An Open-Label, Non-Comparative Clinical Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene (ANB-004 (JSC BIOCAD, Russia)) After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy
Status: Recruiting
Location: See all (7) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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.
Eligibility
Participation Requirements
Sex: All
Maximum Age: 8 months
Healthy Volunteers: f
View:
• Informed consent form for participation in the study signed by the subject's legal representative;
• 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;
• 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;
• 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.
• Subjects with 3 copies of the SMN2 gene can be included in the study if they have symptoms of SMA type 1 and the disease began before the age of 180 days.
• The ability of the subject's legal representative, in the Investigator's opinion, to perceive information and follow the Protocol procedures
Locations
Other Locations
Belarus
1. State Institution Republican Scientific and Practical Center Mother and Child
RECRUITING
Minsk
Russian Federation
Federal State Autonomous Educational Institution of Higher Education N.I. Pirogov Russian National Research Medical University, Ministry of Health of the Russian Federation
RECRUITING
Moscow
Federal State Autonomous Educational Institution of Higher Education N.I. Pirogov Russian National Research Medical University, Ministry of Health of the Russian Federation
RECRUITING
Moscow
Federal State Autonomous Institution National Medical Research Center for Children's Health, Ministry of Health of the Russian Federation
RECRUITING
Moscow
Federal State Budgetary Educational Institution of Higher Education St. Petersburg State Pediatric Medical University, Ministry of Health of the Russian Federation
RECRUITING
Saint Petersburg
Federal State Budgetary Institution V. A. Almazov National Medical Research Center of the Ministry of Health of the Russian Federation
RECRUITING
Saint Petersburg
State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital
RECRUITING
Yekaterinburg
Contact Information
Primary
Maria Morozova, MD, PhD
morozovama@biocad.ru
+7 (985) 910 28 13
Time Frame
Start Date:2023-02-02
Estimated Completion Date:2030-08
Participants
Target number of participants:40
Treatments
Experimental: Cohort 1
Subjects in Cohort 1 will receive ANB-004 at a dose 1. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage.
Experimental: Cohort 2
Subjects in Cohort 2 will receive ANB-004 at a dose 2. The dose for Cohort 2 will be determined at the IDMC meeting. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage.
Experimental: Cohort 3
Subjects of Cohort 3, if included, will receive the drug at a dose 3. The dose for Cohort 3 will be determined at the IDMC meeting. Depending on the DLT, the cohort may include 1 to 6 subjects in the first stage and 9 to 12 in the second stage.