Clinical trial NCT06588803: A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA
Official title: A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 Spinal Muscular Atrophy (SMA)
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Spinal Muscular Atrophy
Keywords: SMA
Lead sponsor: Hangzhou Jiayin Biotech Ltd
Sponsor class: INDUSTRY
First posted: 2024-09-19
Enrollment: 18
Sex: ALL
Minimum age (years): 1
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Type 1 SMA patients who have previously used EXG001-307 in trial EXG001-307-102 .

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)

Detailed description: 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.

Eligibility criteria: 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.

Exclusion Criteria:

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

Primary outcomes:
measure: long-term safety：Assess the types, severity, and incidence of serious adverse events (SAEs) and adverse events of particular concern (AESIs)
timeFrame: 4 years
Secondary outcomes:
measure: Evaluate the long-term effectiveness of EXG001-307 treatment: assess the achievement of exercise milestones based on the scale
timeFrame: 4 years
measure: Evaluate the long-term effectiveness of EXG001-307 treatment: evaluate event free survival rate
timeFrame: 4 years
measure: Evaluate the immunogenicity of EXG001-307 after treatment, including measuring the titers of anti-AAV9-ADA and anti-SMN-ADA antibodies.
timeFrame: 4 years
measure: 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.
timeFrame: 4 years
measure: Exploratory objective: To calculate the proportion of subjects receiving other SMA treatments during the trial period
timeFrame: 4 years
Other outcomes:
Locations:
city: Shanghai
country: China
facility: The Children's Hospital of Fudan University
state: Shanghai
Study officials:
affiliation: Children's Hospital of Fudan University
name: YI WANG, Ph D.
role: PRINCIPAL_INVESTIGATOR
References: