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Investigating a new gene therapy for children with spinal muscular atrophy

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

PHASE1; PHASE2 · Biocad · NCT05747261

This study is testing a new gene therapy called ANB-004 to see if it can help young children with spinal muscular atrophy feel better and stay safe.

Quick facts

PhasePHASE1; PHASE2
Study typeInterventional
Enrollment40 (estimated)
AgesN/A to 240 Days
SexAll
SponsorBiocad (industry)
Drugs / interventionsrituximab, methotrexate, cyclophosphamide
Locations7 sites (Minsk and 6 other locations)
Trial IDNCT05747261 on ClinicalTrials.gov

What this trial studies

This multicenter, open-label study aims to evaluate the safety and efficacy of a gene therapy called ANB-004 in children diagnosed with spinal muscular atrophy (SMA). The trial consists of two stages: the first stage involves a dose-escalation design to identify a potentially therapeutic dose, while the second stage assesses the efficacy and safety of that selected dose. Participants will receive a single intravenous infusion of ANB-004 and will be monitored for any adverse events related to the treatment. The study focuses on children under 240 days old with specific genetic criteria for SMA.

Who should consider this trial

Good fit: Ideal candidates are infants under 240 days old with a confirmed diagnosis of 5q-SMA and specific genetic characteristics.

Not a fit: Patients who do not meet the genetic criteria for SMA or are older than 240 days may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve the health outcomes and quality of life for children suffering from spinal muscular atrophy.

How similar studies have performed: Other studies have shown promise with gene therapies for SMA, indicating a potential for success with this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
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. 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.
6. The ability of the subject's legal representative, in the Investigator's opinion, to perceive information and follow the Protocol procedures

Exclusion Criteria:

1. A diagnosis of HIV infection, hepatitis B, hepatitis C, congenital syphilis in the study subject, as well as a documented diagnosis of HIV infection in the study subject's mother. Note: documented hepatitis B and/or hepatitis C and/or syphilis in the mother of a study subject is not an exclusion criterion in this clinical study, provided that standard breastfeeding rules are followed or the subject is not breastfed due to the low risk of transmission of hepatitis B and C viruses and Treponema pallidum from mother to child with breast milk;
2. Unwillingness of the legal representative to use alternative feeding methods (nasogastric tube, gastrostomy) in case of swallowing disorders and a risk of aspiration;
3. Anti-AAV9 antibody titer \>1:50 determined by ELISA. Note: if a subject's screening anti-AAV9 antibody titer is \>1:50, the anti-AAV9 antibody titer may be determined again. Subjects with anti-AAV9 antibody titers ≤1:50 in the second test may be included in the study;
4. Need for respiratory support for ≥16 hours per day or tracheostomy ;
5. Treatment with nusinersen, risdiplam, branaplam, onasemnogene abeparvovec or other antisense oligonucleotides/selective SMN2 splicing modifiers or gene therapy drugs for SMN1 transduction or other AAV-based gene therapy drugs regardless of serotype used previously (from birth) or planned for the main study period, i.e., within 12 months after the administration of the investigational product.
6. A need to use any medications for the treatment of myopathy or neuropathy, drugs for the treatment of diabetes, ongoing immunosuppressive therapy, or the need for immunosuppressive therapy after the start of the study (for example, glucocorticoids (except for premedication and post-medication), cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab, etc.);
7. Subjects with the following laboratory test results at screening:

   * increased activity of transaminases (ALT, AST) or GGT \>2×ULN;
   * total bilirubin level ≥34 µmol/L;
   * creatinine level ≥160 µmol/L;
   * hemoglobin \<80 g/L and \>180 g/L;
   * WBC count \>20x109/L;
   * Troponin I level \> ULN.
8. Any concomitant diseases that, in the Investigator's opinion, may affect the safety of ANB-004 in the subject or have a significant impact on the assessment of the outcomes of SMA therapy;
9. A diagnosis of acute or chronic hepatic failure at screening;
10. A known allergy or intolerance to any components of the investigational product or pre- and post-medication drug (glucocorticoids);
11. Simultaneous participation of the subject in other clinical studies or previous participation in another clinical study using an experimental therapy.

Where this trial is running

Minsk and 6 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Spinal Muscular Atrophy

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.