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

Plain-language summary

This study evaluates the safety and efficacy of the gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy type 1 (SMA 1). It enrolls infants with a confirmed diagnosis based on bi-allelic mutations in the SMN1 gene who are 180 days old or younger on the day of infusion, with clinical findings consistent with type 1 SMA such as hypotonia, delayed motor skills and poor head control. The trial has no assigned phase category (NA) and is currently active but no longer recruiting.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Active, not recruiting

Sponsor

Kun Sun

Intervention

SKG0201 Injection (SKG0201)

Start date

6/25/2023

Estimated completion

12/1/2025

Countries

China

Related genes

Locations

• Chengdu, China
• Shanghai, China
• Shanghai, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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.