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Tests treatment safety and results for Spinal Muscular Atrophy (SMA)
Official title Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)
ClinicalTrials.gov ID: NCT05747261
What this study is testing
What is ANB-004, dose 1?
ANB-004, dose 1 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for spinal muscular atrophy (sma).
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages up to 240
You may be able to join if
- Informed consent form for participation in the study signed by the subject's legal representative;
- people 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...
- 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)...
- people 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...
- people 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...
You likely can't join if
- A diagnosis of HIV infection, hepatitis B, hepatitis C, congenital syphilis in the study subject, as well as a documented diagnosis of HIV infection...
- Unwillingness of the legal representative to use alternative feeding methods (nasogastric tube, gastrostomy) in case of swallowing disorders and a...
- 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...
- Need for respiratory support for ≥16 hours per day or tracheostomy ;
- Treatment with nusinersen, risdiplam, branaplam, onasemnogene abeparvovec or other antisense oligonucleotides/selective SMN2 splicing modifiers or...
- A need to use any medications for the treatment of myopathy or neuropathy, drugs for the treatment of diabetes, ongoing immunosuppressive therapy, or...
See the full eligibility criteria
- Informed consent form for participation in the study signed by the subject's legal representative;
- people 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...
- people 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...
- people 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
- 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...
- Unwillingness of the legal representative to use alternative feeding methods (nasogastric tube, gastrostomy) in case of swallowing disorders and a risk of aspiration;
- 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. people with anti-AAV9 antibody titers ≤1:50...
- Need for respiratory support for ≥16 hours per day or tracheostomy ;
- 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...
- 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...
- people 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 \ 180 g/L;
- WBC count \>20x109/L;
- Troponin I level \> ULN.
- 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;
- A diagnosis of acute or chronic hepatic failure at screening;
- A known allergy or intolerance to any components of the investigational product or pre- and post-medication drug (glucocorticoids);
- Simultaneous participation of the subject in other clinical studies or previous participation in another clinical study using an experimental therapy.
The study team makes the final eligibility decision.
Where it's taking place
- Minsk, Belarus
- Moscow, Russia
- Saint Petersburg, Russia
- Yekaterinburg, Russia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, up to 240 days. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Minsk, Belarus; Moscow, Russia; Saint Petersburg, Russia; Yekaterinburg, Russia. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.