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    Summary
    EudraCT Number:2019-002611-26
    Sponsor's Protocol Code Number:AVXS-101-LT-002
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2021-08-03
    Trial 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
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2019-002611-26
    A.3Full title of the trial
    A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101
    Studio di follow-up a lungo termine su pazienti che hanno ricevuto AVXS-101 durante le sperimentazioni cliniche per l'atrofia muscolare spinale
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    not applicable
    non applicabile
    A.3.2Name or abbreviated title of the trial where available
    not applicable
    non applicabile
    A.4.1Sponsor's protocol code numberAVXS-101-LT-002
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    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 pointSr. Manager Clin. Trial Operations
    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 number0013126678118
    B.5.5Fax number000000000
    B.5.6E-mailAValluru979@avexis.com
    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 No
    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 Concentrate for solution for 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 Other
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number200000000 to 600000000
    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) No
    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
    Spinal Muscular Atrophy
    Atrofia Muscolare Spinale
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy
    Atrofia Muscolare Spinale
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10041582
    E.1.2Term Spinal muscular atrophy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To collect long-term follow-up safety and efficacy data in patients with spinal muscular atrophy (SMA) Type 1, Type 2 or Type 3 who were treated with AVXS-101 in an AVXS-101 clinical trial, including but not limited to AVXS-101-CL-102 (Phase 1), AVXS-101-CL-302 (Phase 3), AVXS-101-CL-303 (Phase 3), AVXS-101-CL-304 (Phase 3) or AVXS-101-CL-306 (Phase 3).
    raccogliere dati di sicurezza ed efficacia di follow-up a lungo termine in pazienti con atrofia muscolare spinale (SMA) di tipo 1, tipo 2 o tipo 3 trattati con AVXS-101 in una sperimentazione clinica con AVXS-101, compresi, tra gli altri, AVXS-101-CL-102 (fase 1), AVXS-101-CL-302 (fase 3), AVXS-101-CL-303 (fase 3), AVXS-101-CL-304 (fase 3) o AVXS-101-CL-306 (fase 3).
    E.2.2Secondary objectives of the trial
    not applicable
    non applicabile
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    > Patients with SMA (with 1, 2 or 3copies of SMN2) who received AVXS-101 gene replacement therapy in an AveXis clinical study
    > Patient/parent/legal guardian willing and able to complete the informed consent process and comply with study procedures and visit schedule
    > Pazienti con SMA (con 1, 2 o 3 copie del gene di sopravvivenza del motoneurone 2) che hanno ricevuto una terapia di sostituzione genica con AVXS-101 in uno studio clinico di AveXis
    > Paziente/genitore/tutore legale disposto e in grado di completare il processo di consenso informato, rispettare le procedure dello studio e il programma delle visite
    E.4Principal exclusion criteria
    > Parent/legal guardian unable or unwilling to participate in the long-term follow-up safety study
    > Paziente/genitore/tutore legale non in grado o disposto a partecipare allo studio di follow-up a lungo termine
    E.5 End points
    E.5.1Primary end point(s)
    1. Number of participants who reach developmental milestones
    2. Change from baseline in Hammersmith Functional Motor Scale - Expanded (HFMSE) score
    3. Number of participants who experience a clinically significant change from baseline in pulmonary assessment results
    4. Number of participants who experience swallowing dysfunction
    5. Number of participants who experience a clinically significant change from baseline in physical examination findings
    6. Number of participants who experience a clinically significant change from baseline in vital signs measurements
    7. Change from baseline in height measurements
    8. Change from baseline in weight measurements
    9. Number of participants who experience a clinically significant change from baseline in clinical laboratory assessments
    10. Number of participants who experience a clinically significant change from baseline in cardiac assessments
    11. Number of participants who experience a clinically significant change from baseline in observational phase questionnaire results
    12. Number of participants who experience at least one serious adverse event (SAE)
    13. Number of participants who experience at least one adverse event of special interest (AESI)
    1. Numero di partecipanti che raggiungono traguardi di sviluppo
    2. Variazione rispetto al basale del punteggio Hammersmith Functional Motor Scale - Expanded (HFMSE)
    3. Numero di partecipanti che presentano un cambiamento clinicamente significativo rispetto al basale dei risultati della valutazione polmonare
    4. Numero di partecipanti che presentano disfunzione della deglutizione
    5. Numero di partecipanti che sperimentano un cambiamento clinicamente significativo rispetto al basale nei risultati dell'esame obiettivo
    6. Numero di partecipanti che sperimentano un cambiamento clinicamente significativo rispetto al basale nella misurazione dei segni vitali
    7. Variazione rispetto al basale delle misurazioni dell'altezza
    8. Variazione rispetto al basale delle misurazioni del peso
    9. Numero di partecipanti che sperimentano un cambiamento clinicamente significativo rispetto al basale nelle valutazioni cliniche di laboratorio
    10. Numero di partecipanti che presentano un cambiamento clinicamente significativo rispetto al basale nelle valutazioni cardiache
    11. Numero di partecipanti che sperimentano un cambiamento clinicamente significativo rispetto al basale nei risultati del questionario della fase osservazionale
    12. Numero di partecipanti che hanno manifestato almeno un evento avverso grave (SAE)
    13. Numero di partecipanti che sperimentano almeno un evento avverso di interesse speciale (AESI)
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. Up to 5 years
    2. Up to 2 years
    3. Up to 15 years
    4. Up to 5 years
    5. Up to 5 years
    6. Up to 5 years
    7. Up to 5 years
    8. Up to 5 years
    9. Up to 5 years
    10. Up to 5 years
    11. Year 6 to Year 15
    12. Up to 15 years
    13. Up to 15 years
    1. Fino a 5 anni
    2. Fino a 2 anni
    3. Fino a 15 anni
    4. Fino a 5 anni
    5. Fino a 5 anni
    6. Fino a 5 anni
    7. Fino a 5 anni
    8. Fino a 5 anni
    9. Fino a 5 anni
    10. Fino a 5 anni
    11. dall'anno 6 all'anno 15
    12. Fino a 15 anni
    13. Fino a 15 anni
    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 No
    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) No
    E.7.1.1First administration to humans No
    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) Yes
    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 No
    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
    studio di follow-up a lungo termine senza IMP
    long term follow up study without IMP
    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.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA11
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Australia
    Canada
    Japan
    Korea, Republic of
    Taiwan
    United States
    Belgium
    France
    Italy
    Spain
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years15
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years15
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) No
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 85
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 112
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 47
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 64
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2021-08-03. Yes
    F.3.3.2Women of child-bearing potential using contraception Yes
    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.4.1In the member state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 34
    F.4.2.2In the whole clinical trial 308
    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)
    none
    nessuno
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2020-04-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-30
    P. End of Trial
    P.End of Trial StatusOngoing
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