Spinal Muscular Atrophy 1
Conditions
Keywords
SMA type I, SMA I
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.
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).
Interventions
GENETICSKG0201 Injection
SKG0201 is a recombinant adeno-associated virus (rAAV) vector-based gene therapy product.
Sponsors
Lanyue Biotech (Hangzhou) Co., Ltd.
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 180 Days
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of AEs and SAEs | to 18 months of age | AEs: adverse events; SAEs: serious Adverse events |
| Incidence and characteristics of DLT | 4 weeks | DLT: dose-limiting toxicity |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Survival rate | 14 months of age | Survival is defined as avoidance of either death or permanent ventilation. |
| CHOP-INTEND score changes from baseline | to 18 months of age | 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. |
| The proportion of subjects whose CHOP-INTEND score reached 40 or above | to 18 months of age | 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. |
Countries
China
Contacts
CONTACTYongqin Wang
Outcome results
None listed