Clinical Trial Experiences Managing Adverse Events in Gene Therapy for DMD: Natalie L. Goedeker, CPNP


At the 2023 MDA Conference, a neurology nurse practitioner in the Neuromuscular Division at Washington University in St. Louis talked about adverse event management of gene therapies in Duchenne muscular dystrophy. [WATCH TIME: 2 minutes]

WATCH TIME: 2 minutes

“Further treatment considerations [for acute liver injury] depend on the timeline and severity of their symptoms, and the severity of the lab changes, but include optimizing the steroid regimen, whether that be increasing the oral steroids for certain situations—again, the boys have to be tolerating the oral steroid dosing—or the use of IV steroids depending on the circumstances.”

Delandistrogene maxeparvovec (SRP-9001; Sarepta Therapeutics) is an investigational rAAV-based gene therapy designed to counterweigh missing dystrophin in Duchenne muscular dystrophy (DMD) by delivery of a transgene encoding an engineered protein. Sarepta’s biologics license application (BLA) for the treatment is set for review by May 29, 2023, and just recently, the FDA determined that it would hold an advisory committee on the BLA.1 News from a review of 3 clinical trials by the Delphi panel established a set of consensus considerations for the management of relevant adverse events (AEs) related to the use of the Sarepta product in the treatment of DMD.2

The analyses were presented in a late-breaking session at the 2023 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 19-22, in Dallas, Texas, by Natalie L. Goedeker, CPNP. The trials included were Study 101 (NCT03375164; n = 4), Study 102 (NCT03769116; n = 41), and Study 103, also known as the phase 3 ENDEAVOR trial(NCT24626674; n = 40). All told, the Delphi panel offered considerations for patients and caregivers to provide immediate follow-up upon onset of symptoms such as jaundice or abdominal pain, and that close monitoring of liver function should be initiated, and to be increased as clinically indicated.

Goedeker, a neurology nurse practitioner in the Neuromuscular Division at Washington University in St. Louis, sat down with NeurologyLive® in an interview at the conference to discuss more about her late breaking session presentation. She talked about the methodology of the analyses and shared some of the adverse events that patients with DMD experienced following the treatment of gene therapy.

Click here for more coverage of MDA 2023.

1. Sarepta Therapeutics Announces Advisory Committee Meeting will be Held for SRP-9001. News release. Sarepta Therapeutics. March 16, 2023. Accessed March 23, 2023.
2. Goedecker N. Management of Select Adverse Events Following Delandistrogene Moxeparvovec Gene Therapy in Patients with DMD: Delphi Consensus Guidance. Presented at: MDA Clinical & Scientific Conference; March 19-22, 2023; Dallas, TX. Late-breaking session.
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