FDA Clears Way for KIK-AS Phase 1/2 Trial to Resume

FDA Clears Way for KIK-AS Phase 1/2 Trial to Resume

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FDA Clears Way for KIK-AS Phase 1/2 Trial to Resume

The U.S. Food and Drug Administration (FDA) has lifted the clinical hold on a Phase 1/2 study testing GTX-102 — an investigational therapy by GeneTx Biotherapeutics and Ultragenyx Pharmaceutical — in children and adolescents with Angelman syndrome. GeneTx is now cleared to begin dosing treatment-naïve patients — those who have never received GTX-102 — in the U.S. The study (NCT04259281), known as KIK-AS, was placed on a clinical hold late last year after five patients in the U.S. experienced serious,…

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