A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

A Multicenter, Open-label, Dose-escalation Phase I/II Clinical Study to Evaluate the Safety, Preliminary Efficacy and Immunogenicity of SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I

This is a phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I.

Study Overview

• Status: Recruiting
• Conditions: Spinal Muscular Atrophy 1
• Intervention / Treatment: Genetic: SKG0201 Injection

Detailed Description

This is a multicenter, open-label, dose-escalation phase I/II clinical study to evaluate the safety, preliminary efficacy and immunogenicity of SKG0201 injection in subjects with Spinal Muscular Atrophy Type I (SMA I).

• Study Type: Interventional
• Enrollment (Estimated): 11
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Yongqin Wang; Phone Number: +86 18616737445; Email: yongqin.wang@skytx.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China
      • Recruiting
      • Peking University First Hospital
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China
      • Recruiting
      • Children's Hospital of Fudan University
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • Recruiting
      • Children's Hospital, Zhejiang University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Type I SMA, defined by bi-allelic mutations in the SMN1 gene.
2. Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control.
3. On the day of administration, the age of the subjects do not exceed 180 days after birth.

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.
3. Active viral infection.
4. In the presence of other severe infections or diseases that require systemic anti-infection treatment.
5. Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients.
6. Clinically significant abnormal laboratory values prior to administration.
7. Previously used Zolgensma or other SMA gene therapy drugs, or currently participating in other SMA clinical studies on therapeutic drugs.
8. Having previously undergone major surgery or expected to undergo major surgery during the study assessment period.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Dose-escalation; Dosage form: Injection Frequency of administration: one-time deliver	Genetic: SKG0201 Injection; SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.; Other Names: SKG0201

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence and characteristics of DLT	DLT: dose-limiting toxicity	4 weeks
Incidence of AEs and SAEs	AEs: adverse events; SAEs: serious Adverse events	to 18 months of age

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Survival rate	Survival is defined as avoidance of either death or permanent ventilation.	14 months of age
CHOP-INTEND score changes from baseline	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.	to 18 months of age
The proportion of subjects whose CHOP-INTEND score reached 40 or above	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.	to 18 months of age

Collaborators and Investigators

• Sponsor: Lanyue Biotech (Hangzhou) Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): July 15, 2026
• Primary Completion (Estimated): May 25, 2029
• Study Completion (Estimated): May 25, 2029

Study Registration Dates

• First Submitted: April 21, 2026
• First Submitted That Met QC Criteria: April 21, 2026
• First Posted (Actual): April 28, 2026

Study Record Updates

• Last Update Posted (Actual): August 26, 2026
• Last Update Submitted That Met QC Criteria: August 24, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: SMA type I; SMA I
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Spinal Cord Diseases; Motor Neuron Disease; Muscular Atrophy, Spinal; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Spinal Muscular Atrophies of Childhood
• Other Study ID Numbers: SKG0201-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No