The US Food and Drug Administration (FDA) has issued a notification to put Solid Biosciences’ IGNITE DMD clinical on hold.

The clinical hold notice was issued after an unexpected event classified as a Suspected Unexpected Serious Adverse Reaction (SUSAR) was reported in the Phase I/II trial of SGT-001 for the treatment of Duchenne muscular dystrophy (DMD).

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

IGNITE DMD is intended to evaluate the safety and efficacy of SGT-001 in ambulatory and non-ambulatory children and adolescents with DMD.

“Shortly after the administration the patient was hospitalised due to laboratory findings that included a decrease in platelet count.”

Last month, Solid dosed the trial’s first patient, a non-ambulatory adolescent, with 5E13vg/kg of SGT-001.

Shortly after the administration the patient was hospitalised due to laboratory findings that included a decrease in platelet count, followed by a reduction in red blood cell count and evidence of complement activation.

Solid Biosciences said in a statement: “The patient showed no signs or symptoms of coagulopathy (bleeding disorder) and no relevant changes from baseline in liver function tests.

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

“The patient responded well to medical treatment and is currently asymptomatic.

“All laboratory parameters have either improved or returned to normal, and he is continuing outpatient assessments per protocol.”

The event was reported to FDA, which issued a clinical hold on the IGNITE DMD trial.

Following the hold, Solid has stopped enrolment and dosing in IGNITE DMD and is awaiting receipt of the formal Clinical Hold letter from the FDA to understand the requirements for resuming the trial.

The company’s lead candidate, SGT-001, is a new adeno-associated viral (AAV) vector-mediated gene transfer being investigated for its ability to address the underlying genetic cause of DMD. In the condition mutations in the dystrophin gene result in the absence or near-absence of dystrophin protein.

SGT-001 is a systemically administered solution that provides a synthetic dystrophin gene, known as microdystrophin, to the body.