Results and Updates for Each Zolgensma Trial
Results and Updates for Each Zolgensma Trial
Page last updated: 25th April 2023
On this page:
- Administered Intravenously (IV)
- Administered Intrathecally (IN)
Administered Intravenously (IV)
Starting in April 2014 and ending in December 2017, this first trial of Zolgensma (then called AVEXIS-101) included 15 children under 2 years of age with SMA Type 1 and 2 SMN2 copies. It was an open-label study primarily designed to test the safety and tolerability of intravenous Zolgensma injections.
STR1VE EU started in August 2018 with 33 infants aged 6 months or under with SMA Type 1 and with one or two SMN2 copies. It was testing the safety and efficacy of intravenous administration of Zolgensma. It ended in September 2020.
Similar to STR1VE EU in design, this trial started in October 2017 with 22 infants aged 6 months or under with SMA Type 1 and with one or two SMN2 copies. It’s also testing the safety and efficacy of intravenous administration of Zolgensma and is due to complete in June 2021.
Starting in April 2018, this trial was a Phase III, multi-centre, single-arm study that investigated the efficacy and safety of Zolgensma in 30 pre-symptomatic children with SMN1 mutations and either two or three copies of the SMN2 “back-up” gene who were treated at 6 weeks of age or younger. The trial ended in June 2021.
This is a long-term follow-up safety and efficacy study of children in clinical trials who were treated intravenously with Zolgensma. Participants will ‘roll over’ from their respective previous (parent) study for continuous monitoring of safety as well as monitoring of continued efficacy and durability of response to Zolgensma treatment. The study started in February 2020 with an estimated end date of December 2035 and has a planned enrolment of 308 children.
Published in the October 2021 Lancet medical journal, the study was conducted in neuromuscular centres across Germany and Austria, between September 2019 and April 2021.
Started in September 2021, this global trial is investigating the safety, tolerability and efficacy of intravenous Zolgensma in children who have SMA and weigh between 8.5 kgs and 21 kgs. It aims to recruit 24 participants in total across sites in Europe, North America, Australia and Taiwan, and will follow children for a period of 12 months. Completion date is estimated to be August 2023.
Administered Intrathecally (IT)
This US trial started in December 2017 and was designed to assess safety and provide information on efficacy of a one-time administration of Zolgensma™ into the spinal cord fluid (intrathecal injections) for children aged 6 months or over and under 60 months of age. In October 2019 the Food and Drug Administration (FDA) in the US put a partial clinical trial hold on the study. This was lifted in August 2021 and this Phase 1 and 2 study ended in November 2021 with the most recent data published in April 2023.
Announced in August 2021, this global trial will be investigating the safety, tolerability and efficacy of intrathecal Zolgensma in treatment naive children. It aims to recruit over 100 participants aged between 2 and 18 years and is due to start later in 2022. It is a sham-controlled study – which means one groups receives a placebo. This would be unethical in countries where treatments are available. For this reason, this trial won’t operate in the UK or mainland Europe. It will be offered in countries where no treatments are available