Evaluating SKG0201 Injection for Spinal Muscular Atrophy Type 1

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

This study is testing a new gene therapy called SKG0201 Injection to see if it can safely help people with spinal muscular atrophy Type 1.

Quick facts

• Phase: NA
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: N/A to 180 Days
• Sex: All
• Sponsor: Xinhua Hospital, Shanghai Jiao Tong University School of Medicine (other)
• Locations: 3 sites (Chengdu, Sichuan and 2 other locations)
• Trial ID: NCT06191354 on ClinicalTrials.gov

What this trial studies

This clinical study assesses the safety and efficacy of the gene therapy drug SKG0201 Injection in patients diagnosed with spinal muscular atrophy Type 1 (SMA 1). It is a multicenter, open-label, dose-escalation study designed to gather initial efficacy data and evaluate the immunogenicity of the treatment. Participants will receive the injection and be monitored for safety and response to the therapy over the course of the study.

Who should consider this trial

Good fit: Ideal candidates are infants aged 180 days or younger with a confirmed diagnosis of Type 1 SMA due to bi-allelic mutations in the SMN1 gene.

Not a fit: Patients with significant respiratory issues or those who do not meet the weight criteria may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve motor function and quality of life for infants with spinal muscular atrophy Type 1.

How similar studies have performed: Other studies have shown promise with gene therapy approaches for SMA, indicating potential for success with this novel treatment.

Eligibility criteria

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.

Where this trial is running

Chengdu, Sichuan and 2 other locations

• West China Sencond Hospital, Sichuan University / West China women's and children's Hospital — Chengdu, Sichuan, China (RECRUITING)
• Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine — Shanghai, China (RECRUITING)
• National Children's Medical Center, Children's Hospital of Fudan University — Shanghai, China (NOT_YET_RECRUITING)

Study contacts

• Principal investigator: Kun Sun, Doctor — Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
• Study coordinator: Yongguo Yu, Doctor

Conditions: Spinal Muscular Atrophy 1, SMA 1