Duchenne Clinical Trials & COVID-19

With the Coronavirus impacting many facets of day to day life, PPMD recognizes the concern from many in the community in regards to the potential impact on Duchenne clinical trials. Last week, FDA issued a guidance document to sponsors (drug companies) about COVID-19. FDA guidance documents provide the FDA’s current thinking on a given topic.

Each sponsor is in process of figuring out alternative plans (if warranted) for dosing and monitoring patients in trials during the COVID-19 pandemic.  Changes to current trials (also known as protocol modifications) may be required due to COVID-19 illness and/or control measures. You should expect to hear from your clinical trial site on how dosing and monitoring will proceed. For trials yet to enroll, there may be delays to recruitment.

PPMD is working with companies to ensure all participants are kept informed of changes to the study and monitoring plans that could impact them. PPMD has requested community facing information from companies regarding trials which some have already provided (see updates below).

If you are having trouble reaching your trial site for more information please contact rachel@parentprojectmd.org

Updates from Clinical Trial Sponsors & Approved Drugs

As more families begin to self-quarantine, work & school from home, and drastically change day-to-day routines, questions are arising about what it means if you or your child are receiving medication or treatments from a specific company or participating in a clinical trial. PPMD has reached out to our industry partners and asked for updates. We will continue to update this list as more information becomes available.

Astellas

• April 7, 2020: Astellas Pharma shared an update on clinical trial status and COVID-19.

Audentes

• April 5, 2020: Audentes Therapeutics shared an update with the community regarding their Duchenne program and COVID-19.

Catabasis

• March 26, 2020: Catabasis Pharmaceuticals shared the latest edition of their community newsletter, including information about edasalonexent and COVID-19, and responses to questions they have received.
• March 17, 2020: Catabasis Pharmaceuticals shared an update responding to questions about edasalonexent and COVID-19, reporting that they not identified any safety concerns related to COVID-19 and they continue to monitor the trial for the safety of the participants.

Italfarmaco

• March 27, 2020: Italfarmaco shared an update with the community regarding givinostat clinical trials and COVID-19.

NS Pharma

• March 23, 2020: NS Pharma, Inc. shared an update with the community responding to questions about how COVID-19 may be affecting ongoing studies of viltolarsen, the recently launched Expanded Access Program (EAP) and review of the viltolarsen New Drug Application (NDA) by the FDA.

Pfizer

• March 27, 2020: Pfizer shared an update regarding the PF-06939926 gene therapy trial and COVID-19.
• March 18, 2020: Pfizer provided information and resources on COVID-19.

PTC

• March 27, 2020: PTC Therapeutics provided additional details about Emflaza during the COVID-19.
• March 16, 2020: PTC Therapeutics shared a statement on EMFLAZA availability during the coronavirus (COVID-19) crisis, stating that the supply of EMFLAZA will not be affected by the coronavirus (COVID-19). PTC reports that they are closely monitoring any shipping issues in the United States, which could have an impact of delivery of drugs to patients. If patients have questions, you can contact PTC Cares at 1-844-PTC-CARES (844-478-2227).

ReveraGen

• March 23, 2020: ReveraGen shared an update with the community regarding the Vamorolone trials and COVID-19.

Santhera

• April 1, 2020: Santhera Pharmaceuticals shared an update with the community regarding the SIDEROS clinical trial and their activities during the COVID-19 epidemic. To learn more about respiratory issues in Duchenne, visit TakeABreathDMD.com.

Sarepta

• April 13, 2020: Sarepta Therapeutics shares COVID-19 resources.
• March 19, 2020: Sarepta Therapeutics provided an update addressing questions from the community regarding COVID-19, including updates on current clinical trials and accessing approved therapies.

Solid Biosciences

• March 30, 2020: Solid Biosciences Inc. shared an update with the community regarding the IGNITE DMD clinical hold and COVID-19.