Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
Trial Snapshot
- Phase
- Phase 3
- Status
- Active, not recruiting
- Sponsor
- Novartis Pharmaceuticals
- Enrollment
- 20
- Locations
- 32
- Primary Endpoint
- Number of participants with treatment-emergent serious adverse events (SAEs)
Study Overview
Brief Summary
This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.
Detailed Description
The study is comprised of a Baseline Visit and 2 Follow-up Periods. For Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site. For the first 2 years (Follow-up Period 1), visits will occur every 6 months. For Years 3 to 5 (Follow-up Period 2) follow-up visits will be conducted annually. All patients will enter the study at the baseline visit and continue for 5 years.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 0 Years to 100 Years (Child, Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Participated in an OAV101 clinical trial.
- •Written informed consent must be obtained before any assessment is performed.
- •Patient/Parent/legal guardian willing and able to comply with study procedures.
Exclusion Criteria
- •There are no exclusion criteria for this study.
Arms & Interventions
Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec
Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)
Intervention: onasemnogene abeparvovec (Biological)
Outcomes
Primary Outcomes
Number of participants with treatment-emergent serious adverse events (SAEs)
Time Frame: Up to Year 5
An SAE is defined as any adverse event \[appearance of (or worsening of any pre-existing)\] undesirable sign(s), symptom(s), or medical conditions(s) which meets any one of the following criteria: * fatal * life-threatening * results in persistent or significant disability/incapacity * constitutes a congenital anomaly/birth defect, fetal death or congenital abnormality or birth defect * requires in-patient hospitalization or prolongation of existing hospitalization, unless hospitalization is for routine treatment or monitoring of the studied indication, not associated with any deterioration in condition * is medically significant, e.g. defined as an event that jeopardizes the participant or may require medical or surgical intervention to prevent one of the outcomes listed above
Number of participants with treatment emergent Adverse Events of Special Interest (AESI)
Time Frame: Up to Year 5
The following are important identified and important potential risks (AESI) associated with OAV101: Hepatotoxicity, Transient Thrombocytopenia, Cardiac adverse events, Sensory abnormalities suggestive of ganglionopathy, and Thrombotic microangiopathy. These will be assessed by the investigator.
Secondary Outcomes
- The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist(Up to Year 5)
- The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist(Up to Year 5)
- Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score(Up to Year 5)
- Change from Baseline in the Revised Upper Limb Module (RULM) total score(Up to Year 5)
- Systolic and diastolic blood pressure (mmHg)(Up to Year 5)
- Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min)(Up to Year 5)
- Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min)(Up to Year 5)
- Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius)(Up to Year 5)
- Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%).(Up to Year 5)