EAMS TREATMENT REGISTRY PROTOCOL FOR ATB200/AT2221

• Research type

  Research Study

• Full title

  CIPAGLUCOSIDASE ALFA/MIGLUSTAT EARLY ACCESS TO MEDICINES SCHEME TREATMENT REGISTRY PROTOCOL FOR CIPAGLUCOSIDASE ALFA IN CONJUNCTION WITH MIGLUSTAT FOR THE TREATMENT OF ADULT PATIENTS WITH LATE-ONSET POMPE DISEASE

• IRAS ID

  302770

• Contact name

  Mark Roberts

• Contact email

  mark.roberts@srft.nhs.uk

• Sponsor organisation

  Amicus Therapeutics

• Clinicaltrials.gov Identifier

  50636/0001, EAMS Number:

• Duration of Study in the UK

  1 years, 5 months, 20 days

• Research summary

  This observational registry will document the use of cipaglucosidase alfa/miglustat in adult patients with Late Onset Pompe Disease (LOPD) as used under the Early Access to Medicines Scheme (EAMS) in the United Kingdom. The objective of the registry is to evaluate the safety and effectiveness by collecting clinical outcomes in adult patients with LOPD.

• REC name

  South Central - Hampshire B Research Ethics Committee

• REC reference

  21/SC/0263

• Date of REC Opinion

  21 Sep 2021

• REC opinion

  Further Information Favourable Opinion