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    Summary
    EudraCT Number:2018-004089-33
    Sponsor's Protocol Code Number:DYN101-C101
    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:2019-06-04
    Trial results View 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 number2018-004089-33
    A.3Full title of the trial
    A Phase 1/2 trial on the safety, tolerability, pharmacokinetics, pharmacodynamics and exploratory efficacy of DYN101 in patients ≥ 16 years of age with centronuclear myopathies caused by mutations in DNM2 or MTM1
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An early phase human drug trial (called phase 1/2) to investigate DYN101 in patients ≥ 16 years of age with a rare muscle disease group called the centronuclear myopathies caused by genetic changes in DNM2 or MTM1 for the drug’s safety, tolerability, how the body processes the drug as well as interactions of the drug with the body and preliminary effects of the drug.
    A.3.2Name or abbreviated title of the trial where available
    Research Using an Investigational Treatment for CNM (Unite-CNM)
    A.4.1Sponsor's protocol code numberDYN101-C101
    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 SAS
    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 SAS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDynacure SAS
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address300 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 No
    D.2.5.1Orphan drug designation number
    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 myopathies (CNMs) in patients ≥ 16 years of age with mutations in DNM2 or MTM1
    E.1.1.1Medical condition in easily understood language
    Centronuclear myopathies (CNMs) in patients ≥ 16 years of age with mutations in DNM2 or MTM1
    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
    To assess the safety and tolerability of 3 single ascending dose (SAD) levels and 3 multiple ascending dose (MAD) levels of DYN101
    E.2.2Secondary objectives of the trial
    • To assess the pharmacokinetics (PK) of SAD and MAD of DYN101.
    • To explore target engagement in muscle of DYN101.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female aged ≥ 16 years on the date of signing the main ICF.
    2. Have a documented mutation in DNM2 or MTM1.
    3. Meet the following laboratory criteria at screening:
    • Platelet count > 150,000/µL,
    • Normal kidney function, as determined by values of blood urea nitrogen, creatinine, cystatin C, and estimated glomerular filtration rate within normal laboratory reference ranges,
    • Normal liver function, as determined by values of alanine and aspartate aminotransferase, gamma glutamyltransferase, and bilirubin within normal laboratory reference ranges.
    4. Have a symptomatic CNM in the opinion of the investigator, at least mild to moderately affected, i.e. showing clinical symptoms in at least 2 of the domains that will be investigated in this trial (respiratory, muscle strength and function, dysphagia), and be ambulatory, i.e. being able to walk 10 steps, if needed with support/assisted. If a subject is non-ambulatory but highly functioning in the view of the investigator, he/she may be included following discussion with the sponsor.
    5. Have an understanding, ability and willingness to fully comply with visit frequency, trial procedures and restrictions, including contraceptive requirements.
    6. Able to provide written, signed and dated informed consent/assent to participate in the trial. Parental consent (one or both parents) and an assent for subjects < 18 years may be required per local legislation.
    E.4Principal exclusion criteria
    1. Have clinical, laboratory or ultrasound evidence of liver disease.
    Note: Liver ultrasound at screening is mandatory for MTM1 subjects.
    2. Presence of significant co-morbidities or conditions other than CNM or clinically significant findings during screening of medical history, physical examination, laboratory testing, vital signs or ECG recording for which, in the opinion of the investigator and 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).
    3. For female subjects of child-bearing potential: pregnant or breastfeeding, or planning to become pregnant during the trial.
    4. Have a body weight of <34 kg.
    5. Current or past abuse of alcohol or recreational/narcotic drugs (with the exception of caffeine and nicotine), which in the investigator’s opinion would compromise the subject’s safety and/or compliance with the trial procedures.
    6. Currently enrolled in another investigational trial or scheduled to participate in another trial whilst participating in this trial.
    7. Current or relevant history of physical or psychiatric illness, 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.
    8. Intake of any disallowed therapies as noted in the protocol within 12 weeks before the planned first IMP administration.
    9. Known or suspected intolerance or hypersensitivity to IMP ingredients or closely-related compounds, or history of a significant allergic reaction to IMP ingredients as determined by the investigator, such as anaphylaxis requiring hospitalization.
    E.5 End points
    E.5.1Primary end point(s)
    To determine safety and tolerability of SAD and MAD of DYN101, assessed by the number and severity of AEs and SAEs reported from signing of the main ICF onwards until the last trial visit
    E.5.1.1Timepoint(s) of evaluation of this end point
    All visits from baseline to W25 visit and safety follow up call 3 months after last dose
    E.5.2Secondary end point(s)
    • To determine PK parameters of DYN101 in plasma following SAD and MAD of DYN101.
    • To assess effects of DYN101 (including DNM2 mRNA levels and DYN101 concentration) in muscle biopsies.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Blood PK: SAD part: Pre-dose (0h), 30 min post start of infusion, 1h, 2h, 4h, 8h, 24h, 48h and 168h post-dose then 2 weeks, 4 weeks, 8 weeks, 12 weeks and 16 weeks post-dose
    MAD W1 - Pre-dose (0h), 30 min post start of infusion, 1h, 2h, 4h, 8h, 24h, 48h, 72h ( 72h timepoint pre-loading dose) and 168h post-dose
    MAD W12 - Pre-dose, 30 min post start of infusion, 1h, 2h, 4h, 8h, 24h, 48h, 72h and 168h post-dose of Week 12, Day 1
    MAD W25 - 168h post-dose of Week 24*
    Muscle Biopsies:
    2 time points: At SAD baseline (or at MAD baseline for replaced subjects who did not participate in SAD) and At the end of the 12 weeks MAD part (W13)
    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 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 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 trial3
    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 EEA10
    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 years2
    E.8.9.1In the Member State concerned months2
    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 months2
    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: 6
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 10
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 18
    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)
    The subject will receive other treatment and/or have access to other appropriate care by his doctor.
    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 Decision2019-07-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-08-27
    P. End of Trial
    P.End of Trial StatusOngoing
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