Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy. The study will have a standard 3+3 dose-escalation design.
| Condition(s) | Spinal Muscular Atrophy (SMA) |
|---|---|
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Study type | Interventional |
| Summary | The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy. The study will have a standard 3+3 dose-escalation design. |
| Who can participate | Inclusion Criteria: 1. Informed consent form for participation in the study signed by the subject's legal representative; 2. Subjects of either sex under the age of 240 days at the time of signing the Information Sheet for the Legal Representative of the Clinical Study Subject with Informed Consent Form; 3. A diagnosis of 5q-SMA (homozygous deletion of exon 7 of the SMN1 gene or heterozygous deletion of exon 7 + confirmed point mutation of the SMN1 gene) and 2 or 3 copies of the SMN2 gene established based on molecular genetic testing; 4. Subjects with 2 copies of the SMN2 gene can be included in the study both at the presymptomatic stage of the disease and in the presence of SMA symptoms. If symptoms are present, the age of onset of the disease should be up to 180 days from birth. 5. Subj |
| Ages | 240 Days |
| Sex | All |
| Lead sponsor | Biocad |
| Locations | Minsk, Belarus; Moscow, Russia; Moscow, Russia; Moscow, Russia; Saint Petersburg, Russia; Saint Petersburg, Russia (+1 more sites) |
| Start date | 2023-02-02 |
| NCT ID | NCT05747261 |
| Official listing | https://clinicaltrials.gov/study/NCT05747261 |