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    Summary
    EudraCT Number:2021-006794-37
    Sponsor's Protocol Code Number:VRDN-001-101
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-03-22
    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 number2021-006794-37
    A.3Full title of the trial
    A multiple ascending dose (MAD) safety, tolerability and efficacy study of VRDN-001, a humanized monoclonal antibody directed against the IGF-1 receptor, in normal healthy volunteers (NHVs) and subjects with thyroid eye disease (TED)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Safety, Tolerability and Efficacy Study of VRDN 001 in Healthy Volunteers and Persons With Thyroid Eye Disease (TED)
    A.3.2Name or abbreviated title of the trial where available
    A Safety, Tolerability and Efficacy Study of VRDN 001
    A.4.1Sponsor's protocol code numberVRDN-001-101
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05176639
    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 SponsorViridian Therapeutics, 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 supportViridian Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationViridian Therapeutics, Inc.
    B.5.2Functional name of contact pointViridian Clinical Trials Desk
    B.5.3 Address:
    B.5.3.1Street Address221 Crescent Street, Suite 401
    B.5.3.2Town/ cityWaltham
    B.5.3.3Post codeMA 02453
    B.5.3.4CountryUnited States
    B.5.6E-mailviridianclinicaltrials@viridiantherapeutics.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 nameVRDN-001
    D.3.2Product code VRDN-001
    D.3.4Pharmaceutical form Concentrate 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 INNn/a
    D.3.9.1CAS number 2728655-31-8
    D.3.9.2Current sponsor codeVRDN-001
    D.3.9.3Other descriptive nameInsulin-like growth factor-1 receptor (IGF-1R) inhibitor
    D.3.9.4EV Substance CodeSUB265864
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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) 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 injection/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
    Thyroid eye disease (TED)
    E.1.1.1Medical condition in easily understood language
    Thyroid eye disease (TED)
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 23.1
    E.1.2Level LLT
    E.1.2Classification code 10084358
    E.1.2Term Thyroid eye disease
    E.1.2System Organ Class 100000004853
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To establish the safety, tolerability, and preliminary efficacy of VRDN-001, and the pharmacokinetic (PK) and pharmacodynamic (PD) profiles of VRDN-001 in NHV and TED patients over a dose range of 3.0 to 20.0 mg/kg.
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    TED subjects:
    1.Be able to understand the study procedures and the risks involved and be willing to provide written informed consent before the first study-related activity
    2.Be an adult male or female subject, at least 18 years of age or older
    3.Have had a clinical diagnosis of Graves’ disease associated with TED with a CAS of ≥4 on the 7-item scale for the study eye
    4.Have moderate to severe (an appreciable impact on daily living) active TED associated with at least one of the following: lid retraction of ≥2 mm, moderate or severe soft tissue involvement, proptosis of ≥3 mm above normal values for race and gender, and/or periodic or constant diplopia)
    5.Have documented evidence of ocular symptoms or signs associated with TED that began within 1 year prior to study screening
    6.Be euthyroid, or with only mild hyper- or hypothyroidism defined as free thyroxine (FT4) and free triiodothyronine (FT3) levels <50% above or below the normal limits at screening. Every effort must be made to correct any mild hypo- or hyperthyroidism promptly and maintain a euthyroid state for the entire duration of the study. Thyroidectomy is NOT an exclusion
    7.Not require immediate surgical ophthalmological intervention in the study eye for any reason
    8.Have alanine aminotransferase (ALT) or aspartate aminotransferase (AST) levels <3 in times the upper limit of normal (ULN) or serum creatinine <1.5 times the ULN for their age and diabetic subjects must have a glycated hemoglobin (HbA1c) of <8.5% with no new diabetic medication (oral or insulin) commenced within the previous 12 weeks, or have had more than a 10% change in the dose of a currently prescribed diabetic medication within the previous 12 weeks
    9.Have a negative serum pregnancy test at screening and further negative urine tests immediately before each dose of study medication and other applicable visits following the last dose of study medication as described in Appendices 1B and 1C if the subject is a woman of childbearing potential (including those with <2 years since the onset of menopause, amenorrhea for <1 year, or not surgically sterile); such subjects must agree to use an acceptable method of contraception such as a condom and a second highly effective contraception from Screening up to and including 100 days after the last dose of study medication. If the subject is initiating hormonal contraception at time of Screening or within one cycle of Day 1, subject agrees to use a double-barrier method of contraception until completing one-full cycle of hormonal contraception. An acceptable double-barrier combination method is a condom with either diaphragm or sponge with spermicide.
    10.Be surgically sterile males for at least 6 weeks, or agree to use an acceptable method of contraception such as a condom and a second highly effective contraception from Screening up to and including 100 days after the last dose of study medication
    11.Be willing and able to comply with all the requirements of the protocol for the entire duration of the study
    E.4Principal exclusion criteria
    TED Subjects:
    1.Have received prior treatment with another anti-IGF-1R mAb
    2.Have decreased best corrected visual acuity (BCVA) in the study eye, defined as ≥2 Snellen lines, due to optic neuropathy, new visual field defect, or color defect secondary to optic nerve involvement within previous 6 months
    3.Have corneal decompensation in the study eye unresponsive to medical management
    4.Have a decrease in CAS of ≥2 points between screening assessment and Day -1
    5.Have a decrease in proptosis of ≥2 mm in the study eye between screening assessment and Day -1
    6.Have had previous orbital irradiation or surgery in the study eye for TED
    7.Have known history of clinically significant ear pathology, ear surgery or hearing impairment
    8.Have inflammatory bowel disease (e.g. biopsy proven or clinical evidence of inflammatory bowel disease).
    9.Have used oral corticosteroids for any condition, including TED, within 4 weeks prior to the first dose of study medication (topical is permitted)
    10.Have received rituximab, or tocilizumab, or other immunosuppressive agent within 90 days prior to the first dose of study medication
    11.Have received an investigational agent for any condition within 60 days
    12.Have a pre-existing ophthalmic condition in the study eye, which in the opinion of the Investigator, would confound interpretation of the study results
    13.Be a pregnant or lactating woman
    14.Be an active alcoholic or illicit drug user or considered at high risk of relapse by the Investigator
    15.Have a known hypersensitivity to any of the components of VRDN-001 or placebo formulations, or prior hypersensitivity to monoclonal antibodies (mAbs)
    16.Have any condition, which in the opinion of the Investigator, would preclude inclusion in the study
    17.Have a positive test to human immunodeficiency virus (HIV-1 and HIV-2)
    18.Have a positive test for active hepatitis B or hepatitis C infection
    19.Have previously participated in this study or any study of VRDN-001
    E.5 End points
    E.5.1Primary end point(s)
    SAFETY ENDPOINTS:
    Adverse Events (AEs), Serious Adverse Events (SAEs) and Laboratory Assessments will be monitored and recorded throughout the duration of the study.
    PRIMARY EFFICACY ENDPOINT:
    Proptosis Responder Rate (i.e., reduction of proptosis of ≥ 2 mm from baseline as measured by exophthalmometer) at 6 weeks in the MAD study, and 24 weeks in the extension cohorts
    E.5.1.1Timepoint(s) of evaluation of this end point
    Throughout the duration of the study
    At 6 and 12 weeks in the MAD study, and 24 weeks in the extension cohorts
    E.5.2Secondary end point(s)
    SECONDARY EFFICACY ENDPOINTS:
    Proptosis Responder Rate (i.e., reduction of proptosis of ≥ 2 mm from baseline as measured by exophthalmometer) at 12 weeks in the MAD study and at 52 weeks in the extension cohorts.
    Proptosis Responder Rate (i.e., reduction of proptosis of ≥ 2 mm from baseline as measured by magnetic resonance imaging [MRI]) at 6 and 12 weeks in the MAD study, and 24 and 52 weeks in the extension cohorts.
    Change from Baseline within the study eye (more proptotic) at 6 and 12 weeks in the MAD study, and 24 and 52 weeks in the extension cohorts in the following:
    • Change in measurement of proptosis by MRI
    • Change in measurement of proptosis by exophthalmometer
    • Change in volume of orbital fat as determined by MRI
    • Change in volume of extraocular muscles as determined by MRI
    • Change in facial fat volume as determined by MRI
    • Change in digital measurement of eye color
    • Change in digital and manual measurement of lid retraction
    • Change in clinical activity score (CAS)
    • Change in subjective diplopia score
    • Change in Graves’ Orbitopathy-Quality of Life (GO-QoL) combined score
    • Change in GO-QoL activity subscale
    • Change in GO-QoL appearance subscale
    • Change in Snellen best corrected visual acuity (BCVA)
    E.5.2.1Timepoint(s) of evaluation of this end point
    According to the protocol
    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) 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) 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 No
    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 trial4
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA15
    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
    Canada
    United States
    United Kingdom
    European Union
    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.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months1
    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 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: 81
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 15
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers Yes
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 45
    F.4.2.2In the whole clinical trial 96
    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
    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 Decision2022-05-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-11
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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