Status:
COMPLETED
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
Lead Sponsor:
Hangzhou Jiayin Biotech Ltd
Conditions:
Type I Spinal Muscular Atrophy
Eligibility:
All Genders
Up to 180 years
Phase:
EARLY_PHASE1
Brief Summary
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy. The research process inc...
Eligibility Criteria
Inclusion
- SMA was diagnosed by bilateral allele SMN1 mutation (deletion or point mutation) gene, and there were 2 copies of SMN2 gene.
- On the day of administration, the age of the subjects did not exceed the 180th day after birth.
- The clinical history and signs are consistent with the manifestations of type I SMA, that is, hypotonia, lagging development of motor function, poor head control, round shoulder posture and excessive joint activity.
- The legal guardian of the subject understands the purpose, possible risks and rights of the test, agrees the subject to participate in the test, completes all research steps, tests and visits, and voluntarily signs the informed consent.
- During the study period, according to the changes of the subject's condition, the subject's legal guardian was willing to carry out standard treatment requirements such as nasal feeding, noninvasive mechanical ventilation and expectoration machine according to the researcher's suggestions.
Exclusion
- The gestational age at birth was less than 35 weeks (245 days).
- During the screening period, when the subjects were awake or asleep and did not receive any auxiliary oxygen supply or respiratory support, the blood oxygen saturation was less than 96%.
- Invasive ventilation or tracheotomy is required, or the current use of noninvasive ventilation support is ≥ 16 hours / day on average.
- According to the WHO child growth standard (who 2009), the weight is lower than the 3rd percentile by age.
- Before administration, if the subjects have not been vaccinated or delayed vaccination according to the national vaccination plan of the current month, it will significantly affect their safety according to the evaluation of the researcher and the medical manager of the project team;
- Active viral infections (including HIV, covid-19, seropositive for hepatitis B or C, torch virus, EBV virus and syphilis).
- Severe non respiratory diseases within 2 weeks before screening.
- Upper respiratory tract infection or lower respiratory tract infection within 4 weeks before screening.
- There are other severe infections or diseases.
- There are known heart diseases or ECG abnormalities with clinical significance.
- Known allergy to prednisolone, other glucocorticoids or their excipients.
- Receive immunosuppressive therapy (e.g. cyclosporin, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab) requiring preventive administration within 3 months before administration.
- Immunomodulatory drugs (such as thymosin, interferon, etc.) are being used to treat myopathy, neuritis and diabetes (such as immunosuppressive agents, glucocorticoids, insulin).
- Anti AAV9 antibody titer \> 1:50 (determined by ELISA). If the potential subject's anti AAV9 antibody titer \> 1:50, it can be retested during the screening period. When the anti AAV9 antibody titer is ≤ 1:50, it can continue to participate in the screening.
- Abnormal laboratory test values with clinical significance (GGT, ALT and AST \> 2.5) × ULN, bilirubin ≥ 3.0 mg / dl, creatinine ≥ 1.0 mg / dl, hemoglobin \< 8 or \> 18 g / dl; Leukocyte count \> 20000 / cm3; Platelet count \< 100000 / cm3).
- Previously participated in clinical research on the treatment of other SMA drugs.
- Major surgery was expected during study treatment.
- Other situations that the researcher judges are not suitable to participate in this study.
Key Trial Info
Start Date :
January 21 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 21 2024
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT06576388
Start Date
January 21 2022
End Date
January 21 2024
Last Update
August 28 2024
Active Locations (1)
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1
Hangzhou Jiayin Biotechnology Co., Ltd
Hangzhou, Zhejiang, China, 310000