This study is a multicenter,randomized,standard-of-care-controlled,open-label clinical trial.It has been approved by the Ethics Committee of the Seventh Medical Center of the General Hospital of the People's Liberation Army,which has taken the lead in initiating patient recruitment.Other research centers will follow suit.The participating research centers include the Seventh Medical Center of the General Hospital of the People's Liberation Army,Peking University First Hospital,Beijing Children's Hospital Affiliated to Capital Medical University,Wuhan Children's Hospital Affiliated to Huazhong University of Science and Technology,Shanghai Children's Medical Center Affiliated to Shanghai Jiao Tong University School of Medicine,Suzhou University Affiliated Children's Hospital,and Shenzhen Children's Hospital.
Project Introduction
GC101 Adeno-Associated Virus Injection is a gene therapy drug currently in clinical trials.It is administered via a single intrathecal injection through lumbar puncture,delivering the gene that expresses the SMN1 protein into the cells of SMA patients,particularly into the spinal cord anterior horn motor neurons,to express normal human SMN protein and thereby achieve the therapeutic goal.
Seventh Medical Center of the General Hospital of the People's Liberation Army
Recruitment Information
The Seventh Medical Center of the General Hospital of the People's Liberation Army is leading a"Multicenter,Randomized,Standard-of-Care-Controlled,Open-Label Pivotal Clinical Trial to Evaluate the Efficacy and Safety of Intrathecal(IT)Injection of GC101 Adeno-Associated Virus Injection in Patients with Type 2 Spinal Muscular Atrophy(SMA)."This clinical study has been approved by the National Medical Products Administration(Approval Number:CXSL2200400)and the hospital's Ethics Committee.It has now entered the Phase III clinical study stage and is publicly recruiting participants.Patients who are assessed and meet the requirements will have the opportunity to participate in this study.
Note:The study drug is not yet marketed.
Project Introduction
GC101 Adeno-Associated Virus Injection(hereinafter referred to as GC101)is a gene therapy drug currently in clinical trials.It is administered via a single intrathecal injection through lumbar puncture,delivering the gene that expresses the SMN1 protein into the cells of SMA patients,particularly into the spinal cord anterior horn motor neurons,to express normal human SMN protein and thereby achieve the therapeutic goal.
Eligibility Criteria for Participants
This study will recruit 50 patients with Type 2 spinal muscular atrophy(SMA)through a competitive enrollment process nationwide.If you/your child meet the following criteria,you may have the opportunity to participate in this study:
• Age≥2 years and≤12 years,regardless of gender.
• Patients with a confirmed diagnosis of Type 2 5q-SMA through clinical phenotype and genetic testing.
• Patients who have been receiving regular nusinersen treatment for more than 1 year prior to screening.
• Patients who have not received risdiplam treatment 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,with an HFMSE score≥10 points at screening.
• Patients and/or their legal guardians who understand and are willing to comply with the requirements and procedures of the trial protocol,and voluntarily participate and sign the informed consent form.
The study physician will determine whether you/your child is eligible to participate in this study based on your/your child's specific circumstances.
Your decision to participate in this study/whether to allow your child to participate in this clinical study is voluntary,and you have the right to choose to withdraw at any time.
Requirements for Participants During the Study
During the study,you will need to do the following:
• Provide a truthful medical history and treatment history for your child,along with any necessary medical documentation.
• Follow the treatment,follow-up,and record-keeping procedures as specified in the protocol.
• Do not participate in other clinical trials during the study period.
• Follow the medical guidance of the study physician.
Contact Information
If you are interested in this study,please scan/recognize the QR code below for detailed registration information and contact details:
About GC101 Adeno-Associated Virus Injection
GC101 Injection is the first domestically developed AAV gene therapy product for intrathecal administration to treat SMA.The indications cover Types 1,2,and 3 of 5q SMA.The successful completion of the I/II phase clinical trial of the GC101-2 Type IND project has laid the foundation for the Phase III pivotal clinical trial.Clinical trial data have shown positive and significant therapeutic effects in Type 2 SMA subjects treated with GC101 Injection,with several subjects achieving breakthroughs in motor milestones.Compared to existing treatments,GC101 Injection's potential for"one-time treatment,long-term efficacy"demonstrates a differentiated advantage.In December 2024,GC101 Injection was recognized as a breakthrough therapy by the Center for Drug Evaluation(CDE)of the National Medical Products Administration.
About Genecradle Therapeutics
Beijing Genecradle Therapeutics Technology Co.,Ltd.is a national high-tech enterprise with core business in the development of gene therapy drugs mediated by AAV vector delivery technology.The company is committed to advancing rare disease gene drugs from the laboratory to clinical and market applications,benefiting patients and their families.It specializes in the development of gene therapy drugs for genetic neuromuscular diseases,inherited metabolic diseases,lysosomal diseases,and ophthalmic diseases.By promoting the research and clinical application of rare disease gene drugs,the company aims to deepen the understanding of health and life,transitioning gene therapy technology and products from rare diseases to the treatment and rehabilitation of chronic and other major diseases.