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    Summary
    EudraCT Number:2021-005478-24
    Sponsor's Protocol Code Number:DYNE251DMD201
    National Competent Authority:Ireland - HPRA
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2022-08-24
    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 ConcernedIreland - HPRA
    A.2EudraCT number2021-005478-24
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of DYNE-251 in Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
    A.4.1Sponsor's protocol code numberDYNE251DMD201
    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 SponsorDyne 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 supportDyne Therapeutics, Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDyne Therapeutics, Inc
    B.5.2Functional name of contact pointDyne Clinical Trials
    B.5.3 Address:
    B.5.3.1Street Address1560 Trapelo Rd
    B.5.3.2Town/ cityWaltham, MA
    B.5.3.3Post code02451
    B.5.3.4CountryUnited States
    B.5.4Telephone number1781317-1919
    B.5.6E-mailclinicaltrials@dyne-tx.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 nameDYNE-251
    D.3.2Product code DYNE-251
    D.3.4Pharmaceutical form 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 INNNA
    D.3.9.1CAS number 2725863-42-1
    D.3.9.2Current sponsor codeDYNE-251
    D.3.9.3Other descriptive nameFragment antibody targeting human TfR1 conjugated to phosphorodiamidate morpholino oligomer
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNNA
    D.3.9.1CAS number 2725863-42-1
    D.3.9.2Current sponsor codeDYNE-251
    D.3.9.3Other descriptive nameFragment antibody targeting human TfR1 conjugated to phosphorodiamidate morpholino oligomer
    D.3.9.4EV Substance CodeSUB273613
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number350
    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 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
    Duchenne Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Muscular disease causing progressive loss of skeletal, pulmonary, cardiac and muscle function
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10013801
    E.1.2Term Duchenne muscular dystrophy
    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 evaluate the safety and tolerability of multiple IV doses of DYNE-251 administered to participants with DMD

    To evaluate dystrophin protein levels in muscle tissue following multiple IV doses of DYNE-251 administered to participants with DMD
    E.2.2Secondary objectives of the trial
    To evaluate the effects on muscle tissue exon skipping, percent dystrophin-positive fibers (PDPFs), and blood creatine kinase (CK) following multiple IV doses of DYNE-251 administered to participants with DMD

    To evaluate muscle function following multiple IV doses of DYNE-251 administered to participants with DMD

    To evaluate plasma and muscle tissue PK following multiple IV doses of DYNE-251
    administered to participants with DMD

    To evaluate the immunogenicity of multiple IV doses of DYNE-251 administered to participants with DMD
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Age 4 to 16 years inclusive, at the time of informed consent/assent.
    • Male with a confirmed diagnosis of DMD and with a mutation in the dystrophin gene characterized by exon deletion amenable to exon 51 skipping.
    • Upper extremity muscle group that is amenable to muscle biopsy.
    • Brooke Upper Extremity Scale score of 1 or 2.
    • Ambulatory or non-ambulatory. A non-ambulatory participant must have been non-ambulatory for <2 years before enrolment.
    • Receiving a stable dosage of glucocorticoids for at least 12 weeks prior to the start of study drug administration.
    • Left ventricular ejection fraction of ≥50% by echocardiogram or ≥55% by cardiac magnetic resonance imaging (MRI).
    E.4Principal exclusion criteria
    • Uncontrolled clinical symptoms and signs of congestive heart failure (CHF).
    • Any change in prophylaxis/treatment for CHF within 3 months prior to the start of study treatment.
    • History of major surgical procedure within 12 weeks prior to the start of study drug administration or an expectation of a major surgical procedure during the study.
    • Requirement of daytime ventilator assistance.
    • Percent predicted FVC <40 % (applies only for participants who are age ≥7 years).
    • Receipt of eteplirsen, or alternative exon-skipping/dystrophin-modifying therapy, within 12 weeks of randomization.
    • Receipt of non-exon skipping investigational drug within 4 months before the start of study drug administration.
    • Receipt of gene therapy at any time.
    E.5 End points
    E.5.1Primary end point(s)
    1. Number and proportion of participants with treatment-emergent adverse events (TEAEs), treatment emergent serious adverse events (TESAEs), TEAEs considered related to study drug, and TEAEs leading to discontinuation from study drug and discontinuation from the study.
    2. Change from Baseline in dystrophin protein levels in muscle tissue as determined by Western blot analysis at Week 25
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. All visits from screening to end of study
    2. Screening and week 25 visit
    E.5.2Secondary end point(s)
    For cohorts dosed on a Q4W or Q8W interval with a second biopsy performed at Week 25:
    - Change from Baseline in muscle tissue exon 51 skipping levels at Week 25
    - Change from Baseline in muscle tissue PDPF at Week 25
    - Change from Baseline in blood CK levels up to Week 145

    For cohorts dosed on a Q8W interval with a second biopsy performed at Week 49:
    - Change from Baseline in dystrophin protein levels in muscle tissue as determined by Western blot analysis at Week 49
    - Change from Baseline in muscle tissue exon 51 skipping levels at Week 49
    - Change from Baseline in muscle tissue PDPF at Week 49
    - Change from Baseline in blood CK levels up to Week 145
    - Change from Baseline in North Star Ambulatory Assessment (NSAA) total score in ambulatory participants up to Week 145
    - Change from Baseline in time to rise from floor in ambulatory
    participants up to Week 145
    - Change from Baseline in 10-meter run/walk (10MRW) time in
    ambulatory participants up to Week 145
    - Change from Baseline in performance upper limb (PUL) scale Version 2.0 score up to Week 145
    - Change from Baseline in percent predicted forced vital capacity (FVC) up to Week 145
    - Change from Baseline in stride velocity 95th centile (SV95C) in
    ambulatory participants up to Week 145
    - Maximum observed plasma drug concentration (Cmax)
    - Time to maximum observed plasma drug concentration (tmax)
    - Area under the plasma drug concentration-time curve (AUC) from time 0 to the last quantifiable concentration (AUCtlast)
    - Areas under the plasma drug concentration versus time curve from time 0 (dosing) extrapolated to time infinity (AUC∞)
    - Apparent terminal phase elimination rate constant (λZ)
    - Apparent terminal elimination half-life (t½)
    - Total body clearance (CL)
    - Volume of distribution at the terminal phase (Vz), if appropriate
    - Volume of distribution at steady state (Vss), if appropriate
    - Tissue phosphorodiamidate morpholino oligomer (PMO) concentration
    - Incidence of antidrug antibodies (ADAs)
    E.5.2.1Timepoint(s) of evaluation of this end point
    - For endpoints 1 and 2: Screening and week 25 visit
    - For endpoint 3: Screening, D1/pre-dose, D3, D8, W5, W9, W13, W17, W21, W25, W37, W29, W33, W41, W45, W49, W53, W57, W65, W69, W77, W81, W89, W93, W101, W105, W113, W117, W125, W129, W137, W141, W61, W85, W109, W133, W73, W121, W97, W145
    - For endpoints 4 through 8: Screening, W13, W25, W37, W49, W73, W121, W97, W145
    - For endpoints 9 through 17: D1/Pre-dose, D1/EOI, D1/15min-post, D1/1h-post, D1/4h-post, D1/8h-post, D1/12h-post, D2, D3, D8, W5, W25, W49, W97, W145
    - For endpoint 18: Screening and week 25
    - For endpoint 19: Screening, W5, W9, W13, W17, W21, W25, W37, W29, W33, W41, W45, W49, W53, W57, W65, W69, W77, W81, W89, W93, W101, W105, W113, W117, W125, W129, W137, W141, W61, W85, W109, W133, W73, W121, W97, W145
    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 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 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 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
    Australia
    Canada
    United Kingdom
    United States
    Belgium
    Ireland
    Italy
    Spain
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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 Yes
    F.1.1Number of subjects for this age range: 112
    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: 56
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 56
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 53
    F.4.2.2In the whole clinical trial 112
    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)
    Open label study/registry.
    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 Decision2022-10-28
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial Status
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