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

NCT06191354 | Active Not Recruiting DMC

• 1 other identifier: SKG0201-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 3

Brief Summary

This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).

Conditions

Spinal Muscular Atrophy 1

Keywords

SMA 1

Outcome Measures

Primary Outcomes (2)

• Incidence of AEs and SAEs

  AEs: adverse events; SAEs: serious Adverse events

  18 months of age

• Incidence and characteristics of DLT

  DLT: dose-limiting toxicity

  4 weeks

Secondary Outcomes (3)

• Survival rate

  14 months of age

• CHOP-INTEND score changes from baseline

  24 weeks

• Proportion of subjects who achieve developmental milestones with improvement in exercise intensity and function assessed according to BSID-III

  24 weeks

Study Arms (1)

Dose-escalation

EXPERIMENTAL

SKG0201 one-time deliver

Genetic: SKG0201 Injection

Interventions

SKG0201 Injection GENETIC

SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based in vivo gene therapeutic product.

Also known as: SKG0201

Dose-escalation

Eligibility Criteria

• Age: Up to 180 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Type 1 SMA, defined by bi-allelic mutations in the SMN1 gene.
• Age 180 days or younger at day of infusion.
• 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.
• 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.
• 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.

You may not qualify if:

• Pulse oximetry \< 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.
• Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards (WHO 2006).
• Active viral infection with significant signs or symptoms and require systematic hospitalization.
• In the presence of other severe infections or diseases.
• Known allergy to prednisolone, other glucocorticoids, or their excipients.
• Clinically significant abnormal laboratory values prior to administration.
• Previously used other SMA drugs (such as Spinraza, Evrysdi, Zolgensma, etc.) or participated in clinical studies of other SMA drugs.
• Had received previous or anticipated major surgical procedures during the study assessment period.

Sponsors & Collaborators

• Kun Sun: lead

Study Sites (3)

West China Sencond Hospital, Sichuan University / West China women's and children's Hospital

Chengdu, Sichuan, China

Location

Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine

Shanghai, 200092, China

Location

National Children's Medical Center, Children's Hospital of Fudan University

Shanghai, China

Location

MeSH Terms

Conditions

Spinal Muscular Atrophies of Childhood

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Kun Sun, Doctor

  Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

• Yongguo Yu, Doctor

  Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor of Department of Pediatric Cardiology

Study Record Dates

• First Submitted: December 19, 2023
• First Posted: January 5, 2024
• Study Start: June 25, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: November 21, 2025

Record last verified: 2025-11

Locations

CN (3)