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    The EU Clinical Trials Register currently displays   43857   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    Summary
    EudraCT Number:2021-006781-21
    Sponsor's Protocol Code Number:COAV101A12308
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2023-01-10
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2021-006781-21
    A.3Full title of the trial
    Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 IT or OAV101 IV in Clinical Trials
    Seguimiento a largo plazo de pacientes con atrofia muscular espinal tratados en ensayos clínicos con OAV101 por vía IT o IV
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Long-term follow-up of patients with spinal muscular atrophy Treated with OAV101 in Clinical Trials
    Seguimiento a largo plazo de pacientes con atrofia muscular espinal tratados en ensayos clínicos con OAV101
    A.4.1Sponsor's protocol code numberCOAV101A12308
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05335876
    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 SponsorNovartis Pharma AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Pharma AG
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressForum 1, Novartis Campus
    B.5.3.2Town/ cityBasel
    B.5.3.3Post code4056
    B.5.3.4CountrySwitzerland
    B.5.4Telephone number+41 61 324 1111
    B.5.5Fax number+41 61 324 8001
    B.5.6E-mailclinicaltrial.enquiries@novartis.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 Yes
    D.2.1.1.1Trade name Zolgensma 2 x 1013 vg (vector genomes)/mL solution for infusion
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis Gene Therapies EU Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEMA/OD/028/15
    D.3 Description of the IMP
    D.3.1Product nameOAV101
    D.3.2Product code OAV101
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPIntrathecal 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 codeOAV101
    D.3.9.3Other descriptive namepreviously termed sc.AAV9.CB.SMN and AVXS-101
    D.3.9.4EV Substance CodeSUB193254
    D.3.10 Strength
    D.3.10.1Concentration unit Vector genome
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40000000000000
    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 muscular espinal
    E.1.1.1Medical condition in easily understood language
    Spinal Muscular Atrophy
    atrofia muscular espinal
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    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
    The primary objective of this study is to assess long-term safety in terms of treatment-emergent serious adverse events (SAEs) and treatment-emergent adverse events of special interest (AESIs)
    El objetivo principal de este estudio es evaluar la seguridad a largo plazo en términos de acontecimientos adversos graves (serious adverse events [SAEs]) surgidos durante el tratamiento y de acontecimientos adversos de especial interés (adverse events of special interest [AESIs]) surgidos durante el tratamiento.
    E.2.2Secondary objectives of the trial
    The secondary objectives of this study are;
    (1) assess long-term efficacy of OAV101 treatment.
    (2) assess long-term safety for measures other than adverse events .
    Los objetivos secundarios de este estudio son:
    (1) Evaluar la eficacia a largo plazo del tratamiento con OAV101
    (2) Evaluar la seguridad a largo plazo mediante medidas distintas de los acontecimientos adversos.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Participants eligible for inclusion in this study must meet all of the following 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.
    Los participantes elegibles para su inclusión en este estudio deben cumplir todos los criterios siguientes:
    1. Haber participado en un ensayo clínico con OAV101.
    2. Obtención del consentimiento informado por escrito antes de la práctica de cualquier evaluación del estudio
    3. Voluntariedad y capacidad del paciente/progenitores/tutor legal para cumplir con los procedimientos del estudio.
    E.4Principal exclusion criteria
    There are no specific exclusion criteria for this study.
    El estudio carece de criterios de exclusión específicos.
    E.5 End points
    E.5.1Primary end point(s)
    ● Number and proportion of patients reporting treatment-emergent serious adverse events (SAEs) by Medical Dictionary for Regulatory Activities (MedDRA) System Organ Class (SOC) and Preferred Term (PT) for the entire duration of the study (i.e., up to 15 years).
    ● Number and proportion of patients reporting treatment-emergent adverse events of special interest (AESIs) by AESI category and MedDRA SOC and PT within each AESI category for the entire duration of the study (i.e., up to 15 years).
    - Número y el porcentaje de participantes con SAEs surgidos durante el tratamiento, utilizando para ello la clasificación System Organ Class (SOC) y el término preferido (preferred term [PT]) del Medical dictionary for regulatory activities (MedDRA), a lo largo de toda la duración del estudio (es decir, 15 años).
    - Número y el porcentaje de participantes con AESIs surgidos durante el tratamiento, por categoría de AESI y por SOC y PT según el MedDRA, a lo largo de toda la duración del estudio (es decir, 15 años).
    E.5.1.1Timepoint(s) of evaluation of this end point
    up to 15 years
    hasta 15 años
    E.5.2Secondary end point(s)
    ● The number and proportion of participants demonstrating each developmental milestone according to the Developmental Milestone Checklist 
    ● The number and proportion of participants demonstrating maintenance of each developmental milestone
    ● Change from Baseline in the Hammersmith Functional Motor Scale – Expanded (HFMSE) total score
    ● Change from Baseline in the Revised Upper Limb Module (RULM) total score
    ● Number and proportion of patients with potentially clinically significant vital sign findings, summarized individually for each vital sign parameter 
    ● Number and proportion of patients with potentially clinically significant laboratory values, summarized individually for each laboratory parameter
    ● Número y porcentaje de participantes que alcancen cada developmental milestone según el Developmental Milestone Checklist
    ● Número y porcentaje de participantes que muestren mantenimiento de cada developmental milestone
    ● Cambio frente al basal en la puntuación total de la HFMSE
    ● Cambio frente al basal en la puntuación total del RULM
    ●Número y porcentaje de pacientes con hallazgos de signos vitales potencialmente significativos desde el punto de vista clínico, resumidos individualmente para cada parámetro de signo vital
    ●Número y porcentaje de pacientes con valores de laboratorio potencialmente significativos clínicamente, resumidos individualmente para cada parámetro de laboratorio
    E.5.2.1Timepoint(s) of evaluation of this end point
    5 years
    5 años
    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 Yes
    E.6.13.1Other scope of the trial description
    Exploratory
    ● To explore other potential effects of OAV101 on exploratory efficacy and safety biomarkers
    ● To assess long-term safety in terms of adverse events
    ● To describe the time-course of the elevation of anti-AAV9 antibodies after administration of OAV101
    ● To assess long-term Caregiver experience
    Exploratorios
    ● Explorar otros efectos potenciales de OAV101 en biomarcadores exploratorios de eficacia y seguridad
    ● Evaluar la seguridad a largo plazo en términos de acontecimientos adversos
    ● Describir el curso temporal de la elevación de anticuerpos anti-AAV9 después de la administración de OAV101
    ● Evaluar la experiencia del cuidador a largo plazo
    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 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
    Segmto a largo plazo de ptes con AME tratados con OAV101 IT o administración IV. No hay fármaco
    Long-term follow-up of patients with SMA Treated with OAV101 IT or IV administration. No study drug.
    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 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA21
    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 No
    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
    Brazil
    Canada
    China
    Colombia
    Egypt
    India
    Japan
    Malaysia
    Mexico
    Saudi Arabia
    Singapore
    South Africa
    Taiwan
    Thailand
    United States
    Viet Nam
    France
    Netherlands
    Spain
    Switzerland
    Germany
    Greece
    Italy
    Belgium
    Denmark
    United Kingdom
    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
    The end of the LTFU study for each individual Spinal Muscular Atrophy (SMA) patient will be 15 years after treated with OAV101 by intravenous (IV) or intrathecal (IT) administration received from a parent trial.
    The end of study as a whole is defined as the last patient's last visit (LPLV), which is the last patient's last evaluation (visit Year 15), or the time of premature withdrawal.
    El final del estudio LTFU para cada paciente individual con Atrofia Muscular Espinal (AME) será 15 años después del tratamiento con OAV101 por administración intravenosa (IV) o intratecal (IT) recibido de un ensayo principal.
    El final del estudio en su conjunto se define como la última visita del último paciente (LPLV), que es la última evaluación del último paciente (visita Año 15), o el momento de la retirada prematura.
    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 months
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years17
    E.8.9.2In all countries concerned by the trial months5
    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: 260
    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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 210
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 50
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    A parent/guardian consent will be taken for the participants who cannot give consent personally.
    Se tomará el consentimiento de los padres/tutores para los participantes que no puedan dar su consentimiento personalmente.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 29
    F.4.2.2In the whole clinical trial 260
    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
    Ninguno
    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 Decision2023-02-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-02-01
    P. End of Trial
    P.End of Trial StatusOngoing
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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