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Gene Therapy Trial Report

Summary

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients


NCTID NCT05824169 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Spinal Muscular Atrophy
Disease Ontology Term DOID:13137
Drug Product Name GC101
Drug Product Description scAAV9.CBA.CMV.coSMN1
Sponsor GeneCradle Inc
Funder Type Industry
Recruitment Status
Enrollment Count 18 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant SMN1
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Intrathecal
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type AAV9
Dose 1 2.4E14 vg
Dose 2 4.8E14 vg

Study Record Dates


Current Phase Phase1, Phase2
Submit Date 2023-04-10
Completion Date 2026-12
Last Update 2025-07-03

Participation Criteria


Eligible Age 0 Months - 6 Months
Standard Ages Child
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: * Six months of age and younger on day of vector infusion with Type 1 SAM as defined by the following features: * Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2; * Onset of disease before 6 months of age * The 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 a previous gene therapy research trials; * Patient who has received Nusinersen and Risdiplam treatment; * Patient who has AAV9 neutralizing antibody titer ≥1:200; * Patient who requires non-invasive ventilatory support averaging≥16 hours/day; * Patient with a point mutation in SMN2 (c.859G\>C); * Patient who requires non-invasive ventilatory support averaging≥16 hours/day at screening; * Patient who use invasive ventilatory support or pulse oximetry \< 95% saturation while awake and calm at screening; * Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody; * Abnormal laboratory values considered clinically significant, including gamma-glutamyl transferase(GGT), Aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin \> 3x upper limit of normal (ULN), Hemoglobin (Hgb)\< 110 or \>150 g/L, platelet \<183x10\^9/L or 614x10\^9/L; * Class IV patient based on Modified Ross Heart Failure Classification for Children; * Patient with a history of glucocorticoid allergy; * Contraindication that would interfere with the lumbar puncture procedures; * Presence of an untreated active infection requiring systemic antiviral therapy at any time during the screening period; * Vaccination less than 2 weeks before infusion of vector; * Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study. Note: Other protocol defined inclusion/exclusion criteria may apply.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 4
Locations China

Regulatory Information


Has US IND False
FDA Designations Orphan Drug Designation
Recent Updates Phase 3 pivotal study for Type 2 SMA has completed enrollment

Resources/Links