Solid BioSciences has announced that the IGNITE DMD trial – a Phase I/II clinical trial of the company’s SGT-001 gene therapy – has been placed on hold by the FDA (the medicines regulator in the US) after a participant experienced severe side effects (called a severe adverse event or SAE).
Solid announced plans to increase the dose of the potential gene therapy being used in the trial earlier this year. This was the third participant to receive the higher dose. The SAE led to a reduced number of some blood cells (platelets and red blood cells). It also caused acute kidney injury, and reduced heart and lung function. The participant is now recovering in hospital, and the company hopes he will be discharged shortly.
Clinical holds effectively pause a clinical trial. They can be used for a variety of reasons, including unexpected events, or serious side effects. Solid continues to monitor the participant alongside the team at the University of Florida and will be working hard to better understand what caused the event.
What is SGT-001
SGT-001 is a potential gene therapy. It uses a harmless virus to deliver a functional copy of a dystrophin gene to cells. The gene is so large – too big to be carried by the virus – that researchers have developed smaller genes. These can produce smaller but functional versions of the dystrophin protein (called microdystrophin). By using a virus that preferentially infects muscle cells, researchers hope to be able to restore some dystrophin production where it is needed most.
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