Spinal Muscular Atrophy 1NCT06191354
A Clinical Study Evaluating the Safety and Efficacy of SKG0201 Injection in Patients With Spinal Muscular Atrophy Type 1
A study testing SKG0201 Injection in people with Spinal Muscular Atrophy 1, run by Kun Sun at 3 sites in China.
Trial summary
This is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1). 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
- 12 participants
- Where
- 3 sitesChina
- Study dates
- 25 Jun 2023to Dec 2025
- Phase
- Interventional
- 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 1 SMA, defined by bi-allelic mutations in the SMN1 gene.
- Age 180 days or younger at day of infusion.
- 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.
- 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.
- 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.
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 (WHO 2006).
- Active viral infection with significant signs or symptoms and require systematic hospitalization.
- In the presence of other severe infections or diseases.
- Known allergy to prednisolone, other glucocorticoids, or their excipients.
- Clinically significant abnormal laboratory values prior to administration.
- Previously used other SMA drugs (such as Spinraza, Evrysdi, Zolgensma, etc.) or participated in clinical studies of other SMA drugs.
- Had received previous or anticipated major surgical procedures during the study assessment period.
Trial locations and contacts
3 sites in 1 country.
China3 sites
West China Sencond Hospital, Sichuan University / West China women's and children's Hospital
Chengdu, Sichuan
Xinhua Hospital Affiliated To Shanghai Jiao Tong University School Of Medicine
Shanghai, 200092
National Children's Medical Center, Children's Hospital of Fudan University
Shanghai
About this study
Official title: 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 is a clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
Read the detailed description
This is a multicenter, open, dose-escalation clinical study to evaluate the safety, initial efficacy, and immunogenicity of SKG0201 injection in patients with spinal muscular atrophy type 1 (SMA 1).
- Conditions
- Spinal Muscular Atrophy 1
- Being tested
- SKG0201 Injection
- Keywords
- SMA 1
- 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 in vivo gene therapeutic product.
| Study group | Role | What they receive |
|---|---|---|
| Dose-escalation | Experimental | SKG0201 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: 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: 24 weeks
Proportion of subjects who achieve developmental milestones with improvement in exercise intensity and function assessed according to BSID-III
Developmental milestones are defined according to BSID-III (Bayley Scales of Infant and Toddler Development Third Edition) criteria.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Jan 2024
- Study start25 Jun 2023
- Primary completionDec 2025 (estimated)
- Study completionDec 2025 (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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kun Sun
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Kun Sun, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
- Investigators
- Kun Sun, Doctor, Principal investigator, Xinhua Hospital, Shanghai Jiao Tong University School of MedicineYongguo Yu, Doctor, Principal investigator, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06191354
- Sponsor's protocol ID
- SKG0201-001
- Status last verified
- Nov 2025
- First submitted
- 19 Dec 2023
- Last updated
- 21 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06191354
- Acronym / sponsor ID
- SKG0201-001
- Conditions
- Spinal Muscular Atrophy 1
- Keywords
- SMA 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Kun SunUniversity, hospital or other
- Study start
- 25 Jun 2023 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 5 Jan 2024 (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 clinical study to evaluate the safety and efficacy of gene therapy drug SKG0201 Injection in patients with spinal muscular atrophy Type 1 (SMA 1).
Detailed description
This is a multicenter, open, dose-escalation clinical study to evaluate the safety, initial efficacy, and immunogenicity of SKG0201 injection in patients with spinal muscular atrophy type 1 (SMA 1).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Dose-escalation | Experimental | Genetic: SKG0201 InjectionSKG0201 one-time deliver |
Who may qualify
- Ages
- Up to 180 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Type 1 SMA, defined by bi-allelic mutations in the SMN1 gene.
- Age 180 days or younger at day of infusion.
- 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.
- 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.
- 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.
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 (WHO 2006).
- Active viral infection with significant signs or symptoms and require systematic hospitalization.
- In the presence of other severe infections or diseases.
- Known allergy to prednisolone, other glucocorticoids, or their 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 in vivo gene therapeutic product. | Genetic | Dose-escalation |
Primary outcome
Incidence of AEs and SAEs
Time frame: 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: 24 weeks
Proportion of subjects who achieve developmental milestones with improvement in exercise intensity and function assessed according to BSID-III
Time frame: 24 weeks
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 21 Nov 2025.
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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. This study is not recruiting at the moment.
Kun Sun, Doctor principal investigator
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Yongguo Yu, Doctor principal investigator
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing SKG0201 Injection in people with Spinal Muscular Atrophy 1, run by Kun Sun 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 5 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: under way, not looking for participants, as of the sponsor's last update on 21 Nov 2025. 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of AEs and SAEs (time frame: 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 21 Nov 2025.