First-in-Kids gene editing trial aims to fix rare, dangerous fat disorder

NCT ID NCT07371767

Recruiting now Disease control Sponsor: Shanghai Jiao Tong University School of Medicine Source: ClinicalTrials.gov ↗

Summary

This early-stage study is testing a new, one-time gene editing treatment called CS-121 in children and teenagers with a rare, severe genetic disorder that causes extremely high blood fats. The goal is to see if safely editing a specific gene in the liver can lower fat levels and reduce the risk of life-threatening pancreatitis. Researchers will give the therapy to up to 15 young patients to check for safety and find the right dose.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Shanghai Children's Medical Center
RECRUITING

Shanghai, Shanghai Municipality, China

Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

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HYPERCHYLOMICRONEMIA