- Trial Name
- Bluebell
- Age:
- < 8 months old Diagnosed at < 6 months
- Aim:
- To investigate the safety, immunogenicity, and efficacy of ANB-004.
- Status:
- Recruiting
- Location:
- Moscow (3), St. Petersburg (2) & Yekaterinsburg
- Identifier:
- 481
Clinical Trials
Overview
ANB-4
ANB-4 is being developed by Biocad. It is a gene therapy designed to address the genetic root cause of SMA. A vector (AAV9) containing a new working copy of the SMN1 gene is administered intravenously (IV) to deliver the new gene into a patient’s cells, halting disease progression.
Bluebell, a clinical trial of ANB-4 is an open-label, non-comparative clinical study of the safety and efficacy of an adeno-associated viral vector carrying the SMN gene (ANB-004) after a single intravenous administration of escalating doses in children who live with SMA.
Inclusion criteria (criteria of eligibility to take part in the clinical study):
- 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) established clinically and confirmed by molecular genetic testing and 2 copies of the SMN2 gene and aged up to 180 days (6 months) at disease onset (first symptoms)
- aged up to 240 days (8 months)
Exclusion criteria (criteria of ineligibility to take part in the clinical study) include:
- Anti-AAV9 antibody titer (test for antibodies to adeno-associated virus serotype 9) > 1:50
- Need for respiratory support for ≥ 16 hours per day or the presence of a tracheostomy tube
- Prior (from birth) or planned (within 12 months after the administration of the investigational product) treatment with nusinersen, risdiplam, branaplam, onasemnogene abeparvovec or other antisense oligonucleotides / selective SMN2 gene splicing modifiers, or gene therapy products for SMN1 transduction or other gene therapy products based on an AAV vector of any serotype.
- Need to use any medications for the treatment of myopathy or neuropathy, drugs used for the treatment of diabetes mellitus, ongoing immunosuppressive therapy, or the need for immunosuppressive therapy after the start of the study (e.g., glucocorticoids (except for drugs specified in the clinical study protocol), cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab, etc.).
- Other concomitant diseases or conditions that may affect the safety of ANB-004 or preclude any of the necessary study procedures.
- Simultaneous participation of the subject in other clinical studies or previous participation in another clinical study involving the use of an experimental therapy.
Authorisation of the Ministry of Health of the Russian Federation for this clinical study, No. 481 dated August 11, 2022, and the list of approved study centres have been published in the State Register of Medicines.
The vector that delivers the SMN gene is made from a virus called adeno-associated virus 9, or AAV9.
This type of virus is harmless. To make the vector, the DNA of the virus is removed and the new SMN gene inserted in its place. Vectors are used because they can travel throughout the body and deliver the new, working gene to the cells where it is needed.
When the new gene reaches its destination, it is ready to tell the motor neurons to start making SMN protein. This happens throughout the body, with many vectors delivering a new, working copy of the SMN gene to motor neurons.
Motor neurons now make sufficient SMN protein to survive, function, and be maintained.
Trial of ANB-4
Related pages & resources