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

RECRUITINGPHASE1/PHASE2

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

A Multi-center, Open Label, Single-arm, Dose Ascending Clinical Trial for Evaluation of Safety and Efficacy of Gene Therapy Drug GC101 in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients

NCT05824169•GeneCradle Inc

View on ClinicalTrials.gov

Summary

The study will evaluate safety and efficacy of intrathecal delivery of GC101 gene therapy drug as a treatment of spinal muscular atrophy Type 1 (SMA 1) patients.

Detailed Description

The purpose of this trial is to evaluate safety and efficacy of gene therapy drug GC101 in SMA 1 patients. Open-label, dose-escalation clinical trial of GC101 will be conducted in multiple centers in China. GC101 will be administrated intrathecally. Short-term safety will be evaluated in 52 weeks and enter long-term follow-up study of 5 years at will. Patients will be tested at baseline and followed up on various time points. The primary analysis for efficacy will be assessed when all patients reach 18 months of age on the motor milestone of sit unassisted for at least 10 seconds.

Eligibility

Age

0 - 0 years

Sex

ALL

Healthy Volunteers

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;
+5 more criteria

Locations (4)

Peking University， First Hospital, Department of Pediatrics

Beijing, China

Bayi Children's Hospital, Seventh Medical Center, PLA general hospital

Beijing, China

Shengjing Hospital of China Medical University

Shenyang, China

Children's Hospital of Soochow University

Suzhou, China

Key Dates

Start Date

February 25, 2023

Primary Completion Date

December 1, 2026

Completion Date

December 1, 2026

Last Updated

July 3, 2025

Enrollment

ESTIMATED participants

Conditions

Spinal Muscular Atrophy

Interventions

GC101

GENETIC

Contacts

GeneCradle, Inc. China

CONTACT

86-13501380583 ind@bj-genecradle.com

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: July 3, 2025Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

Long Read Analysis in Spinal Muscular Atrophy - LOREASI

Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).

Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases

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