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Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
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 15 years from the date of OAV101 administration in the previous clinical trial.
Study details:
The study is comprised of a Baseline Period and 3 Follow-up Periods. Follow-up Periods 1 and 2 consist of in-person visits and Period 3 consists of tele-visits. Follow-up Periods 1 and 2, which includes Baseline through Year 5 visits, assessments will be performed at the Investigational site.
During Follow-up Period 3 (Year 6 to up to Year 15 after OAV101 administration), participants/caregivers will be contacted using tele-visits annually for remote assessments. All patients will enter the study at the baseline visit and continue until 15 years since OAV101 administration is reached. Total duration of participation in the study will be dependent upon time of enrollment relative to OAV101 administration and will vary by participant.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Eligibility
Age eligible for study : 0 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2022-12-19
Primary completion: 2039-10-18
Study completion finish: 2039-10-18
Study type
TREATMENT
Phase
PHASE3
Trial ID
NCT05335876
Intervention or treatment
BIOLOGICAL: onasemnogene abeparvovec
Conditions
- • Spinal Muscular Atrophy (SMA)
Find a site
Closest Location:
Novartis Investigative Site
Research sites nearby
Select from list below to view details:
Novartis Investigative Site
Randwick, New South Wales, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
| Participant Group/Arm | Intervention/Treatment |
|---|---|
EXPERIMENTAL: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec
| BIOLOGICAL: onasemnogene abeparvovec
|
What is the study measuring?
Primary outcome
| Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
|---|---|---|
| Number of participants with treatment-emergent serious adverse events (SAEs) | 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 | Up to Year 15 |
| Number of participants with treatment emergent Adverse Events of Special Interest (AESI) | 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. | Up to Year 15 |
Secondary outcome
| Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
|---|---|---|
| The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist | The Developmental Milestone Checklist is a sponsor created list of items using relevant definitions obtained from World Health Organization Multicentre Growth Reference Study (WHO-MGRS). These will be assessed via the milestone checklist, formed of 6 yes/no questions. The developmental milestones are: sitting with support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone. | Up to Year 5 |
| The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist | The Developmental Milestone Checklist is a sponsor created list of items using relevant definitions obtained from World Health Organization Multicentre Growth Reference Study (WHO-MGRS). These will be assessed via the milestone checklist, formed of 6 yes/no questions. The developmental milestones are: sitting with support, hands-and-knees crawling, standing with assistance, walking with assistance, standing alone and walking alone. A yes response indicates that the patient reached a particular development milestone. | Up to Year 5 |
| Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score | The HFMSE is a validated SMA specific assessment devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE contains 33 items rated from 0 (unable to perform) to 2 (performs without modification/adaptation/compensation). Total scores range from 0-66. Higher scores indicate higher levels of motor ability. | Up to Year 5 |
| Change from Baseline in the Revised Upper Limb Module (RULM) total score | The RULM is a validated SMA specific assessment of motor performance in the upper limbs from childhood through adulthood in ambulatory and non-ambulatory individuals with SMA. The scale consists of 19 scorable items: 18 items scored on 0 (unable) to 2 (full achievement) scale, and one item that is scored from 0 (unable) to 1 (able). Total scores range from 0-37 points. Higher scores reflect higher level of motor ability. | Up to Year 5 |
| Systolic and diastolic blood pressure (mmHg) | Not Specified | Up to Year 15 |
| Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min) | Not Specified | Up to Year 15 |
| Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min) | Not Specified | Up to Year 15 |
| Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius) | Not Specified | Up to Year 15 |
| Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%). | Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100. | Up to Year 15 |
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