Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular A… (NCT06888661) | Clinical Trial Compass
By InvitationEarly Phase 1
Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy
China4 participantsStarted 2025-03-10
Plain-language summary
The purpose of this trial is to evaluate safety and efficacy of intrathecal delivery of EXG001-307 as a treatment of spinal muscular atrophy .
Who can participate
Age range
1 Day – 2 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. On the day of dosing, type 1 SMA age ≤180 days, type 2 SMA age \> 180 days ≤2 years, male or female.
. Clinical history and physical signs are consistent with SMA manifestations; SMA was diagnosed by bilateral allelic SMN1 mutation (deletion or point mutation). Type 1 has 2 copies of SMN2 gene. Type 2 has ≤3 copies of SMN2 gene.
.The subject's legal guardian understands the purpose, possible risks and interests of the study, agrees to participate in the study, completes all study procedures, tests and visits, and voluntarily signs the informed consent form.
.During the study, the subject's legal guardian was willing to perform standard treatment requirements such as nasogastric feeding, noninvasive mechanical ventilation, and expectoration machine as recommended by the investigator.
Exclusion criteria
. The presence of contraindications to lumbar puncture (including, but not limited to, signs or symptoms of skin infection at the administration site and elevated intracranial pressure), the receipt of any active intrathecal therapy, the presence of an implantable shunt tube for draining CSF, the presence of an implantable central nervous system (CSF) cannula, or any condition that interferes with CSF collection.
. Imaging shows severe scoliosis (defined as curvature of the spine ≥ 50°).
. Gestational age at birth was less than 35 weeks (245 days).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
To evaluate the safety and tolerability of EXG001-307 following a single intrathecal injection
. At screening, the subject had an oxygen saturation \< 95% while awake or sleeping and did not receive any supplemental oxygen or respiratory support.
. Requirement of invasive ventilation or tracheotomy, or current use of noninvasive ventilatory support for an average of ≥ 12 hours/day.
. Weighed below the 3rd percentile by age according to the WHO Child Growth Criteria (WHO 2009).
. Before administration, if the subject has not received or delayed vaccination according to the current month-old national vaccination plan, it will significantly affect the safety of the subject as assessed by the investigator and the medical manager of the project team;
. Active viral infections (including HIV, hepatitis B or C seropositivity, torch virus, Epstein-Barr virus, and syphilis).