ATB200-07: Open Label Extension of ATB200 & AT2221 in Adults with LOPD

• Research type

  Research Study

• Full title

  A PHASE 3 OPEN-LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY AND EFFICACY OF INTRAVENOUS ATB200 CO-ADMINISTERED WITH ORAL AT2221 IN ADULT SUBJECTS WITH LATE-ONSET POMPE DISEASE

• IRAS ID

  274987

• Contact name

  Mark Roberts

• Contact email

  Mark.roberts@srft.nhs.uk

• Sponsor organisation

  Amicus Therapeutics, Inc.

• Eudract number

  2019-000954-67

• Clinicaltrials.gov Identifier

  NCT04138277

• Clinicaltrials.gov Identifier

  127,387, US IND

• Duration of Study in the UK

  3 years, 4 months, 18 days

• Research summary

  This research study is an open-label extension study to evaluate the long-term safety and efficacy of, intravenous ATB200 co-administered with oral AT2221, in the treatment of adult participants with Late-Onset Pompe Disease (LOPD) who participated in the previous study ATB200-03.

  LOPD is a genetic disorder caused by mutations in the gene encoding human acid a-glucosidase (GAA). GAA is responsible for the breakdown of glycogen. Therefore, the complete absence or reduced functioning of GAA means there is more glycogen within cells. This causes cellular disruption, particularly in the heart, skeletal muscles and diaphragm. Most patients experience difficulty walking, climbing stairs and have progressive limitations in motor functions.
  
  The study drugs being investigated are designed to improve the breakdown of glycogen. ATB200 is the main active ingredient that initiates this breakdown. AT2221 is a pharmacological chaperone, meaning it supports in the delivery of ATB200 to affected cells and protects ATB200 from being broken down in the body, ultimately enhancing its therapeutic effect.

  The study will be conducted in 90 sites globally. Participants who are currently in the ATB200-03 study will be asked on their last study visit if they wish to continue receiving study drug in the proposed extension study. Participants will be required to attend numerous study visits where any changes in their motor function, pulmonary function, muscle strength and the 6-Minute Walk test will be measured. Participants will also complete patient reported outcomes to determine any changes in their quality of life. Participants will receive ATB200/AT2221 every 2 weeks either at the research site or at home (if eligible). Participants will remain in the study until regulatory approval or marketing authorisation is in place.

• REC name

  North West - Greater Manchester South Research Ethics Committee

• REC reference

  20/NW/0053

• Date of REC Opinion

  8 Apr 2020

• REC opinion

  Further Information Favourable Opinion