Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

PHASE3RECRUITING

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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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.

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Eligibility criteria

Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.

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.
  • 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

    Study type

    TREATMENT

    phase

    Phase

      PHASE3

    trial

    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

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    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/ArmIntervention/Treatment
    EXPERIMENTAL: Intravenous (IV) & Intrathecal (IT) Onasemnogene Abeparvovec
    • Patients who received OAV101 IT or OAV101 IV in clinical trials (COAV101A12306, COAV101B12301 and COAV101B12302)
    BIOLOGICAL: 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 β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.

    What is the study measuring?

    Primary outcome

    Primary Outcome MeasurePrimary Outcome DescriptionPrimary 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 aboveUp 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 MeasureSecondary Outcome DescriptionSecondary Outcome Time Frame
    The number of participants demonstrating each developmental milestone according to the Developmental Milestone ChecklistThe 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 ChecklistThe 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 scoreThe 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 scoreThe 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 SpecifiedUp to Year 15
    Number of patients with potentialy clinically significant vital sign findings - Respiratory Rate (breaths/min)Not SpecifiedUp to Year 15
    Number of patients with potentialy clinically significant vital sign findings -Pulse Rate (beats/min)Not SpecifiedUp to Year 15
    Number of patients with potentialy clinically significant vital sign findings -Temperature (Degrees Celsius)Not SpecifiedUp 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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    References

    Clinical Trials Gov: Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

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