Clinical trial NCT06019637: A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma
Official title: A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Spinal Muscular Atrophies
Keywords: Spinal Muscular Atrophies;
Onasemnogene Abeparvovec;
Brazil;
NIS
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2023-08-31
Enrollment: 50
Sex: ALL
Minimum age (years): 0
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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.

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

Detailed description: This study is a non-interventional Post Authorization Safety Study (PASS) to evaluate long-term, real-world safety data of Brazilian pediatric patients diagnosed with SMA and treated with Onasemnogene Abeparvovec (Zolgensma®) for up to 15 years after the treatment. This study will support the benefit-risk assessment of Onasemnogene Abeparvovec in the approved indications and may also allow for detection of new safety signals and provide further guidance on the management of safety risks associated with Onasemnogene Abeparvovec to patients/caregivers, health care providers (HCPs) and treating physicians, as required by Brazilian Health Authority ANVISA as a conditional measure for granting Zolgensma®'s authorization.

Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Incidence and severity of treatment-emergent SAEs
timeFrame: Up to 5 years
Secondary outcomes:
measure: Number of patients who experience at least one AESI and number of patients by AESI
timeFrame: Up to 15 years
measure: Number of patients who have survived and have not required permanent ventilatory assistance
timeFrame: Up to 15 years
measure: Time until death or the need for permanent ventilatory assistance
timeFrame: Up to 15 years
measure: Number of patients who achieve each Developmental Motor Milestone
timeFrame: Up to 15 years
measure: Number of patients who achieve each WHO Developmental Milestone within age percentiles
timeFrame: Up to 15 years
Other outcomes:
Locations:
city: Curitiba
country: Brazil
facility: Novartis Investigative Site
state: PR
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: