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

Not Applicable

Recruiting

• Conditions: Spinal Muscular Atrophy 1
• Interventions: Genetic: SKG0201 Injection

• Registration Number: NCT06191354
• Lead Sponsor: Kun Sun

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).

Detailed Description

This is a multicenter, open, dose-escalation clinical study to evaluate the safety, initial efficacy, and immunogenicity of SKG0201 injection in patients with spinal muscular atrophy type 1 (SMA 1).

Study Timeline

• Study Start: 2023-06-25
• Study First Submitted: 2023-12-19
• Study First Submitted QC: 2023-12-19
• Study First Posted: 2024-01-05
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-30
• Last Update Posted: 2024-05-01
• Primary Completion: 2025-06
• Study Completion: 2025-12

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 12

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Dose-escalation	SKG0201 Injection	SKG0201 one-time deliver

Primary Outcome Measures

Name	Time	Method
Incidence of AEs and SAEs	18 months of age	AEs: adverse events; SAEs: serious Adverse events
Incidence and characteristics of DLT	4 weeks	DLT: dose-limiting toxicity

Secondary Outcome Measures

Name	Time	Method
CHOP-INTEND score changes from baseline	24 weeks	CHOP-INTEND (Children's hospital of Philadelphia Infant Test of Neuromuscular Disorders) score ranges from 0 to 64 with higher scores indicating higher motor function.
Proportion of subjects who achieve developmental milestones with improvement in exercise intensity and function assessed according to BSID-III	24 weeks	Developmental milestones are defined according to BSID-III (Bayley Scales of Infant and Toddler Development Third Edition) criteria.
Survival rate	14 months of age	Survival is defined as avoidance of either death or permanent ventilation.

Trial Locations

Locations (3)

National Children's Medical Center, Children's Hospital of Fudan University; 🇨🇳 Shanghai, China

West China Sencond Hospital, Sichuan University / West China women's and children's Hospital; 🇨🇳 Chengdu, Sichuan, China

Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine; 🇨🇳 Shanghai, China