ANB-004–1/ BLUEBELL Open, incomparable clinical trial of the safety and efficacy of an adenoassociated viral vector with the SMN gene (ANB-004 (JSC "BIOCAD", Russia)) with its single intravenous administration in increasing doses to children with spinal muscular atrophy
Clinical trial ANB-004-1/BLUEBELL will assess the main safety and efficacy parameters after a single intravenous administration of various doses of ANB-004 in patients who:
Patients diagnosed with “5q SMA” based on the results of molecular genetic testing (homozygous deletion of the 7th exon of the SMN1 gene or heterozygous deletion of the 7th exon + confirmed point mutation of the SMN1 gene) with the presence of 2 or 3 copies of the SMN2 gene.1
Patients with two copies of the SMN2 gene can be included in the study both at the presymptomatic stage of the disease and if symptoms of SMA are present. If symptoms are present, the onset age must be up to 180 days from birth.
Patients with three copies of the SMN2 gene can be included in the study if symptoms of type 1 SMA are present, with the onset of the disease at an age of up to 180 days from birth.
Age up to 240 days (8 months).
This trial uses exclusion criteria (criteria of ineligibility for the particular clinical study) a potential participant will be fully informed of by the study doctor. In particular, exclusion criteria 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.2, 3
Prior (from birth) 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.
If necessary, following agreement with the researcher doctor and consent of a legal representative of the child participating in this clinical trial, the legal representative of the child and the child participating in the clinical trial may receive accommodation and transportation services for trips required during the child’s participation in this trial (e.g., for the child’s hospitalization to the study center for the administration of ANB-004 etc.).
The researcher will explain in detail to the legal representative of the child participating in this clinical trial in which cases such trips may be required and the procedure for ordering transportation and accommodation services. When transportation and/or accommodation services are ordered, the Sponsor will not receive data or information that can identify the child participating in this clinical trial.
You can ask questions about participation in the clinical study, including about the contact information about trial centers, using a special form on the site ct.biocad.ru.
The researcher will tell the participant’s legal representative about the other eligibility criteria and contraindications for participation in this clinical trial. The participant’s legal representative must read the full information about the trial and consult the researcher and/or attending physician.
ANB Research-004-1/ BLUEBELL takes place in research centers in different cities of the Russian Federation and the Republic of Belarus: Moscow, St. Petersburg, Yekaterinburg, Minsk.
You can ask questions about participating in a clinical trial, including clarifying contact information about research centers, using the form "Any questions left?"
The research physician will tell the participant's legal representative about other selection criteria and contraindications for participation in this clinical trial. The legal representative of the participant should familiarize himself with the full information about the study and consult with the researcher and/or attending physician.
The results of testing performed before screening are taken into account if they confirm the presence of the diagnosis specified in the inclusion criteria and are confirmed by the appropriate conclusion of a geneticist.
A tracheostomy is a temporary or permanent junction between the trachea and the environment.
Participants can use non-invasive respiratory support (BIPAP, etc.) for less than 16 hours a day.
The permission of the Ministry of Health of the Republic of Belarus to conduct this clinical trial No. 90/1924/2023 dated 12/28/2023 and the list of approved research centers are published in The State Register of Clinical Trials of the Republic of Belarus.
This section of the website contains information about a clinical trial of an unregistered medicinal product.