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Clinical trial

A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

NCT05824169 is registered in ClinicalTrials.gov with GeneCradle Inc as lead sponsor.

NCT NCT05824169
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Registry record

As filed in ClinicalTrials.gov.

Title
A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
Lead sponsor
GeneCradle Inc
Phase
Phase 1/2
Overall status
Recruiting
Start date
February 25, 2023
Completion date
December 31, 2026
Enrollment
18 (estimated)
Sites
4

Conditions

As the registry lists them; a condition with its own page is linked.

spinal muscular atrophy