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 baby must be 6 months old or younger on the day they receive the treatment
- The baby must have been diagnosed with Type 1 Spinal Muscular Atrophy (SMA) confirmed by a genetic test showing changes in both copies of the SMN1 gene and exactly 2 copies of the SMN2 gene
- Symptoms of SMA must have started before the baby was 6 months old
- The baby's parent or legal guardian must be able to understand the purpose and risks of the study and sign a consent form before any study procedures begin
Who may not be able to join:
- Babies who have previously taken part in a gene therapy research trial
- Babies who have already been treated with Nusinersen (Spinraza) or Risdiplam (Evrysdi)
- Babies who have a high level of a specific antibody (AAV9 neutralizing antibody) in their blood (confirm with trial site)
- Babies who need breathing support through a mask or device for 16 or more hours per day on average
- Babies who have a specific genetic variation in the SMN2 gene called c.859G>C (confirm with trial site)
- Babies who need a breathing machine inserted into their airway, or whose blood oxygen level falls below 95% while they are awake and calm
- Babies who test positive for HIV, hepatitis B, hepatitis C, or syphilis
- Babies who have certain abnormal blood test results related to liver function, red blood cells, or platelets (confirm with trial site for specific values)
- Babies with severe heart failure classified as Class IV under the Modified Ross Heart Failure Classification for Children (confirm with trial site)
- Babies who have a known allergy to steroids (glucocorticoids)
- Babies for whom a lumbar puncture (spinal tap) procedure would not be safe
- Babies who have an active infection being treated with antiviral medication during the screening period
- Babies who have received a vaccination less than 2 weeks before the planned treatment date
- Babies with any other serious illness or condition that the study doctor believes would make participation unsafe or unsuitable
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
Phone: 86-13501380583
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
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]; Proportion of patients treated with GC101 who achieve motor milestone of sit unassisted for at least 10 seconds at 18 months of age
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.