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Study identifier: NCT06421831 Synced from ClinicalTrials.gov · July 29, 2026
● Recruiting

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients

Condition: Spinal Muscular Atrophy Type 3  ·  Sponsor: GeneCradle Inc

PhasePhase 1/Phase 2
Planned participants21
Who can joinAll sexes, 2 Years to no upper limit
Healthy volunteersNo

About this study

The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 3 (SMA 3) patients.

This description comes directly from the study's public registry record.

Talk to the study team

GeneCradle, Inc China  ·  +8613501380583  ·  ind@bj-genecradle.com

Always discuss trial participation with your own doctor first.

Locations (1)

Beijing Tiantan Hospital, Capital Medical UniversityBeijing, Beijing Municipality, ChinaRecruiting

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Source record: clinicaltrials.gov/study/NCT06421831