A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

18 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-03-31

Study Completion Date

2030-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

An Open-label, Long-term Follow-Up Study to Evaluate the Safety and Tolerability of Gene Therapy with EXG001-307 in SMA1 patients ,who joined the parent study (EXG001-307-102)

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

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

NCT06888661

Spinal Muscular Atrophy (SMA)

ENROLLING_BY_INVITATION EARLY_PHASE1

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

NCT05614531

Spinal Muscular Atrophy Type I

ENROLLING_BY_INVITATION PHASE1/PHASE2

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

NCT06576388

Type I Spinal Muscular Atrophy

COMPLETED EARLY_PHASE1

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

NCT05824169

Spinal Muscular Atrophy

RECRUITING PHASE1/PHASE2

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients

NCT05901987

SMA II

ACTIVE_NOT_RECRUITING PHASE1/PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

This study plans to enroll type 1 SMA patients who have previously received EXG001-307 treatment in the parent trial EXG001-307-102 to observe the long-term safety and efficacy of gene therapy with EXG001-302.

The first year of long-term follow-up will be conducted every 3 months ; In the second year, follow-up visits will be conducted every six months, with telephone follow ups at 15 and 21 months during this period; From 3 to 5 years after gene therapy, follow up by phone every six months and follow on on site once a year.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Spinal Muscular Atrophy

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

OTHER

Study Time Perspective

OTHER

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

No intervention, only for observational studies

Intervention Type OTHER

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

1. Previously received treatment with EXG001-307 in trial EXG001-307-102;
2. Parents or guardians understand the research procedure and sign the ICF; Good compliance and willingness to follow research procedures. Voluntarily participate in this clinical trial.