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Not yet recruiting NCT07554924

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

Phase I / Phase II Interventional Spinal Muscular Atrophy 1

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An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: SKG0201 Injection.
Who it may be relevant to
Registry conditions: Spinal Muscular Atrophy 1. Basic parameters: up to 180 Days · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

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Official title

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

Overview

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.

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).

Interventions

  • Genetic SKG0201 Injection
    SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.

Primary outcome measures

  • Incidence of AEs and SAEs [Time frame: to 18 months of age]
  • Incidence and characteristics of DLT [Time frame: 4 weeks]
Secondary outcome measures (3)
  • Survival rate [Time frame: 14 months of age]
  • CHOP-INTEND score changes from baseline [Time frame: to 18 months of age]
  • The proportion of subjects whose CHOP-INTEND score reached 40 or above [Time frame: to 18 months of age]

Eligibility criteria

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

China · 3 centers
  • Peking University First Hospital — Beijing
  • Children's Hospital of Fudan University — Shanghai
  • Children's Hospital, Zhejiang University School of Medicine — Hangzhou

Identifiers

NCT: NCT07554924 · SKG0201-101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗