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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/NCT04042025
Registration number
NCT04042025
Ethics application status
Date submitted
31/07/2019
Date registered
1/08/2019
Date last updated
22/02/2024
Titles & IDs
Public title
Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi
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Scientific title
A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
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Secondary ID [1]
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2019-002611-26
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Secondary ID [2]
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AVXS-101-LT-002
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Spinal Muscular Atrophy Type I
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Spinal Muscular Atrophy Type II
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Spinal Muscular Atrophy Type III
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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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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Neurodegenerative diseases
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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-xioi
Other: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec-xioi - Participants received treatment with IV onasemnogene abeparvovec-xioi in an onasemnogene abeparvovec-xioi or received treatment with IT onasemnogene abeparvovec-xioi in an onasemnogene.
Other interventions: Onasemnogene Abeparvovec-xioi
Onasemnogene abeparvovec-xioi is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the cytomegalovirus enhancer/chicken ß-actin-hybrid promoter. Onasemnogene abeparvovec-xioi 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 Who Reach Developmental Milestones
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Assessment method [1]
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Assessed via the developmental milestone checklist, formed of 10 yes/no questions. The developmental milestones are: head control, sitting with support, sitting without support, sitting without support for 30 seconds, hands-and-knees crawling, pulls to stand, standing with assistance, walking with assistance, standing alone and walking alone.
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Timepoint [1]
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Up to 5 years
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Primary outcome [2]
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Change From Baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) Score
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Assessment method [2]
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The HFMSE was devised for use in children with SMA to give objective information on motor ability and clinical progression. The HFMSE is formed of 33 assessments rated from 0 (unable to perform functional task) to 2 (able to perform functional task unassisted). Higher scores on the total scale of 0-66 indicates higher levels of motor ability.
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Timepoint [2]
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Up to 5 years
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Primary outcome [3]
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Pulmonary Assessment Results and Require Ventilatory Support
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Assessment method [3]
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Participants will receive pulmonary assessments by a pulmonologist or appropriate clinician. Respiratory device data will be reviewed for participants receiving non-invasive ventilatory support.
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Timepoint [3]
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Up to 15 years
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Primary outcome [4]
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Number of Participants Who Experience Swallowing Dysfunction and Require Nutritional Support
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Assessment method [4]
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Assessed via the swallowing function questionnaire, formed of 4 yes/ no questions and 1 body weight question.
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Timepoint [4]
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Up to 5 years
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Primary outcome [5]
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Number of Participants Who Experience a Clinically Significant Change from Baseline in Physical Examination Findings
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Assessment method [5]
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The physical examination includes review of the following systems: head, ears, eyes, nose and throat, lungs/thorax, cardiovascular, abdomen, musculoskeletal, neurologic, dermatologic, lymphatic, and genitourinary. In addition, visual inspection of the spine, back, shoulders, and hips looking for spinal curvature and asymmetry will be carried out. Joints will be assessed for loss of mobility and contractures.
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Timepoint [5]
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Up to 5 years
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Primary outcome [6]
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Vital Signs Measurements
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Assessment method [6]
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Vital sign measurements will include blood pressure, respiratory rate, pulse, axillary temperature, and pulse oximetry.
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Timepoint [6]
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Up to 5 years
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Primary outcome [7]
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Change From Baseline in Height Measurements
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Assessment method [7]
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Timepoint [7]
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Up to 5 years
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Primary outcome [8]
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Change From Baseline in Weight Measurements
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Assessment method [8]
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Timepoint [8]
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Up to 5 years
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Primary outcome [9]
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Clinical Laboratory Assessments
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Assessment method [9]
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Blood samples will be collected for hematology (including complete blood cell count) and chemistry.
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Timepoint [9]
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Up to 5 years
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Primary outcome [10]
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Cardiac Assessments
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Assessment method [10]
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Cardiac assessments will include a 12-lead electrocardiogram, transthoracic echocardiogram and Troponin-I.
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Timepoint [10]
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Up to 5 years
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Primary outcome [11]
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Number of Participants Who Experience a Clinically Significant Change From Baseline in Observational Phase Questionnaire Results
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Assessment method [11]
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The observational phase questionnaire includes 7 yes/no questions. Observation categories include: adverse events, hospitalizations, concomitant medications, ventilatory support and feeding support.
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Timepoint [11]
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Year 6 to Year 15
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Primary outcome [12]
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Number of Participants Who Experience at Least One Serious Adverse Event (SAE)
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Assessment method [12]
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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 abnormality or birth defect
Requires in-patient hospitalization or prolongation of existing hospitalization
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 [12]
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Up to 15 years
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Primary outcome [13]
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Number of Participants Who Experience at Least One Adverse Event of Special Interest (AESI)
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Assessment method [13]
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An AESI is defined as an AE occurring during any study phase that fulfills one of the following criteria:
Hepatotoxicity
Thrombotic microangiopathy
Cardiac adverse events
Dorsal root ganglia toxicity
New malignancies
New incidence of a neurologic disorder
New incidence of an autoimmune disorder
New incidence of hematologic disorder
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Timepoint [13]
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Up to 15 years
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Primary outcome [14]
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Change From Baseline in Bayley Scales of Infant and Toddler Development
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Assessment method [14]
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Third Edition (Bayley-III) to be performed in all patients up to 42 months, 15 days of age.
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Timepoint [14]
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Up to 42 months, 15 days of age
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Primary outcome [15]
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Change From Baseline in Revised Upper Limb Module (RULM) Score
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Assessment method [15]
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RULM score is based on a scale from 0 to 37 where lower scores reflect poorer upper limb functional ability.
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Timepoint [15]
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Up to 5 years
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Primary outcome [16]
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Change From Baseline in Cogstate Computerized Cognitive Battery Performed in Age 48 Months and Older
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Assessment method [16]
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The Cogstate Computerized Cognitive Battery consists of the Identification Test (scored 0 (best) to 1.5708 (worst)), the International Shopping List Test (scored 0 (worst) to 999 (best)), the International Shopping List Test-Delayed Recall (scored 0 (worst) to 999 (best)), the One Card Learning Test (scored 0 (worst) to 1.5708 (best)), and the One Back Test (scored 0 (worst) to 1.5708 (best)).
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Timepoint [16]
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Up to 5 years
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Primary outcome [17]
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Change From Baseline in Clinical Evaluation of Language Fundamentals Fifth Edition (CELF-5) Performed in All Participants 5 to 21 Years of Age
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Assessment method [17]
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The CELF-5 Following Directions and Sentence Repetition subtests use scoring that varies based on age, but will be administered to participants 5-21 years of age. The Following Directions subtest will be scored from 0-33 with higher score being more advanced and the Recalling Sentences subtest will be scored from 0-78 with higher score being more advanced.
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Timepoint [17]
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Up to 5 years
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Primary outcome [18]
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Change From Baseline in Assessment of Caregiver Experience With Neuromuscular Disease (ACEND)
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Assessment method [18]
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ACEND score is based on a scale from 1 to 41 where higher scores represent a better caregiver experience
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Timepoint [18]
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Up to 5 years
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Primary outcome [19]
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Number of Participants With Concomitant Medications Overall and by Type of Medications
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Assessment method [19]
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Timepoint [19]
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Up to 5 years
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Primary outcome [20]
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Number of Participants With Other SMA Therapies Overall and by Type of Medications
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Assessment method [20]
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Timepoint [20]
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Year 6 to Year 15
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Eligibility
Key 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
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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
- Parent/legal guardian unable or unwilling to participate in the long-term follow-up
safety 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
Active, not 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
10/02/2020
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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
31/12/2035
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Actual
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Sample size
Target
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Accrual to date
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Final
85
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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Sydney Children's Hospital - Randwick
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Recruitment postcode(s) [1]
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2145 - 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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California
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Country [2]
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United States of America
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State/province [2]
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Colorado
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Country [3]
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United States of America
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State/province [3]
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Illinois
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Country [4]
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United States of America
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State/province [4]
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Maryland
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Country [5]
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United States of America
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State/province [5]
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Massachusetts
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Country [6]
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United States of America
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State/province [6]
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Michigan
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Country [7]
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United States of America
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State/province [7]
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Missouri
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Country [8]
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United States of America
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State/province [8]
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New York
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Country [9]
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United States of America
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State/province [9]
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North Carolina
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Country [10]
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United States of America
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State/province [10]
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Ohio
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Country [11]
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United States of America
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State/province [11]
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Pennsylvania
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Country [12]
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United States of America
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State/province [12]
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Texas
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Country [13]
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United States of America
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Utah
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Country [14]
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United States of America
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State/province [14]
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Virginia
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Country [15]
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United States of America
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State/province [15]
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Wisconsin
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Country [16]
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Belgium
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State/province [16]
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Gent
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Country [17]
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Belgium
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State/province [17]
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Liège
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Country [18]
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Canada
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State/province [18]
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Ontario
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Country [19]
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France
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State/province [19]
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Paris
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Country [20]
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Italy
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State/province [20]
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Genova
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Country [21]
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Italy
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State/province [21]
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Milan
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Country [22]
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Italy
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State/province [22]
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Roma
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Country [23]
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Japan
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State/province [23]
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Tokyo
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Country [24]
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Taiwan
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State/province [24]
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Taipei
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Country [25]
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United Kingdom
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State/province [25]
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London
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Country [26]
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United Kingdom
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State/province [26]
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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 Gene Therapies
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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 long-term follow-up safety and efficacy study of participants in clinical trials
for spinal muscular atrophy (SMA) who were treated with onasemnogene abeparvovec-xioi.
Participants will roll over from their respective previous (parent) study into this long-term
study for continuous monitoring of safety as well as monitoring of continued efficacy and
durability of response to onasemnogene abeparvovec-xioi treatment.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT04042025
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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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Sitra Tauscher-Wisniewski, MD
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Address
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Novartis Gene Therapies, Inc.
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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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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT04042025
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