Efficacy: LTFU
START Long-Term Follow-Up (LTFU):
ZOLGENSMA demonstrates durability of effect up to 7.5 years after treatment1
An ongoing, long-term follow-up study of patients from the START clinical trial (N=13)1,2
The long-term follow-up study of START began after the completion of the 24-month START trial and is planned to last 15 years. A total of 13 patients, 10 from the high-dose cohort and 3 from the low-dose cohort, are enrolled in the study.
EXPAND ENDPOINTS AND ENROLLMENT CRITERIA
Endpoints2
Primary endpoint:
- Long-term safety as assessed by incidence of serious adverse events (SAEs) and adverse events (AEs) of special interest
Key inclusion and exclusion criteria2
Key inclusion criteria:
- Received ZOLGENSMA in the START trial for SMA Type 1
- Parent/legal guardian willing and able to complete the informed consent process, comply with study procedures, and visit schedule
Key exclusion criteria:
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
![](https://www.zolgensma-hcp.com/wp-content/uploads/2023/06/study2-icon.png)
The latest START long-term follow-up data
(May 2022 data cut)
A total of 10 out of 12 high-dose patients enrolled in an ongoing, observational, long-term follow-up of the START trial1
As of May 2022, the mean time since treatment was 6.9 years (range 6.4-7.5) and the mean age at last follow up was 7.1 years (range 6.6-7.9).1,a
aA 15-year, voluntary follow-up of patients in the START trial who received a high dose of ZOLGENSMA (n=10). In long-term follow-up, 6 patients had used another disease-modifying therapy.1
As of the May 2022 data cut1:
Event-free survival
100% (10/10) of patients
were alive and free of permanent ventilation1,a
ZOLGENSMA continues to provide durable efficacy up to 7.5 years after treatment1
The long-term follow-up study is an ongoing follow-up of the Phase 1 START trial.
All 10 patients from the START high-dose cohort who enrolled in the long-term follow-up were alive and free of permanent ventilation as of May 20221
![Results from the Phase 1 START clinical trial and long-term follow-up including motor milestone achievements](https://www.zolgensma-hcp.com/wp-content/uploads/2023/06/ZOLGENSMA-continues-to-provide-durable-efficacy-over-5-years-after-treatment-1024x598.png)
aAs of the last visit prior to December 2019 data cut.
bBased on adverse event reporting death, withdrawal, or permanent ventilation. There were no in-person assessments between December 2019 and June 2020.
START Trial
An open-label, single-arm, Phase 1, dose-escalation clinical trial supported the efficacy of ZOLGENSMA
START TrialReferences: 1. Data on file. Novartis Gene Therapies, Inc. 2023. 2. Novartis Gene Therapies, Inc. Long-term follow-up study for patients from AVXS-101-CL-101 (START). https://clinicaltrials.gov/study/NCT03421977. ClinicalTrials.gov identifier: NCT03421977. Updated July 11, 2023. Accessed October 19, 2023. 3. Finkel RS, McDermott MP, Kaufmann P, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology. 2014;83(9):810-817. 4. Data on file. AveXis, Inc. 2020.