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

Spinal Muscular Atrophy 1 Clinical Trial

Official title:

A Multicenter, Open, Dose-escalation Clinical Study Evaluating the Safety, Initial Efficacy, and Immunogenicity of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06191354
• Other study ID #: SKG0201-001
• Status: Recruiting
• Phase: N/A
• Start date: June 25, 2023
• Est. completion date: December 2025

Study information

• Verified date: April 2024
• Source: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
• Contact: Yongguo Yu, Doctor
• Phone: +86 13801794114
• Email: yuyongguo[@]shsmu.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. completion date: December 2025
• Est. primary completion date: June 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 180 Days

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Muscular Atrophy
• Muscular Atrophy, Spinal
• Spinal Muscular Atrophy 1

Intervention

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

Locations

Country	Name	City	State
China	West China Sencond Hospital, Sichuan University / West China women's and children's Hospital	Chengdu	Sichuan
China	National Children's Medical Center, Children's Hospital of Fudan University	Shanghai
China	Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
Kun Sun

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of AEs and SAEs	AEs: adverse events; SAEs: serious Adverse events	18 months of age
Primary	Incidence and characteristics of DLT	DLT: dose-limiting toxicity	4 weeks
Secondary	Survival rate	Survival is defined as avoidance of either death or permanent ventilation.	14 months of age
Secondary	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.	24 weeks
Secondary	Proportion of subjects who achieve developmental milestones with improvement in exercise intensity and function assessed according to BSID-III	Developmental milestones are defined according to BSID-III (Bayley Scales of Infant and Toddler Development Third Edition) criteria.	24 weeks