International Clinical Trial: Single-Arm, Open-Label Clinical Trial on the Safety, Tolerability, and Efficacy of GC301 Adeno-Associated Virus Injection in Neonatal Patients with Infantile Pompe Disease

Geneva, March 23 -- International Clinical Trials Registry received information related to the study (ChiCTR2600120429) titled 'Single-Arm, Open-Label Clinical Trial on the Safety, Tolerability, and Efficacy of GC301 Adeno-Associated Virus Injection in Neonatal Patients with Infantile Pompe Disease' on March 13.

Study Type: Interventional study

Study Design: Single arm

Primary Sponsor: The Seventh Medical Center of the PLA General Hospital

Condition: Neonatal Patients with Infantile Pompe Disease

Recruitment Status: Not Recruiting

Phase: N/A

Date of First Enrollment: 2026-03-13

Target Sample Size: Experimental group:4;

Countries of Recruitment: China

To know more, visit https://www.chictr.org.cn/showproj.html?proj=302132 ...