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

NCT06588803 | Enrolling By Invitation

• 1 other identifier: EXG001-307-LTFU
• Study Type: observational
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

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)

Conditions

Spinal Muscular Atrophy

Keywords

SMA

Outcome Measures

Primary Outcomes (1)

• long-term safety：Assess the types, severity, and incidence of serious adverse events (SAEs) and adverse events of particular concern (AESIs)

  Assess the types, severity, and incidence of serious adverse events (SAEs) and adverse events of particular concern (AESIs)

  4 years

Secondary Outcomes (5)

• Evaluate the long-term effectiveness of EXG001-307 treatment: assess the achievement of exercise milestones based on the scale

  4 years

• Evaluate the long-term effectiveness of EXG001-307 treatment: evaluate event free survival rate

  4 years

• Evaluate the immunogenicity of EXG001-307 after treatment, including measuring the titers of anti-AAV9-ADA and anti-SMN-ADA antibodies.

  4 years

• Evaluate the distribution of viral vectors after treatment with EXG001-307: detect the level of vector genome in saliva, feces, and urine samples of subjects.

  4 years

• Exploratory objective: To calculate the proportion of subjects receiving other SMA treatments during the trial period

  4 years

Interventions

No intervention, only for observational studies OTHER

No intervention, only for observational studies

Eligibility Criteria

• Age: 1 Month+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Type 1 SMA patients who have previously used EXG001-307 in trial EXG001-307-102 .

You may qualify if:

• Previously received treatment with EXG001-307 in trial EXG001-307-102;
• 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.

You may not qualify if:

• \. Parents or guardians are unwilling or unable to participate in long-term follow-up studies.

Sponsors & Collaborators

• Hangzhou Jiayin Biotech Ltd: lead

Study Sites (1)

The Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

Single Person

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Intervention Hierarchy (Ancestors)

Marital Status; Family Characteristics; Demography; Population Characteristics; Socioeconomic Factors

Study Officials

• YI WANG, Ph D.

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 21, 2024
• First Posted: September 19, 2024
• Study Start: March 1, 2025
• Primary Completion (Estimated): September 10, 2030
• Study Completion (Estimated): December 31, 2030
• Last Updated: March 5, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)