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    Summary
    EudraCT Number:2020-003147-29
    Sponsor's Protocol Code Number:ACT-01_P2_01
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-07-29
    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 ConcernedFrance - ANSM
    A.2EudraCT number2020-003147-29
    A.3Full title of the trial
    A two-arm randomized double-blind placebo-controlled monocentric study to evaluate the safety and tolerability of ACT-01 compared to placebo in patients with acute optic neuritis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study to evaluate the safety of ACT-01 in patients with acute optic neuritis
    A.4.1Sponsor's protocol code numberACT-01_P2_01
    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 SponsorACCURE TX FRANCE SASU
    B.1.3.4CountryFrance
    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 supportACCURE TX FRANCE
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationACCURE TX FRANCE SASU
    B.5.2Functional name of contact pointLucia Septien
    B.5.3 Address:
    B.5.3.1Street Address38 rue Dunois
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75013
    B.5.3.4CountryFrance
    B.5.6E-maillseptien@accure.health
    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 Yes
    D.2.5.1Orphan drug designation numberEU/3/14/1248
    D.3 Description of the IMP
    D.3.1Product nameACT-01
    D.3.2Product code ACT-01
    D.3.4Pharmaceutical form Powder for solution for infusion
    D.3.4.1Specific paediatric formulation No
    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 INNACT-01
    D.3.9.2Current sponsor codeACT-01
    D.3.9.3Other descriptive nameBN201
    D.3.9.4EV Substance CodeSUB191574
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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 No
    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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Subjects from 18 to 60 years old with a diagnosis of a unilateral Acute Optic Neuritis with a demyelinating origin, whose symptoms have appeared in the last 10 days before randomization and whose expanded disability status scale score is between 0 and 5.5
    E.1.1.1Medical condition in easily understood language
    Confirmed diagnosis of a unilateral Acute Optic Neuritis with a demyelinating origin
    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
    The primary objective of the study is to compare the safety and tolerability of ACT-01 versus placebo at month 6 in subjects with acute optic neuritis (AON)
    E.2.2Secondary objectives of the trial
    The main secondary objective is:
    • To compare the efficacy of ACT-01 versus placebo on the retinal layers’ thickness in the affected eye at month 1, month 3, month 6 and month 12;
    The other secondary objectives are:
    • To compare the efficacy of ACT-01 versus placebo on clinical vision parameters in the affected eye at month 1, month 3, month 6 and month 12;
    • To compare the efficacy of ACT-01 versus placebo on electrophysiological parameters in the affected eye at month 3, month 6 and month 12;
    • To compare the efficacy of ACT-01 versus placebo on neurological parameters;
    • To compare the safety of ACT-01 versus placebo at month 12
    • To characterize the pharmacokinetic profile of repeated doses of ACT-01 in subjects with AON
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    The inclusion criteria are:
    - Aged 18-60 years
    - Diagnosed with a unilateral acute optic neuritis with a demyelinating origin
    - Onset of visual loss symptoms in the last 10 days before randomization
    - Expanded Disability Status Scale (EDSS) score between 0 and 5.5
    - Able to understand study information and provide signed written informed consent
    - Affiliated to/beneficiary of the French Health Social Security System
    E.4Principal exclusion criteria
    • Optic neuropathy of non-demyelinating origin: infectious diseases, ischemic, traumatic, tumor or other
    • Known Neuromyelitis optica with autoantibodies against aquaporin-4 (AQP4-Abs)
    • Patients with widespread and symmetric white matter alterations in the screening MRI suggestive of other demyelinating disorders (e.g. metabolic disorders, mitochondrial disorders)
    • Active, chronic disease (or stable but treated with immune therapy) of the immune system other than Multiple Sclerosis (MS) (e.g. Sjögren’s disease, systemic lupus erythematosus) or with a known immunodeficiency syndrome (AIDS, hereditary immune deficiency, drug induced immune deficiency)
    • An alternative cause of visual loss (e.g. compressive or infiltrative lesion of the optic nerve, infections, genetic forms of visual loss.
    • Diagnosed with macular oedema, severe myopia (>6 δ) or other disease of the retina at inclusion
    • Known diabetic retinopathy
    • Known glaucoma
    • Mean ganglion cell inner plexiform layer (GCIPL) thickness measured on ocular coherence tomography (Cirrus HD-OCT) below 65µm at inclusion in the affected eye
    • Difference in mean GCIPL thickness measured on Cirrus HD-OCT larger than 20µm between the 2 eyes, when the thinnest GCIPL is measured in the affected eye
    • Treatment started with any disease modifying therapy (DMT) for MS within the last 12 weeks
    • Requiring an anticipated switch of DMT within 3 months after inclusion
    • Treated with:
    - Class Ia (e.g. quinidine, disopyramide) or Class III (e.g. amiodarone, stalol) anti-arrhythmic drugs at inclusion
    - Concurrently treated with heart-rate-lowering drugs at inclusion (e.g.: β blockers, heart rate lowering Ca2+ channel blockers (e.g. verapamil, diltiazem or ivabradine), digoxin, cholinesterase inhibitors agents, pilocarpine or S1P inhibitors)
    - S1P modulators (e.g. fingolimod, siponimod), anti-CD20 antibodies (e.g. rituximab, ocrelizumab), natalizumab, cladribine and alemtuzumab
    • Treatment for MS or Myelin oligodendrocyte glycoprotein–immunoglobulin G (MOG-IgG) associated disorder (MOGAD) other than : Interferon beta, Glatiramer acetate, Dimethyl fumarate, Teriflunomide, Mycophénolate mofetil, Azathioprine
    • Any severe cardiac disease or significant findings on the screening ECG
    • Past history of convulsion and/or seizure other than petit mal
    • Known history of, or concomitant severe hepatic, gastrointestinal, cardiovascular, respiratory, hematological, renal, dermatological or psychiatric disease or substance abuse or any condition that, in the opinion of the Investigator might interfere with the evaluation of study treatment or warrant exclusion
    • Patients who, in the opinion of the Investigator, have any clinically relevant findings that warrant exclusion. Examples of clinically relevant problems include, but are not limited to, serious non-malignancy-associated medical conditions that may be expected to limit life expectancy or significantly increase the risk of SAEs and any condition, psychiatric, substance abuse, or otherwise, that, in the opinion of the Investigator, would preclude informed consent, consistent follow-up, or compliance with any aspect of the study
    • History of malignancy within 5 years, other than effectively treated carcinoma in-situ of the cervix, or adequately treated non-metastatic squamous or basal cell carcinoma of the skin
    • Active, chronic diabetes or uncontrolled hypertension
    • Active systemic bacterial, viral or fungal infections, including tuberculosis
    • Known Severe renal insufficiency (GFR <30 ml/min/1.73 m2)
    • Female patients of child-bearing potential who are unwilling to use an effective contraception while enrolled on study and for the duration of the study.
    • Breastfeeding or pregnant women
    • Treatment with any investigational agent within 4 weeks prior to randomization or 5 half-lives of the investigational drug (whichever is longer)
    • Magnetic Resonance Imaging (MRI) contraindications or incapacity to undergo it
    • Any medically unstable condition, as assessed by the primary treating physician or the Investigator
    • History of hypersensitivity to any of the study drugs or to drugs of similar chemical classes
    • Patient is the Investigator or any sub-Investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the protocol
    E.5 End points
    E.5.1Primary end point(s)
    Assessment of the primary endpoint will be carried through :
    Safety parameters: Incidence of adverse events at month 6 in ACT-01-treated subjects versus placebo treated subjects
    E.5.1.1Timepoint(s) of evaluation of this end point
    Month 6
    E.5.2Secondary end point(s)
    Main secondary endpoints:
    • Change in ganglion cell and inner plexiform layer thickness in the affected eye measured with optical coherence tomography (OCT) at month 1, month 3, month 6 and month 12 compared to baseline in the affected eye
    • Change in Retinal Nerve Fibre Layer thickness in the affected eye measured with OCT at month 1, month 3, month 6 and month 12 compared to baseline in the affected eye
    Other secondary endpoints:
    • Change on the 2.5% ETDRS Low Contrast Letter Acuity chart and ETDRS high contrast visual acuity measured at M1, M3, M6 and M12 in the affected eye compared to baseline
    • Change of Humphrey Visual Field evaluations measured in the affected eye at month 1, month 3, month 6 and month 12 compared to baseline
    • Change of latency and amplitude of Visual Evoked Potential at month 3, month 6 and month 12 compared to baseline
    • Change of Expanded Disability Status Scale at month 1, month 3, month 6 and month 12 compared to baseline
    • Rate of treatment switch at 12 months for subjects receiving Disease modifying therapy for multiple sclerosis
    • Safety parameters: Incidence of adverse events at M12 in ACT-01-treated subjects versus placebo
    • Pharmacokinetics parameters: Individual and mean plasma ACT-01 concentration time data at t1 (first day of treatment): 0 (pre-dose) and 0.5 h, 1h, 1.5h, 2h, 3h and 4h post start of the infusion; t2-t5: 0 (pre-dose); with Cmax (Maximum concentration), Tmax (time to maximum observed concentration, AUC0-t (Area under the concentration time curve (AUC) from the time of dosing to the time of the last measurable concentration)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Month 6
    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 No
    E.6.6Pharmacokinetic Yes
    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) 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 trial2
    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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 Trial is defined as either the date of the last visit of the last subject to complete the post-treatment follow-up, or the date of receipt of the last data point from the last subject that is required for primary, secondary and/or exploratory analysis, as prespecified in the protocol, whichever is the later date.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    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: 36
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state36
    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)
    No plans for treatment or care for treatment after participation different from the expected local standard of care
    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 Decision2020-09-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-11-06
    P. End of Trial
    P.End of Trial StatusOngoing
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