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    Summary
    EudraCT Number:2020-001235-27
    Sponsor's Protocol Code Number:AVXS-101-CL-101
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-04-06
    Trial results View results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2020-001235-27
    A.3Full title of the trial
    Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Single Dose Gene Transfer Clinical Trial for patients with Spinal
    Muscular Atrophy Type 1
    A.4.1Sponsor's protocol code numberAVXS-101-CL-101
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/315/2019
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAveXis, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAveXis, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAveXis, Inc
    B.5.2Functional name of contact pointClinical Trial Operations Manager
    B.5.3 Address:
    B.5.3.1Street Address2275 Half Day Road, Suite 200
    B.5.3.2Town/ cityBannockburn IL
    B.5.3.3Post code60015
    B.5.3.4CountryUnited States
    B.5.4Telephone numberunite224804 9762
    B.5.6E-mailmary.gegenhuber@avexis.net
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation Yes
    D.2.1.1.1Trade name Zolgensma
    D.2.1.1.2Name of the Marketing Authorisation holderAveXis, Inc.
    D.2.1.2Country which granted the Marketing AuthorisationUnited States
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/15/1509
    D.3 Description of the IMP
    D.3.1Product nameAVXS-101
    D.3.2Product code AVXS-101
    D.3.4Pharmaceutical form Infusion
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNONASEMNOGENE ABEPARVOVEC
    D.3.9.1CAS number 1922968-73-7
    D.3.9.2Current sponsor codeAVXS-101
    D.3.9.4EV Substance CodeSUB193254
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number20000000000000 to 60000000000000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms Yes
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Gene Replacement Therapy Clinical Trial for Patients with Spinal Muscular Atrophy Type 1
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy Type 1
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Determination of safety based on the development of unacceptable toxicity: defined as the occurrence of any one Grade III or higher, unanticipated, treatment-related toxicity.
    E.2.2Secondary objectives of the trial
    The time from birth until death or until patient requires at least 16-hour per day of ventilation support for breathing for ≥14 consecutive days in the absence of an acute reversible illness, excluding perioperative use.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Six months of age and younger at day of vector infusion with Type 1 SMA as defined by the following features:
    • Diagnosis of SMA based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and 2 copies of SMN2.
    • Onset of disease at birth: up to 6 months of age.
    • Hypotonia by clinical evaluation with delay in motor skills, poor head control, round shoulder posture and hypermobility of joints
    E.4Principal exclusion criteria
    • Active viral infection (includes HIV or serology positive for hepatitis B or C)
    • Use of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry <95% saturation at the screening visit.
    o Patients may be managed using non-invasive ventilator support (BiPAP) for less than 16 hours per day at the discretion of their physician or study staff.
    • Concomitant illness that in the opinion of the PI creates unecessary risks for gene transfer
    • Concomitant use of any of the following drugs: drugs for treatment of myopathy or neuropathy, agents used to treat diabetes mellitus, or ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial
    • Patients with Anti-AAV9 antibody titers >1:50 as determined by ELISA binding immunoassay.
    • Participation in recent SMA treatment clinical trial that in the opinion of the PI creates unnecessary risks for gene transfer.
    • Patient with signs of aspiration based on a swallowing test and unwilling to use an alternative method to oral feeding.
    • Patients with a single base substitution in SMN2 (c.859G>C in exon 7) will be excluded based on predicted mild phenotype.
    E.5 End points
    E.5.1Primary end point(s)
    Determine the Incidence of Grade III or higher, unanticipated, treatment-related toxicity.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary analysis for efficacy will be assessed when all patients reach 13.6 months of age. A follow-up safety analysis will be completed at the time point at which the last patient reaches 24 months post-dose.
    E.5.2Secondary end point(s)
    • Time to death or until patient requires at least 16-hour per day of ventilation support for breathing for ≥14 consecutive days in the absence of an acute reversible illness, excluding perioperative use
    • A successful measure for efficacy for this study will be 50% of SMA Type 1 subjects living independently of 16 or more hours of ventilator support at 2 years.
    • Change in CHOP-INTEND from baseline measure score of age.
    • To determine efficacy by demonstrating improvement of motor function and muscle strength as determined by achievement of significant development milestones including but not limited to the
    ability to sit alone and roll over unassisted.
    E.5.2.1Timepoint(s) of evaluation of this end point
    The primary analysis for efficacy will be assessed when all patients reach 13.6 months of age. A follow-up safety analysis will be completed at the time point at which the last patient reaches 24 months post-dose.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) No
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Open-label, single-arm, single-dose
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.3 Will this trial be conducted at a single site globally? Yes
    E.8.4 Will this trial be conducted at multiple sites globally? No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA Yes
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    United States
    E.8.7Trial has a data monitoring committee Yes
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years2
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 15
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) Yes
    F.1.1.3.1Number of subjects for this age range: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 13
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 15
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    After the End of Trial visit, eligible patients will be asked to rollover into the long-term follow up trial.
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: United States
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