The Food and Drug Administration in the United States (FDA) has lifted the clinical hold on Part B of the Phase 2 MOMENTUM clinical trial of SRP-5051 (vesleteplirsen), the investigational treatment for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
The hold was initially placed on the trial in the US in June 2022 after a patient experienced a serious adverse event (SAE) of hypomagnesemia after treatment with high-dose SRP-5051. At the time, the FDA requested information on all cases of hypomagnesemia, including a small number of non-serious grade 2 cases, and to assess the adequacy of the risk mitigation and safety monitoring plan.
As a result of this lift, Sarepta will now need to adjust the global trial protocol to include expanded monitoring of urine biomarkers as part of the risk mitigation and safety monitoring plan.
“We would like to thank FDA for working closely with us to expeditiously resolve this clinical hold. We will implement the changes in the protocol to resume dosing in the US as quickly as possible. Our monitoring plan is designed to mitigate the risks of hypomagnesemia. MOMENTUM has continued enrolling participants outside the US, and we remain on track to complete enrollment by the end of 2022.”
Louise Rodino-Klapac, Ph.D., executive vice president and chief scientific officer, Sarepta Therapeutics
About MOMENTUM (Study SRP-5051-201)
MOMENTUM is a Phase 2, multi-arm, ascending dose trial of SRP-5051, infused monthly and will assess dystrophin protein levels in skeletal muscle tissue following SRP-5051 treatment. The trial will enroll up to 60 ambulant and non-ambulant participants between the ages of 7 to 21 at sites in the U.S., Canada, and the European Union. The trial will also assess safety and tolerability.