Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV (NCT07398872) | Clinical Trial Compass
By InvitationPhase 1
Safety and Efficacy of AAV9. hMCOLN1co For Patients With Mucolipidosis Type IV
China1 participantsStarted 2026-01-13
Plain-language summary
Safety and Efficacy of AAV9.hMCOLN1co for patients with Mucolipidosis Type IV(MLIV): A Single-Center, Interventional, Open-Label, Single-Arm Clinical Study. The goal of this clinical trial is to evaluate whether a gene therapy can safely treat children with MLIV.
Who can participate
Age range
18 Months – 8 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
. Must be between 1.5 and 8 years of age(inclusive) at the time of signing the informed consent form
. Diagnosed with Mucolipidosis type IV caused by MCOLN1 gene mutations
. The legal guardian and/or the participant(if applicable) has signed the informed consent form
. Meet the criteria for anesthesia and sedation appropriate for their age(as assessed by the investigator)
. Has completed age-appropriate immunizations according to the National Immunization Program Schedule for Children
Exclusion criteria
. Presence of any contraindication to lumbar puncture or intrathecal therapy (e.g., spina bifida, meningitis, coagulation disorders, obstructive spinal internal fixation devices) or presence of a cerebrospinal fluid (CSF) diversion/shunting device
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 assess the types, severity, and incidence of adverse events(AEs) and serious adverse events(SAEs) following treatment
Timeframe: through study completion, an average of 5 years
Trial details
NCT IDNCT07398872
SponsorThe Children's Hospital of Zhejiang University School of Medicine
. At screening, severe joint contracture as assessed by the physiotherapist, affecting functional assessment or intrathecal administration
. X-ray examination indicating severe scoliosis (Cobb angle ≥ 50°)
. History of, or planned, scoliosis corrective surgery within 1 year before or after dosing
. Use of invasive respiratory support (e.g., tracheostomy with positive pressure ventilation) or oxygen saturation while awake \< 95% (or \< 92% at altitudes \> 1000 m)
. Requirement for ≥ 12 hours/day of non-invasive respiratory support within 2 weeks prior to dosing
. Dependence on gastric tube feeding with the majority of nutrition taken non-orally, or body weight below the 3rd percentile for age according to World Health Organization (WHO) standards (patients with an existing gastrostomy are not excluded)
. Active viral infection, including human immunodeficiency virus (HIV), hepatitis B, hepatitis C, etc.