A Phase 1/2 interventional study of SKG0201 Injection in Spinal Muscular Atrophy 1, sponsored by Lanyue Biotech (Hangzhou) Co., Ltd.. Recruiting at 3 sites in China. Open to participants aged Up to 180 Days. Per ClinicalTrials.gov, last updated 2026-10-14.
Sponsored by Lanyue Biotech (Hangzhou) Co., Ltd. · Phase 1/2, Interventional, and Treatment
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.
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).
61 studies on the registry are indexed under Spinal Muscular Atrophies of Childhood; 18 are open to participants now.
This study's planned enrollment of 11 is below the median of 15 across 41 interventional studies indexed under Spinal Muscular Atrophies of Childhood.
Browse Spinal Muscular Atrophies of Childhood studies →This is the only study on the registry with Lanyue Biotech (Hangzhou) Co., Ltd. as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Dosage form: Injection Frequency of administration: one-time deliver
Genetic: SKG0201 Injection
SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.
Also known as: SKG0201
Incidence of AEs and SAEs
AEs: adverse events; SAEs: serious Adverse events
Time frame: to 18 months of age
Incidence and characteristics of DLT
DLT: dose-limiting toxicity
Time frame: 4 weeks
Survival rate
Survival is defined as avoidance of either death or permanent ventilation.
Time frame: 14 months of age
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.
Time frame: to 18 months of age
The proportion of subjects whose CHOP-INTEND score reached 40 or above
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.
Time frame: to 18 months of age
Plan to share: No
No publications or documents are linked to this record.
Started Jul 2026 (actual). Primary completion is estimated for May 2029, 34 months after the start.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Spinal Muscular Atrophies of Childhood →