NCT06576388

Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I

Clinical Trial Evaluating the Safety and Efficacy of EXG001-307 in Patients With Type I Spinal Muscular Atrophy

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Guangzhou Jiayin Biotech Ltd · Industry
• Sex: All
• Age: 180 Days
• Healthy volunteers: Not accepted

Summary

The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy. The research process includes the screening period (the screening period is from the time the subject signs the informed consent to the time before hormone pretreatment, with a maximum of 28 days), the treatment period (the subject receives hospitalization and observation including hormone pretreatment and single infusion of study drugs), and the follow-up period (the end of the treatment period until the subject reaches the age of 18 months, loss of follow-up, active withdrawal from the study or death). The qualified subjects in the screening period enter the treatment period, receive exg001-307 treatment, and enter the follow-up period after hospitalization observation. At the end of the study visit (subjects 18 months old), eligible subjects will be asked to transfer to the long-term follow-up study.

Conditions

• Type I Spinal Muscular Atrophy

Interventions

Type	Name	Description
DRUG	EXG001-307 injection	Research process includes filter (subjects signed informed consent for screening period before the pretreatment to the hormone, the longest 28 days), treatment period (the subjects accept including single hormone pretreatment, the study drug infusion) hospitalization and observation and follow-up treatment period (subjects to the participants to 18 months, lost to follow-up, active exit research subjects, or death). Subjects qualified in the screening period entered the treatment period and received EXG001-307, and entered the follow-up period after hospitalization. At the end of the study visit (subject at 18 months of age), eligible subjects will be asked to transfer to the long-term follow-up study.

Timeline

• Start date: 2022-01-21
• Primary completion: 2023-09-21
• Completion: 2024-01-21
• First posted: 2024-08-28
• Last updated: 2024-08-28

Locations

1 site across 1 country: China