Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    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).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2020-005174-94
    Sponsor's Protocol Code Number:ISIS696844-CS5
    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:2021-06-09
    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 number2020-005174-94
    A.3Full title of the trial
    A Phase 2, Randomized, Placebo-Controlled, Double-Masked Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRX, an Antisense Inhibitor of Complement Factor B, in Patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
    Estudio en fase II, aleatorizado, controlado con placebo, doble ciego, para evaluar la seguridad y la eficacia de dosis múltiples de IONIS-FB-LRX, un inhibidor antisentido del factor B del complemento, en pacientes con atrofia geográfica secundaria a degeneración macular asociada a la edad (DMAE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to Asses the Safety and Efficacy of Multiple Doses of Ionis-FB-LRX in patients with Geographic Atrophy Secondary to Age-Related Macular Degeneration
    Estudio para evaluar la seguridad y eficacia de múltiples dosis de Ionis-FB-LRX en pacientes con atrofia geográfica secundaria a degeneración macular asociada con la edad
    A.4.1Sponsor's protocol code numberISIS696844-CS5
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03815825
    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 SponsorIonis Pharmaceuticals, 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 supportIonis Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIonis Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointIonis Clinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address2855 Gazelle Court
    B.5.3.2Town/ cityCarlsbad
    B.5.3.3Post codeCA 92010
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1760931 9200
    B.5.5Fax number+1760603 2504
    B.5.6E-mailclinicaltrials@ionisph.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.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameISIS 696844
    D.3.2Product code ISIS 696844
    D.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNISIS 696844
    D.3.9.1CAS number 2272983-11-4
    D.3.9.2Current sponsor codeISIS 696844
    D.3.9.4EV Substance CodeSUB181817
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    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) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    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 No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product Yes
    D.3.11.13.1Other medicinal product typeAntisense Oligonucleotide
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboInjection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Geographic atrophy (GA) secondary to age-related macular degeneration (AMD)
    Atrofia geográfica (AG) secundaria a degeneración macular relacionada con la edad (DMAE)
    E.1.1.1Medical condition in easily understood language
    Progressive loss of central vision, the distortion of images and straight lines, and the presence of blurry and dark areas in the central vision
    Pérdida progresiva de la visión central, distorsión de imágenes y líneas rectas, y presencia de áreas borrosas y oscuras en la visión central.
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10064930
    E.1.2Term Age-related macular degeneration
    E.1.2System Organ Class 10015919 - Eye disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level LLT
    E.1.2Classification code 10063947
    E.1.2Term Geographic atrophy
    E.1.2System Organ Class 10015919 - Eye disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the effect of ISIS 696844 on the rate of change of the area of Geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by fundus autofluorescence (FAF)
    Evaluar el efecto de ISIS 696844 sobre la tasa de cambio del área de AG secundaria a DMAE medida por autofluorescencia del fondo del ojo (AFF)
    E.2.2Secondary objectives of the trial
    To evaluate the effect of ISIS 696844 on plasma FB levels and serum AH50 activity in AMD patients

    To assess the effect of ISIS 696844 to slow reduction of low luminance visual acuity (LLVA)
    Evaluar el efecto de ISIS 696844 sobre los niveles plasmáticos de FB y la actividad sérica de AH50 en pacientes con DMAE.

    Evaluar el efecto de ISIS 696844 para frenar la reducción de la agudeza visual de baja luminancia (LLVA)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal.
    2. Vaccination against Neisseria meningitidis and Streptococcus pneumoniae received at least 2 weeks prior to first dose of investigational product
    3. Well-demarcated geographic atrophy (GA) due to AMD
    4. Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen equivalent) or better on the ETDRS chart
    5. Must have clear ocular media and adequate pupillary dilation in the study eye to permit high-quality fundus imaging
    1. Las mujeres no deben estar embarazadas ni en período de lactancia, y deben ser quirúrgicamente estériles o posmenopáusicas.
    2. Haberse vacunado contra Neisseria meningitidis y Streptococcus pneumoniae al menos 2 semanas antes de la primera dosis del producto en investigación
    3. AG debida a DMAE bien delimitada
    4. Puntuación de mejor agudeza visual corregida (MAVC) de 35 letras (aprox. 20/200 en su equivalente de Snellen) o mejor en la tabla del Estudio del tratamiento precoz de la retinopatía diabética (Early Treatment Diabetic Retinopathy Study, ETDRS)
    5. Se debe tener una dilatación pupilar adecuada para permitir la obtención de imágenes del fondo de ojo de alta calidad
    E.4Principal exclusion criteria
    1.Clinically-significant abnormalities in medical history
    2. A lack of full recovery from any infection for at least 14 days prior to the Study Drug administration
    3. Chronic treatment with steroids, including topically or intravitreally administered
    4. History or presence of diabetic retinopathy or diabetic macular edema (DME)
    5. History or presence of a disease other than AMD that could affect vision or safety assessments
    6. Prior treatment with another investigational drug, biological agent, or device
    7. Other protocol-specified inclusion/exclusion criteria may apply
    1. Anomalías clínicamente significativas (CS) en los antecedentes médicos
    2. La falta de recuperación completa de alguna infección durante al menos 14 días antes de la administración del fármaco del estudio
    3. Tratamiento crónico con esteroides, incluidos los administrados por vía tópica o intravítrea
    4. Historia o presencia de retinopatía diabética o edema macular diabético (EMD)
    5. Historial o presencia de una enfermedad distinta de la DMAE que podría afectar las evaluaciones de la visión o la seguridad
    6. Tratamiento previo con otro medicamento, agente biológico o dispositivo en investigación
    7. Pueden aplicarse otros criterios de inclusión / exclusión especificados según el protocolo.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the absolute change in the GA area at Week 49 compared to baseline, as measured by FAF in study eye.
    El criterio de valoración principal de la eficacia es el cambio absoluto en el área de AG en la semana 49 en comparación con el inicio, medido mediante AFF en el ojo de estudio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 49
    Semana 49
    E.5.2Secondary end point(s)
    The secondary efficacy endpoints include:
    • Percent change in levels of plasma FB from Baseline
    • Percent change in levels of serum AH50 activity from Baseline
    • Absolute change in LLVA score from Baseline at Week 49
    Los criterios de valoración secundarios la eficacia son:
    • Cambio porcentual de la concentración de FB en plasma desde el inicio
    Los criterios de valoración secundarios la eficacia son:
    • Cambio porcentual en los niveles de actividad de AH50 en suero desde el inicio
    • Cambio absoluto en la puntuación de AVBI desde el inicio hasta la semana 49
    E.5.2.1Timepoint(s) of evaluation of this end point
    Please, refer to Appendix A in the protocol for a detailed schedule of procedures
    Consulte el Apéndice A del protocolo para obtener un calendario detallado de procedimientos.
    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 Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 concerned10
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA26
    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
    Canada
    New Zealand
    United States
    Austria
    Hungary
    Netherlands
    Poland
    Spain
    Czechia
    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
    The End-of-Study is last patient, last Post-Treatment visit or last study-related procedure.
    El final del estudio es el último paciente, la última visita posterior al tratamiento o el último procedimiento relacionado con el estudio.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years5
    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 No
    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) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 330
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 59
    F.4.2.2In the whole clinical trial 355
    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 Decision2021-08-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-17
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Thu Apr 25 10:37:10 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA