NCT07554924 A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
| NCT ID | NCT07554924 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Lanyue Biotech (Hangzhou) Co., Ltd. |
| Condition | Spinal Muscular Atrophy 1 |
| Study Type | INTERVENTIONAL |
| Enrollment | 11 participants |
| Start Date | 2026-07-15 |
| Primary Completion | 2029-05-25 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 11 participants in total. It began in 2026-07-15 with a primary completion date of 2029-05-25.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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.
Eligibility Criteria
Inclusion Criteria: 1. Type I SMA, defined by bi-allelic mutations in the SMN1 gene. 2. Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control. 3. On the day of administration, the age of the subjects do not exceed 180 days after birth. 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. 3. Active viral infection. 4. In the presence of other severe infections or diseases that require systemic anti-infection treatment. 5. Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients. 6. Clinically significant abnormal laboratory values prior to administration. 7. Previously used Zolgensma or other SMA gene therapy drugs, or currently participating in other SMA clinical studies on therapeutic drugs. 8. Having previously undergone major surgery or expected to undergo major surgery during the study assessment period.
Contact & Investigator
Frequently Asked Questions
Who can join the NCT07554924 clinical trial?
This trial is open to participants of all sexes, up to 180 Days, studying Spinal Muscular Atrophy 1. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07554924 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT07554924 currently recruiting?
Yes, NCT07554924 is actively recruiting participants. Contact the research team at mingxiu.li@skytx.com for enrollment information.
Where is the NCT07554924 trial being conducted?
This trial is being conducted at Beijing, China, Shanghai, China, Hangzhou, China.
Who is sponsoring the NCT07554924 clinical trial?
NCT07554924 is sponsored by Lanyue Biotech (Hangzhou) Co., Ltd.. The trial plans to enroll 11 participants.