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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05335876
Registration number
NCT05335876
Ethics application status
Date submitted
12/04/2022
Date registered
20/04/2022
Date last updated
24/05/2024
Titles & IDs
Public title
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials
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Scientific title
Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials
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Secondary ID [1]
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COAV101A12308
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Universal Trial Number (UTN)
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Trial acronym
SPECTRUM
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Spinal Muscular Atrophy (SMA)
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Neurological
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Other neurological disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - onasemnogene abeparvovec
Experimental: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec - Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)
Other interventions: onasemnogene abeparvovec
Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken ß-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of participants with treatment-emergent serious adverse events (SAEs)
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Assessment method [1]
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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
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Timepoint [1]
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Up to Year 15
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Primary outcome [2]
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Number of participants with treatment emergent Adverse Events of Special Interest (AESI)
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Assessment method [2]
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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.
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Timepoint [2]
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Up to Year 15
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Secondary outcome [1]
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The number of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist
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Assessment method [1]
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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.
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Timepoint [1]
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Up to Year 5
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Secondary outcome [2]
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The number of participants demonstrating maintenance of each developmental milestone according to the Developmental Milestone Checklist
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Assessment method [2]
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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.
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Timepoint [2]
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Up to Year 5
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Secondary outcome [3]
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Change from Baseline in the Hammersmith Functional Motor Scale - Expanded (HFMSE) total score
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Assessment method [3]
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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.
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Timepoint [3]
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Up to Year 5
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Secondary outcome [4]
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Change from Baseline in the Revised Upper Limb Module (RULM) total score
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Assessment method [4]
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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.
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Timepoint [4]
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Up to Year 5
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Secondary outcome [5]
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Systolic and diastolic blood pressure (mmHg)
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Assessment method [5]
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Timepoint [5]
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Up to Year 15
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Secondary outcome [6]
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Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min)
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Assessment method [6]
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Timepoint [6]
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Up to Year 15
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Secondary outcome [7]
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Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min)
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Assessment method [7]
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Timepoint [7]
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Up to Year 15
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Secondary outcome [8]
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Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius)
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Assessment method [8]
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Timepoint [8]
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Up to Year 15
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Secondary outcome [9]
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Number of patients with potentialy clinically significant vital sign findings -Oxygen saturation level (%).
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Assessment method [9]
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Oxygen saturation is the fraction of oxygen-saturated hemoglobin relative to total hemoglobin (unsaturated+saturated) in the blood and then multiplied by 100.
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Timepoint [9]
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Up to Year 15
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Eligibility
Key inclusion criteria
1. Participated in an OAV101 clinical trial.
2. Written informed consent must be obtained before any assessment is performed.
3. Patient/Parent/legal guardian willing and able to comply with study procedures.
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Minimum age
No limit
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
There are no exclusion criteria for this study.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
19/12/2022
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
18/10/2039
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Actual
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Sample size
Target
260
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Novartis Investigative Site - Randwick
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Recruitment postcode(s) [1]
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2031 - Randwick
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Virginia
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Country [2]
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Belgium
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State/province [2]
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Leuven
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Country [3]
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Canada
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State/province [3]
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Quebec
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Country [4]
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China
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State/province [4]
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Sichuan
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Country [5]
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China
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State/province [5]
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Beijing
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Country [6]
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Denmark
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State/province [6]
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Copenhagen
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Country [7]
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France
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State/province [7]
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Garches
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Country [8]
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France
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State/province [8]
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Strasbourg
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Country [9]
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Italy
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State/province [9]
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RM
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Japan
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State/province [10]
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Fukuoka
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Japan
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State/province [11]
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Tokyo
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Country [12]
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Malaysia
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State/province [12]
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Kuala Lumpur
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Country [13]
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Singapore
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State/province [13]
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Singapore
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Country [14]
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Spain
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State/province [14]
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Catalunya
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Country [15]
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Taiwan
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State/province [15]
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Kaohsiung
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Country [16]
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Taiwan
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State/province [16]
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Taipei
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Country [17]
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Thailand
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State/province [17]
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Bangkok
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Country [18]
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United Kingdom
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State/province [18]
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London
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Country [19]
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United Kingdom
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State/province [19]
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Newcastle Upon Tyne
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Novartis Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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 15 years from the
date of OAV101 administration in the previous clinical trial.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05335876
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Novartis Gene Therapies Med Info (US, Latin America, Canada)
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Address
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Country
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Phone
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+1-833-828-3947
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Fax
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Email
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[email protected]
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05335876
Download to PDF