Phase 2 Spinal Muscular Atrophy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- The child's parent or legal guardian must be willing to sign a consent form to allow participation in the study
- The child must be younger than 240 days old (about 8 months) at the time the consent form is signed
- The child must have a confirmed genetic diagnosis of spinal muscular atrophy (SMA) caused by a specific change in the SMN1 gene, with 2 or 3 copies of a related gene called SMN2
- Children with 2 copies of the SMN2 gene may join either before symptoms appear or after symptoms begin, as long as symptoms started before 180 days (about 6 months) of age
- Children with 3 copies of the SMN2 gene may join if they have Type 1 SMA symptoms that began before 180 days of age
- The child's parent or legal guardian must be able to understand the study information and follow the study procedures, in the opinion of the study doctor
Who may not be able to join:
- Children diagnosed with HIV, hepatitis B, hepatitis C, or congenital syphilis, or whose mother has a confirmed HIV diagnosis
- Children whose parent or legal guardian is unwilling to use alternative feeding methods (such as a feeding tube) if the child has trouble swallowing safely
- Children whose blood test shows a high level of antibodies against a substance called AAV9 (above a certain threshold) — a repeat test may be allowed to confirm this
- Children who need breathing support for 16 or more hours per day, or who have a tracheostomy (a breathing tube in the throat)
- Children who have previously received or are planned to receive other SMA treatments, such as nusinersen, risdiplam, or gene therapy drugs, within the main study period
- Children who need certain medications for muscle or nerve conditions, diabetes treatment, or ongoing immune-suppressing drugs (confirm with trial site)
- Children whose blood test results at the start of the study show abnormal levels of certain liver markers, bilirubin, creatinine, hemoglobin, white blood cells, or a heart protein called Troponin I (confirm specific values with trial site)
- Children with any other health condition that the study doctor believes could affect their safety or the study results
- Children diagnosed with acute or chronic liver failure
- Children with a known allergy or intolerance to any ingredient in the study drug or the medications given before or after treatment (including steroids)
- Children who are currently taking part in another clinical trial, or who have previously received an experimental treatment in another clinical trial
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Arina V Zinkina-Orikhan, PhD, Director of Clinical Development Department, BIOCAD
Phone: +7 (985) 910 28 13
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Proportion of subjects with adverse reactions; Proportion of subjects with serious adverse reactions; Proportion of subjects with CTCAE 5.0 or DAIDS grade 3 or higher adverse reactions; Time from date of birth to event; Motor development score; Change in the The Hammersmith Infant Neurological Examination (HINE) score
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.