Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the Treatment of Patients With Type 2 Spinal Muscular Atrophy (SMA) - Phase III
NCT ID: NCT06971094
Last Updated: 2025-06-05
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
50 participants
INTERVENTIONAL
2025-05-27
2026-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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GC101
single dose of GC101 at dosage of 1.2E+14 vg per person via intrathecal injection
GC101 adeno-associated virus injection
Self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene
Control
Participants will continue to receive treatment according to their previous standard-of-care regimen with nusinersen.
No interventions assigned to this group
Interventions
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GC101 adeno-associated virus injection
Self-complementary recombinant adeno-associated viral vector (scAAV) containing a single-stranded transgene encoding a codon-optimized human SMN1 gene
Eligibility Criteria
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Inclusion Criteria
* Patients who have been receiving regular treatment with nusinersen for more than one year prior to screening.
* Patients who have not received treatment with risdiplam within 2 months prior to screening and have no plans to receive risdiplam treatment within 12 months after enrollment.
* Patients who can sit independently but cannot walk independently at the time of screening (according to the definitions of independent sitting and walking in the WHO-MGRS motor milestones scale), and have an HFMSE score of ≥10 points.
* Patients and/or their legal guardians are able to understand and are willing to comply with the requirements and procedures of the trial protocol, and voluntarily participate and sign the informed consent form
Exclusion Criteria
* Patients who have received nusinersen treatment within 2 months prior to enrollment.
* Patients with any medical conditions that may affect the interpretation of study results or pose a risk to the safety of the participants, including but not limited to organ dysfunction of any cause, acute infectious diseases, primary/acquired immunodeficiency diseases, severe cardiovascular/cerebrovascular diseases, gastrointestinal diseases, diabetes, known epilepsy, meningitis, seizure or convulsion history, or a family history of psychiatric disorders; and those with cerebrospinal fluid circulation disorders.
* Patients with severe liver injury/hepatic insufficiency of any cause, including but not limited to alanine aminotransferase (ALT), aspartate aminotransferase (AST) ≥3 times the upper limit of normal (ULN); total bilirubin (TBil) ≥1.5 times the ULN.
* Patients deemed by the investigator to have contraindications to glucocorticoid use, such as severe hypertension, diabetes, systemic infectious diseases, fungal infections, glaucoma, osteoporosis, peptic ulcer disease, tuberculosis, etc.
* Patients with contraindications to lumbar puncture or intrathecal injection therapy.
* Patients with any medical conditions that may affect the assessment of motor function, such as severe scoliosis, severe joint contracture deformities, planned spinal correction surgery during the trial period, severe osteoporosis, or a history of fractures.
* Patients positive for hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibodies, hepatitis C virus (HCV) antibodies, or syphilis antibodies.
* Patients who have received vaccinations within 2 weeks prior to dosing.
* Patients who have previously received gene therapy or participated in any clinical trial within 3 months prior to screening.
* Patients deemed by the investigator to be unsuitable for participation in this study.
2 Years
12 Years
ALL
No
Sponsors
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GeneCradle Inc
INDUSTRY
Responsible Party
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Locations
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The Seventh Medical Center of Chinese PLA General Hospital
Beijing, , China
Children's Medical Center of Peking University First Hospital
Beijing, , China
Beijing Children's Hospital, Capital Medical University
Beijing, , China
National Children's Medical Center，Shanghai Jiaotong University
Shanghai, , China
Shenzhen Children's Hospital
Shenzhen, , China
Children's Hospital of Soochow University
Suzhou, , China
Wuhan Children's Hospital, Tongji Medical College, Huazhong University of Science & Technology
Wuhan, , China
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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JLJY-GC101-SMA-011
Identifier Type: -
Identifier Source: org_study_id
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