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Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients with Spinal Muscular Atrophy Type 1

NCT05614531 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2024-09-19

No results posted yet for this study

Summary

The purpose of this trial is to evaluate safety and efficacy of intravenous delivery of EXG001-307 as a treatment of spinal muscular atrophy Type 1 (SMN1).

Conditions

  • Spinal Muscular Atrophy Type I

Interventions

GENETIC

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
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Day
Max Age
180 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-16
Primary Completion
2025-08-31
Completion
2025-08-31

Countries

  • China

Study Locations

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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 NCT05614531 on ClinicalTrials.gov