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

NCT07554924 | Not Yet Recruiting Phase 1

• 1 other identifier: SKG0201-101
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 4

Brief Summary

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.

Conditions

Spinal Muscular Atrophy 1

Keywords

SMA type I; SMA I

Outcome Measures

Primary Outcomes (2)

• Incidence of AEs and SAEs

  AEs: adverse events; SAEs: serious Adverse events

  to 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

  to 18 months of age

• The proportion of subjects whose CHOP-INTEND score reached 40 or above

  to 18 months of age

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

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

Sponsors & Collaborators

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

Study Sites (4)

Peking University First Hospital

Beijing, Beijing Municipality, China

Location

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, China

Location

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine

Shanghai, Shanghai Municipality, China

Location

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 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

Central Study Contacts

Yongqin Wang

CONTACT

+86 18616737445; yongqin.wang@skytx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 21, 2026
• First Posted: April 28, 2026
• Study Start (Estimated): May 15, 2026
• Primary Completion (Estimated): May 25, 2029
• Study Completion (Estimated): May 25, 2029
• Last Updated: April 28, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (4)