A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma

NCT06019637 | Recruiting

• 1 other identifier: COAV101ABR01
• Study Type: observational
• Target: 50
• Locations: 1 country
• Sites: 2

Brief Summary

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

Conditions

Spinal Muscular Atrophies

Keywords

Spinal Muscular Atrophies;; Onasemnogene Abeparvovec;; Brazil;; NIS

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of treatment-emergent SAEs

  Incidence and severity of treatment-emergent Serious AEs (SAEs)

  Up to 5 years

Secondary Outcomes (5)

• Number of patients who experience at least one AESI and number of patients by AESI

  Up to 15 years

• Number of patients who have survived and have not required permanent ventilatory assistance

  Up to 15 years

• Time until death or the need for permanent ventilatory assistance

  Up to 15 years

• Number of patients who achieve each Developmental Motor Milestone

  Up to 15 years

• Number of patients who achieve each WHO Developmental Milestone within age percentiles

  Up to 15 years

Study Arms (1)

Spinal Muscular Atrophy Patients

Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec

Other: Onasemnogene Abeparvovec

Interventions

Onasemnogene Abeparvovec OTHER

Retrospective and prospective observational study. There is no treatment allocation.

Also known as: Zolgensma

Spinal Muscular Atrophy Patients

Eligibility Criteria

• Age: 0 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

The study population will consist of approximately 50 Brazilian pediatric patients with SMA who were treated with Onasemnogene Abeparvovec (Zolgensma®) in the commercial setting, the closed MAP, or the phase IV OFELIA trial. Patients will be recruited in up to 3 centers in Brazil, over one year of recruitment.

You may qualify if:

• 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

You may not qualify if:

• Patients currently enrolled in any interventional clinical trial\*\* other than the phase IV OFELIA trial will be excluded from the study.
• Subjects who were enrolled in a clinical trial (independently of the disease indication and interventional treatment) but are not currently enrolled, can be included in this study.
• During the follow-up, subjects who enroll any clinical trial with pharmacological intervention will discontinue from this study.

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (2)

Novartis Investigative Site

Curitiba, Paraná, 81520-060, Brazil

RECRUITING

Novartis Investigative Site

São Paulo, São Paulo, 05403-000, Brazil

RECRUITING

MeSH Terms

Interventions

Zolgensma

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Novartis Pharmaceuticals

CONTACT

+41613241111; novartis.email@novartis.com

Novartis Pharmaceuticals

CONTACT

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 25, 2023
• First Posted: August 31, 2023
• Study Start: November 22, 2023
• Primary Completion (Estimated): September 30, 2038
• Study Completion (Estimated): September 30, 2038
• Last Updated: January 15, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

BR (2)