Clinical Trial Results
Clinical Trial Results
STEER Trial – Completed
This was a randomised, double-blind, sham-controlled Phase 3 study with a primary objective to evaluate the clinical efficacy, safety, and tolerability of a one-time intrathecal dose of Itvisma in treatment naïve children and young people with Type 2 SMA who are between 2 and 18 years of age, able to sit, but have never walked.
126 children were randomly selected to receive either Itvisma by intrathecal injection or to receive a sham (placebo) procedure. A sham-controlled study is unethical in countries where treatments are available. For this reason, this trial did not operate in the UK or mainland Europe. It was offered in countries where no treatments were available (e.g., Brazil, China, India).
Results showed that Itvisma was safe and that patients that received this drug demonstrated a significant improvement in motor function compared to those who had received a placebo.
The published findings of the trial can be found here: https://pubmed.ncbi.nlm.nih.gov/41360993/
SPECTRUM Trial – Ongoing
This is a global, prospective, multi-centre study that will assess the long-term safety and efficacy of Itvisma in patients who participated in an Itvisma clinical trial.
Over a 5-year period, this trial will assess the safety profile of Itvisma. It will also determine how well the drug has worked though assessment of motor abilities, including maintenance and improvement of motor milestones. This study is no longer looking for participants.
STREAM Trial – Upcoming
This is a prospective, multicentre, Phase 4 study that will assess improvement of motor abilities over 2-years in patients 2 years or older that have received Itvisma. This study is based in the United States and will not be recruiting in the UK.