Trials Sponsors Conditions Drugs Pricing

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 studiesOther

No intervention, only for observational studies

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age1 Month+

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-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;

Sponsors & Collaborators

Guangzhou Jiayin Biotech Ltd
Industrylead

Study Sites (1)

The Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

Single Person

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Intervention Hierarchy (Ancestors)

Marital StatusFamily CharacteristicsDemographyPopulation Characteristics

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 2025

Estimated (date passed)

This estimate has passed. The registry has not confirmed an actual date for this milestone.

Primary Completion

September 10, 2030

Estimated

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (5)

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Keywords

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (5)

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Study Officials

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations