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Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy

NCT06888661 · Status: ENROLLING_BY_INVITATION · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2025-03-21

No results posted yet for this study

Summary

The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of EXG001-307 as a treatment of spinal muscular atrophy .

Conditions

  • Spinal Muscular Atrophy (SMA)

Interventions

BIOLOGICAL

EXG001-307 injection

non-replicating, rAAV vector based on AAV9 containing cDNA encoding the human SMN protein.

Sponsors & Collaborators

  • Hangzhou Jiayin Biotech Ltd

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Day
Max Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-03-10
Primary Completion
2026-03-31
Completion
2026-12-30

Countries

  • China

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06888661 on ClinicalTrials.gov