisation setting are assessed for up to 15 years following treatment in line with regulatory requirements.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Orchard Therapeutics

Study timelines

Date when funding contract was signed

Planned:19/05/2022

Actual:19/05/2022

Study start date

Planned:30/12/2022

Actual:21/12/2022

Date of final study report

Planned:29/03/2041

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Orchard Therapeutics

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 1 (imposed as condition of marketing authorisation)

Other study registration identification numbers and links

OTL-200-10