Spark Therapeutics has announced that the first participant has been dosed in phase 1/2 study of investigational gene therapy for Late-Onset Pompe Disease.

To read the full press release click here: https://sparktx.com/press_releases/first-patient-dosed-pompe/

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.

In March 2020, after consultation with global investigators, advocacy leadership, and healthcare experts, and in an abundance of caution for the health and safety of people with Pompe disease, Spark Therapeutics decided to voluntarily suspend enrollment into SPK-GAA-100 (seroprevalence study in participants with late-onset Pompe disease (LOPD)) and SPK-GAA-101 (Phase 1/2 gene transfer study, RESOLUTE℠, for LOPD).  This voluntary suspension was solely related to the COVID-19 (SARS CoV-2) pandemic and by suspending enrollment we aimed to minimize the risk of exposure to COVID-19 for participants who would have been traveling to and from investigational sites.

After carefully reviewing the emerging data and ongoing measures in place to minimize and prevent the spread of COVID-19 (SARS CoV-2), consulting the recent US FDA-issued guidance for clinical trial conduct during the COVID-19 public health emergency, and discussing with clinicians and scientists with extensive Pompe disease and immunology experience, Spark Therapeutics has restarted enrollment into both studies mentioned above in August 2020.

Spark has thoroughly reviewed and is continuing to evaluate additional aspects of the protocols and study conduct that may also be impacted by COVID-19.  The schedule of assessments currently included in the study protocols have been carefully evaluated to minimize visits to study sites.  Clinical site personnel are being instructed to conduct any and all COVID-19 related screening procedures that may be mandated by the health care system in which a clinical trial is being conducted.  Additional safety measures may be initiated, as per the judgment of the Investigator, per institutional guidelines and in consultation with the Sponsor Medical Monitor.

As a leader in the field of gene therapy, Spark Therapeutics has always placed patient safety first and foremost in our research and remains committed to the investigation of a potential gene therapy for Pompe disease and to safely restarting the seroprevalence study and RESOLUTE℠.

For more information regarding the studies, please visit www.ClinicalTrials.gov or forpatients.roche.com. 

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.

Ria Broekgaarden who is one of the founders of the IPA (in 1999) retired from her job with the Dutch Neuromuscular Association (SN) after working there for over 30 years.

Postponed because of the corona crisis a Zoom Meeting was organized by the Dutch Neuromuscular Association on Friday the June 26th to say goodbye to her. Around 100 people, members of SN, members of the IPA and members of other neuromuscular associations were present.

She was praised for her tireless efforts on behalf of all people with neuromuscular disorders but in particular the IPA where she stood on barricades for the development of therapy.

She was addressed by amongst others Dr. Ans van der Ploeg from the Erasmus MC who referred to everything Ria had done for the Pompe Community as a whole, the Erasmus Pompe Survey, the supply shortage, reimbursement and many other occasions. Wilma Treur (IPA secretary) referred to the many trips they made for the IPA and the Pompe community as a whole to advocate for the interest of Pompe patients worldwide.

At the end of the session the mayor of Baarn (home to SN) arrived and presented Ria with royal accreditation: she received a ribbon and became Officer in the Order of Oranje Nassau the Dutch Royal House.