A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study

Status: Recruiting
Location: See all (2) locations...
Intervention Type: Other
Study Type: Observational
SUMMARY

A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

Eligibility
Participation Requirements
Sex: All
Maximum Age: 100
Healthy Volunteers: f
View:

• Subject's parent or legal guardian has provided signed eICF.

• Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene, and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene.

• Subject treated\* with Onasemnogene Abeparvovec (Zolgensma®) prior to enrolling in this study.

⁃ Subjects treated with nusinersen or risdiplam prior to Onasemnogene Abeparvovec (Zolgensma®) can be enrolled if currently not receiving it.

⁃ \*Subjects can be enrolled in this study on the day treated with Onasemnogene Abeparvovec (Zolgensma®) or if prior medical history is available to complete all assessments retrospectively, in accordance with local ethical requirements.

• Subject and parent/guardian are willing and able to comply with the phone contacts through the course of the study

Locations
Other Locations
Brazil
Novartis Investigative Site
RECRUITING
Curitiba
Novartis Investigative Site
RECRUITING
São Paulo
Contact Information
Primary
Novartis Pharmaceuticals
novartis.email@novartis.com
+41613241111
Backup
Novartis Pharmaceuticals
Time Frame
Start Date: 2023-11-22
Estimated Completion Date: 2038-09-30
Participants
Target number of participants: 50
Treatments
Spinal Muscular Atrophy Patients
Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec
Sponsors
Leads: Novartis Pharmaceuticals

This content was sourced from clinicaltrials.gov