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    Summary
    EudraCT Number:2020-004608-32
    Sponsor's Protocol Code Number:DYN101-C102
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:
    Date on which this record was first entered in the EudraCT database:2021-05-27
    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 ConcernedGermany - BfArM
    A.2EudraCT number2020-004608-32
    A.3Full title of the trial
    A Phase 1/2, multicenter, open-label, dose-confirmation trial to evaluate the safety and preliminary efficacy of DYN101 in subjects 2 to 17 years of age with centronuclear myopathy caused by mutations in MTM1 or DNM2.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    N/A
    A.3.2Name or abbreviated title of the trial where available
    Research Using an Investigational Treatment for CNM (DyNaMic)
    A.4.1Sponsor's protocol code numberDYN101-C102
    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 SponsorDynacure
    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 supportDynacure
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDynacure
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street AddressBioparc 3, 850 Boulevard Sébastien Brant
    B.5.3.2Town/ city67400 Illkirch – Graffenstaden
    B.5.3.3Post code67400
    B.5.3.4CountryFrance
    B.5.4Telephone number+336 98 75 98 44
    B.5.6E-mailChris.Freitag@Dynacure.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/19/2171
    D.3 Description of the IMP
    D.3.1Product nameDYN101
    D.3.2Product code DYN101
    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 INNDYN101
    D.3.9.2Current sponsor codeDYN101
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Centronuclear myopathy (CNM), in subjects 2 to 17 years of age (all ages inclusive)caused by mutations in the myotubularin1 (MTM1) or dynamin 2 (DNM2) gene
    E.1.1.1Medical condition in easily understood language
    Centronuclear myopathy (CNM), in subjects 2 to 17 years of age (all ages inclusive)caused by mutations in the myotubularin1 (MTM1) or dynamin 2 (DNM2) gene
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level HLT
    E.1.2Classification code 10028640
    E.1.2Term Myopathies
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Part 1:
    • To assess the safety and tolerability of multiple doses of DYN101.
    Part 2:
    • To assess the safety and tolerability of multiple doses of DYN101.
    E.2.2Secondary objectives of the trial
    Part 1:
    •To assess the plasma and muscle pharmacokinetics (PK) of multiple doses of DYN101.
    •To explore the pharmacodynamics (PD) of multiple doses of DYN101 in muscle biopsies.

    Part 2:
    •To assess the plasma PK of multiple doses of DYN101.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Subject must be male or female aged ≥2 to <18years on the date the main ICF is signed.
    2.Subject must have a clinically symptomatic CNM, with a documented MTM1orDNM2 mutation.
    3.Subjects must have impaired muscle function as evidenced by:
    -MFM20 score between 5% and 80% for subjects ≥2 and <6 years of age, or
    -MFM32 score between 5% and 80% for subjects ≥6 years of age.
    4.Subject should have sufficient skeletal muscle (vastus lateralis, gastrocnemius, or biceps brachii as last resort) to perform 2 open muscle biopsies during the trial, as determined by ultrasound imaging at screening.
    5.Subject must meet have platelet count >150,000/μLat screening.
    6.Parent(s) or legally-authorized representative must be able to provide written, signed and dated informed consent for their child to participate in the trial. Informed assent can be obtained from the child according to local regulations.
    7.Parent(s) or legally-authorized representative must be at or above the age of legal consent in the jurisdiction of the country in which the trial is taking place.
    8.Subject, parent(s), and/or legally-authorized representative must have an understanding, ability, and willingness to fully comply with visit frequency, trial procedures, videorecording of assessments where applicable, and restrictions, including contraceptive requirements.
    E.4Principal exclusion criteria
    1. Subject has evidence of clinically significant liver disease with:
    -alkaline phosphatase, alanine aminotransferase, aspartate aminotransferase, gamma glutamyltransferase, or bilirubin values > 2x upper limit of normal (ULN); and/or
    -clinically significant abnormal liver ultrasound results;and/or-clinically significant abnormal hepatic elastography results.
    2.Subject has evidence of clinically significant renal disease with:
    -Cystatin C ≥ULN and urinary protein to creatinine ratio ≤150 mg/g or 15mg/mmol; and-Total daily protein >50 mg/24 hours.
    3. Presence of significant comorbidities or conditions other than CNM or clinically significant findings during screening of medical history, physical examination, clinical laboratory evaluation, vital signs, or ECG recording for which, in the opinion of the investigator and/or the medical monitor, participation would not be in the best interest of the subject (e.g.compromise the safety or well-being) or that could prevent, limit, or confound the protocol-specified assessments (e.g.taking a muscle biopsy).
    4. Currently enrolled in any interventional trial or scheduled to participate in such a trial whilst participating in the current trial.
    5. Has previously received gene therapy for CNM.
    6.Subject has severe muscle contractures that would preclude the ability to show improvement in the MFM32 assessment, in the opinion of the investigator.
    7. Subject has severe airway malacia which could impact the capacity to wean off ventilatory support.
    8. Subject requires oxygen supplementation.
    9. For female subjects of childbearing potential: pregnant, breastfeeding, or planning to become pregnant during the trial.
    10. Current or relevant history of physical or psychiatric illness, and/or any medical disorder that may require treatment or make the subject unlikely to fully complete the trial, or any condition that presents undue risk from the IMP or procedures.
    11. Intake of any disallowed therapies by the subject, within 12 weeks before the planned first IMP administration.
    12. Known or suspected intolerance or hypersensitivity to IMP ingredients or closely related compounds.
    13. Parent(s) or legally-authorized representative are legally incapacitated or have limited legal capacity, or have lack of mental capacity to fully understand the protocol requirements and ensure completion of all required trial procedures.
    E.5 End points
    E.5.1Primary end point(s)
    Part 1:
    •To assess the safety and tolerability of multiple doses of DYN101.
    Part 2:
    •To assess the safety and tolerability of multiple doses of DYN101.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Part 1: After 12 weeks of treatment with DYN101 at the starting dose level.
    Part 2: After 12 weeks of treatment with DYN101 at the newly selected dose level (if required).
    E.5.2Secondary end point(s)
    Part 1:
    •To assess the plasma and muscle pharmacokinetics (PK) of multiple doses of DYN101.
    •To explore the pharmacodynamics (PD) of multiple doses of DYN101 in muscle biopsies.
    Part 2:
    •To assess the plasma PK of multiple doses of DYN101.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Part 1: After 12 weeks of treatment with DYN101 at the starting dose level.
    Part 2: After 12 weeks of treatment with DYN101 at the newly selected dose level (if required).
    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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of DYN101
    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 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 No
    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.8.2.4Number of treatment arms in the trial1
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA5
    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 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
    last visit of the last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months6
    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: 9
    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: 6
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 3
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 0
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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 Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Minor patient population
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 9
    F.4.2.2In the whole clinical trial 9
    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)
    If, after analysis, the results would be encouraging (i.e acceptable safety/tolerability, ASO concentration in muscle biopsy acceptable, and a degree of benefit for the subjects is observed), the sponsor will consider providing IMP to subjects after the last visit in this trial.
    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 Decision2021-10-22
    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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