A Two-Part, International, Real-World, Observational Registry of Participants Diagnosed with Aromatic L-Amino Acid Decarboxylase Deficiency (AADC-d) With or Without Treatment With Eladocagene Exuparvovec (PTC-AADC-MA-406)

Study identification

• EU PAS number:
  EUPAS105422
• Study ID:
  105423
• Official title and acronym:
  A Two-Part, International, Real-World, Observational Registry of Participants Diagnosed with Aromatic L-Amino Acid Decarboxylase Deficiency (AADC-d) With or Without Treatment With Eladocagene Exuparvovec (PTC-AADC-MA-406)
• DARWIN EU® study:
  No
• Study countries:
  Brazil

  France

  Germany

  Hong Kong

  Italy

  Saudi Arabia

  Spain

  Türkiye

  United Kingdom

  United States
• Study description:
  This is a 2-part, international, multicenter, longitudinal, real- world, observational registry of participants diagnosed with AADC-d. Part A of the registry is to observe the natural history of AADC-d in participants who receive standard of care (SoC), with the intention of gathering data about the AADC-d diagnosis, disease progression among other aspects registry that seeks to describe the natural history of AADC-d (Part A). Part B of the registry aims to evaluate the long-term effectiveness and safety of eladocagene exuparvovec in participants treated for AADC-d. By involving sites who manage AADC-d from different regions of the world which reflect different medical practices and diverse patient populations, this registry will provide a deeper and broader knowledge about the natural history and management of this condition. Furthermore, clinical outcomes resulting from this natural history registry may be compared with outcomes from future registries/studies with AADC-d gene therapy to be implemented by PTC. Part B of the registry will provide long- term follow-up data from participants treated with eladocagene exuparvovec to extend the safety profile of the product and to help characterize the long-term effectiveness of the gene therapy.
• Study status:
  Ongoing

Research institutions and networks

Institutions

PTC Therapeutics

Study timelines

Date when funding contract was signed

Actual:31/03/2020

Study start date

Actual:26/02/2021

Date of final study report

Planned:29/10/2038

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

PTC 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 2 (specific obligation of marketing authorisation)
• Regulatory procedure number:
  EMEA/H/C/005352/SOB/002

Other study registration identification numbers and links

SOB002.1