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Gene Therapy Trial Report

Summary

A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1


NCTID NCT06191354 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Spinal Muscular Atrophy
Disease Ontology Term DOID:13137
Compound Name SKG0201
Sponsor Kun Sun
Funder Type Other
Recruitment Status
Active not recruiting
Enrollment Count 12 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant SMN1
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV
Dose 1 Undisclosed dose escalation

Study Record Dates


Current Stage Na
Submit Date 2023-12-19
Completion Date 2025-12
Last Update 2025-11-21

Participation Criteria


Eligible Age <=180 Days
Standard Ages Child
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Type 1 SMA, defined by bi-allelic mutations in the SMN1 gene. 2. Age 180 days or younger at day of infusion. 3. Clinical history and signs are consistent with type I SMA, that is hypotonia on clinical examination, with delay in motor skills, poor head control, rounded shoulder posture, and joint hypermobility. 4. The legal guardian of the subject understands the purpose of the study, the possible risks and rights of the study, agrees that the subject can participate in the study, complete all research steps, tests and visits, and sign the ICF voluntarily. 5. During the study period, according to the change of the subject's condition, the subject's legal guardian is willing to perform standard treatment requirements as suggested by the researcher. Exclusion Criteria: 1. Pulse oximetry \< 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support. 2. Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards (WHO 2006). 3. Active viral infection with significant signs or symptoms and require systematic hospitalization. 4. In the presence of other severe infections or diseases. 5. Known allergy to prednisolone, other glucocorticoids, or their excipients. 6. Clinically significant abnormal laboratory values prior to administration. 7. Previously used other SMA drugs (such as Spinraza, Evrysdi, Zolgensma, etc.) or participated in clinical studies of other SMA drugs. 8. Had received previous or anticipated major surgical procedures during the study assessment period.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations China

Regulatory Information


Has US IND False
FDA Designations
Recent Updates

Resources/Links