There are multiple clinical trials that are currently underway for Pompe disease. Because there are so many different trials, which is unique for a rare disease like Pompe, it is important to understand the clinical trial process, the trials phases, and other basic information.

Please find in the attachment a short overview of clinical trials in general.

The International Pompe Association

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(courtesy of the AMDA - Acid Maltase Deficiency Association)

• Overview of Clinical Trials (PDF, 320.97 kB)

CRANBURY, N.J., Dec. 20, 2018 (GLOBE NEWSWIRE) --  Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, today announced the dosing of the first patient in a global phase 3 clinical study (ATB200-03, or PROPEL) of AT-GAA in adult patients with late onset Pompe disease. PROPEL is a 52-week, double-blind randomized study designed to assess the efficacy, safety and tolerability of AT-GAA compared to the current standard of care, alglucosidase alfa, an enzyme replacement therapy (ERT).

All participants randomized to AT-GAA in the PROPEL study will receive drug manufactured at the 1000L scale intended for clinical and commercial supply. Amicus also expects to initiate a smaller, open-label study of AT-GAA in pediatric patients in 2019.

“The initiation of our global PROPEL study is a true example of our capabilities to discover, develop and manufacture promising therapies, and deliver them to patients as quickly as we can,” said John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc.

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• Press release (external link)

Disclaimer: The IPA does not endorse any of the products, medications, treatments or information reported herein. Articles on the IPA web pages are intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

The New Zealand Organisation for Rare Disorders (NZORD) will present the New Zealand Rare Disease Day Awards (RDD Awards) for the first time in 2019. The winner in the Patient Advocacy and Support Award category is Allyson Lock. She has been nominated as a tireless fighter for the Pompe community.

Allyson has spent the past eight years lobbying and supporting patients in New Zealand. She is President of the New Zealand Pompe Network and since her diagnosis has fought to ensure that the cost of treating Pompe with enzyme replacement therapy is covered by the New Zealand health care system.

We congratulate Allyson on the award.

(Photo courtesy of Allyson Lock and the New Zealand Organisation for Rare Disorders, photographed by Levi Gershkowitz of Living In The Light Of Rare Diseases.)