Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi

• Register: ClinicalTrials.gov
• Last refreshed on: 4 March 2024
• Main ID: NCT04042025
• Date of registration: 31/07/2019
• Prospective Registration: Yes
• Primary sponsor: Novartis Gene Therapies
• Public title: Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
• Scientific title: A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
• Date of first enrolment: February 10, 2020
• Target sample size: 85
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT04042025
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Belgium, Canada, France, Italy, Japan, Korea, Republic of, Spain, Taiwan, United Kingdom, United States

Contacts

• Name: Sitra Tauscher-Wisniewski, MD
• Affiliation: Novartis Gene Therapies, Inc.

Key inclusion & exclusion criteria

Inclusion Criteria:

- Any participant with SMA who received onasemnogene abeparvovec-xioi gene replacement
therapy in a Novartis Gene Therapies-sponsored clinical study

- Participant/parent/legal guardian willing and able to complete the informed consent
process and comply with study procedures and visit schedule

Exclusion Criteria:

- Parent/legal guardian unable or unwilling to participate in the long-term follow-up
safety study



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Spinal Muscular Atrophy Type III

Spinal Muscular Atrophy Type I

Spinal Muscular Atrophy Type II

SMA

Intervention(s)

Biological: Onasemnogene Abeparvovec-xioi

Primary Outcome(s)

Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements [Time Frame: Up to 5 years]

Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings [Time Frame: Up to 5 years]

Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score [Time Frame: Up to 5 years]

Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments [Time Frame: Up to 5 years]

Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI) [Time Frame: Up to 15 years]

Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older [Time Frame: Up to 5 years]

Number of Participants Who Reach Developmental Milestones [Time Frame: Up to 5 years]

Number of Participants With Concomitant Medications Overall and by Type of Medications [Time Frame: Up to 5 years]

Change From Baseline in Revised Upper Limb Module (RULM) Score [Time Frame: Up to 5 years]

Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments [Time Frame: Up to 5 years]

Change From Baseline in Weight Measurements [Time Frame: Up to 5 years]

Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results [Time Frame: Year 6 to Year 15]

Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support [Time Frame: Up to 15 years]

Change From Baseline in Bayley Scales of Infant and Toddler Development [Time Frame: Up to 42 months, 15 days of age]

Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support [Time Frame: Up to 5 years]

Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND) [Time Frame: Up to 5 years]

Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age [Time Frame: Up to 5 years]

Change From Baseline in Height Measurements [Time Frame: Up to 5 years]

Number of Participants Who Experience at Least One Serious Adverse Event (SAE) [Time Frame: Up to 15 years]

Number of Participants With Other SMA Therapies Overall and by Type of Medications [Time Frame: Year 6 to Year 15]

Secondary ID(s)

205305

2019-002611-26

COAV101A12103

AVXS-101-LT-002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors