Gene therapy for treating spinal muscular atrophy type 3

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the safety and efficacy of the GC101 gene therapy drug administered intrathecally to patients with spinal muscular atrophy type 3 (SMA 3). It is an open-label, dose-escalation study conducted across multiple centers in China. Participants will be monitored for short-term safety over 52 weeks and will enter a long-term follow-up phase lasting five years. Efficacy will be assessed at 12 months post-treatment using the Hammersmith Functional Motor Scale Expanded (HFMSE) and Revised Upper Limb Module (RULM) scores.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* ≥2 years of age on the day of signing the informed consent form;
* Genetic and clinical diagnosis of type 3 SMA with bi-allelic deletion of SMN1 of 5qSMA;
* Hammersmith Functional Motor Scale - Expanded (HFMSE) score is between 10 and 54 at screening;
* Female patients of childbearing age who are pregnant or lactating, as well as all enrolled patients （both male and female), should take effective contraceptive measures within 6 months after the treatment;
* Patients or patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.

Exclusion Criteria:

* Patient who has participated in any previous gene therapy research trials;
* Patient who has AAV9 neutralizing antibody titer ≥1:200;
* Patient who has received Nusinersen within 120 days and Risdiplam within 15 days before treatment;
* Patient who requires invasive or non-invasive ventilatory support averaging≥16 hours/day at screening;
* SMN2 copy numbers \>4；
* Patient who needs nasal or gastric tube feeding for eating;
* Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
* Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients
* Severe contractures at screening that interfere with either the ability to attain/demonstrate functional measures or with the ability to receive intrathecal (IT) dosing;
* Patient who has other serious diseases, such as severe cardiovascular and cerebrovascular diseases, digestive system diseases, urinary system diseases, endocrine system diseases, hematological diseases, immune system diseases, nervous system diseases (including but not limited to epilepsy, meningitis, history of convulsions or seizures, cerebrospinal fluid circulation disorders), and mental illnesses, etc.;
* Patient with previous injuries (such as upper or lower limb fractures) or surgical operations that have not fully recovered or reached a stable state;
* Vaccination no longer than 2 weeks before treatment;
* Patient who has any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.

Where it's running

• Beijing Tiantan Hospital, Capital Medical University
  Beijing, Beijing Municipality, China Recruiting

Study contacts

• Study coordinator: GeneCradle, Inc China
• Email: ind@bj-genecradle.com
• Phone: +8613501380583

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Linking walking patterns to bone strength in ambulatory SMA

  This will try to see if wearable insole gait data can predict bone density and fracture risk in ambulatory people with spinal muscular atrophy who are on disease-modifying therapy.

  Recruiting Columbia University · New York, New York · NCT07400198

• Apitegromab for infants under 2 with spinal muscular atrophy

  This trial will test whether adding apitegromab to approved SMA therapy helps infants under 2 with 5q SMA reach better motor milestones.

  Recruiting at 23 sites Phase 2 Scholar Rock, Inc. · Phoenix, Arizona and 22 other locations · NCT07047144

• Identifying walking-related digital biomarkers in rare childhood neuromuscular disorders

  This study is testing if special insoles with sensors can help track walking patterns in kids with spinal muscular atrophy or Duchenne muscular dystrophy to better understand their mobility.

  Recruiting at 3 sites Columbia University · Palo Alto, California and 2 other locations · NCT06839469

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.