Spinal Muscular Atrophy 1Phase 1/2NCT07554924
A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I
A Phase 1/2 study testing SKG0201 Injection in people with Spinal Muscular Atrophy 1, run by Lanyue Biotech (Hangzhou) Co., Ltd. at 3 sites in China.
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 180 days
- Conditions the study is for
- Spinal Muscular Atrophy 1
- What is being tested?
- SKG0201 Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 3 sitesChina
- Study dates
- 15 Jul 2026to 25 May 2029
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 180 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Type I SMA, defined by bi-allelic mutations in the SMN1 gene.
- Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control.
- On the day of administration, the age of the subjects do not exceed 180 days after birth.
Who cannot join
- Pulse oximetry < 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.
- Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards.
- Active viral infection.
- In the presence of other severe infections or diseases that require systemic anti-infection treatment.
- Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients.
- Clinically significant abnormal laboratory values prior to administration.
- Previously used Zolgensma or other SMA gene therapy drugs, or currently participating in other SMA clinical studies on therapeutic drugs.
- Having previously undergone major surgery or expected to undergo major surgery during the study assessment period.
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China3 sites, 3 recruiting
- Recruiting
Peking University First Hospital
Beijing, Beijing Municipality
- Recruiting
Children's Hospital, Zhejiang University School of Medicine
Hangzhou, Zhejiang
- Recruiting
Children's Hospital of Fudan University
Shanghai, Shanghai Municipality
About this study
Official title: 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
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.
Read the detailed description
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).
- Conditions
- Spinal Muscular Atrophy 1
- Being tested
- SKG0201 Injection
- Keywords
- SMA type I, SMA I
- Medical subject headings
- Spinal Muscular Atrophies of Childhood
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SKG0201 InjectionGenetic
Also called SKG0201
SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.
| Study group | Role | What they receive |
|---|---|---|
| Dose-escalation | Experimental | Dosage form: Injection Frequency of administration: one-time deliver Receives: Genetic: SKG0201 Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of AEs and SAEs
AEs: adverse events; SAEs: serious Adverse events
Measured: to 18 months of age
Incidence and characteristics of DLT
DLT: dose-limiting toxicity
Measured: 4 weeks
Secondary outcomes (3)
Survival rate
Survival is defined as avoidance of either death or permanent ventilation.
Measured: 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.
Measured: 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.
Measured: to 18 months of age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Apr 2026
- Study start15 Jul 2026
- Primary completion25 May 2029 (estimated)
- Study completion25 May 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular Atrophy 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 11 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07554924
- Sponsor's protocol ID
- SKG0201-101
- Status last verified
- Oct 2026
- First submitted
- 21 Apr 2026
- Last updated
- 14 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT07554924
- Acronym / sponsor ID
- SKG0201-101
- Conditions
- Spinal Muscular Atrophy 1
- Keywords
- SMA type I, SMA I
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 11 participants (estimated)
- Lead sponsor
- Lanyue Biotech (Hangzhou) Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 15 Jul 2026 (actual)
- Primary completion
- 25 May 2029 (estimated)
- Study completion
- 25 May 2029 (estimated)
- First posted
- 28 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose-escalation | Experimental | Genetic: SKG0201 InjectionDosage form: Injection Frequency of administration: one-time deliver |
Who may qualify
- Ages
- Up to 180 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Type I SMA, defined by bi-allelic mutations in the SMN1 gene.
- Clinical history and signs are consistent with type I SMA, such as hypotonia, delayed motor function development, and poor head control.
- On the day of administration, the age of the subjects do not exceed 180 days after birth.
Key exclusion criteria (8 listed)
- Pulse oximetry < 96% saturation at screening while the patient is awake or asleep without any supplemental oxygen or respiratory support.
- Weight-for-age below the 3rd percentile for the same sex and age based on WHO Child Growth Standards.
- Active viral infection.
- In the presence of other severe infections or diseases that require systemic anti-infection treatment.
- Known allergy to prednisolone, other glucocorticoids, or SKG0201's excipients.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SKG0201 InjectionAlso: SKG0201SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product. | Genetic | Dose-escalation |
Primary outcome
Incidence of AEs and SAEs
Time frame: to 18 months of age
AEs: adverse events; SAEs: serious Adverse events
Incidence and characteristics of DLT
Time frame: 4 weeks
DLT: dose-limiting toxicity
3 secondary outcomes
Survival rate
Time frame: 14 months of age
CHOP-INTEND score changes from baseline
Time frame: to 18 months of age
The proportion of subjects whose CHOP-INTEND score reached 40 or above
Time frame: to 18 months of age
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing SKG0201 Injection in people with Spinal Muscular Atrophy 1, run by Lanyue Biotech (Hangzhou) Co., Ltd. at 3 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mingxiu Li (+86 19121738351, mingxiu.li@skytx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of AEs and SAEs (time frame: to 18 months of age). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Oct 2026.