Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   43801   clinical trials with a EudraCT protocol, of which   7259   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2019-004602-94
    Sponsor's Protocol Code Number:BIO101_CL04
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-02-03
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2019-004602-94
    A.3Full title of the trial
    A 3-part, Randomized, Double Blind, Adaptive Seamless Phase 1-3 Study to Evaluate the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO101 in Non-Ambulatory Patients with a Genetically Confirmed Diagnosis of Duchenne Muscular Dystrophy and Evidence of Respiratory Deterioration
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    NA
    A.3.2Name or abbreviated title of the trial where available
    Adaptive Seamless Phase 1-3 Study of Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of BIO
    A.4.1Sponsor's protocol code numberBIO101_CL04
    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 SponsorBiophytis
    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 supportBiophytis
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationBiophytis
    B.5.2Functional name of contact pointMounia Chabane De Saint Aubin
    B.5.3 Address:
    B.5.3.1Street Address14 Avenue de l' Opéra
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75001
    B.5.3.4CountryFrance
    B.5.4Telephone number+33(0)144 27 23 87
    B.5.6E-mailmounia.chabane@biophytis.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/3/18/2030 (EMA/OD/020/18)
    D.3 Description of the IMP
    D.3.2Product code BIO101
    D.3.4Pharmaceutical form Powder for oral suspension
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBIO101
    D.3.9.1CAS number 5289-74-7
    D.3.9.2Current sponsor codeBIO101
    D.3.9.3Other descriptive nameBIO101
    D.3.9.4EV Substance CodeSUB193629
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number12.5
    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.IMP: 2
    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/2/18/2030 (EMA/OD/020/18)
    D.3 Description of the IMP
    D.3.2Product code BIO101
    D.3.4Pharmaceutical form Powder for oral suspension
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBIO101
    D.3.9.1CAS number 5289-74-7
    D.3.9.2Current sponsor codeBIO101
    D.3.9.3Other descriptive nameBIO101
    D.3.9.4EV Substance CodeSUB193629
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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 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.IMP: 3
    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/3/18/2030 (EMA/OD/020/18)
    D.3 Description of the IMP
    D.3.2Product code BIO101
    D.3.4Pharmaceutical form Powder for oral suspension
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNBIO101
    D.3.9.1CAS number 5289-74-7
    D.3.9.2Current sponsor codeBIO101
    D.3.9.3Other descriptive nameBIO101
    D.3.9.4EV Substance CodeSUB193629
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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 placeboPowder for oral suspension
    D.8.4Route of administration of the placeboOral 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 regardless the genotype
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscular Dystrophy regardless the genotype
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.1
    E.1.2Level PT
    E.1.2Classification code 10052655
    E.1.2Term Duchenne muscular dystrophy gene carrier
    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
    Part 1
    To evaluate the safety, tolerability and PK profile of BIO101 and its main metabolites after a single dose (Day 1) and after multiple doses at Day 7, 14 and 56

    Part 2
    To evaluate the safety, tolerability, and efficacy on respiratory function, of BIO101 following 48 weeks double blind dosing, in a small population (48 participants)

    Part 3
    To evaluate the safety, tolerability, and efficacy on respiratory function of BIO101 following 48 weeks double blind dosing, in a large population (100-200 participants)
    E.2.2Secondary objectives of the trial
    Part 2
    Population PK analysis to characterize the PK of BIO101 following 48 weeks double blind dosing
    To determine the number of new participants to enroll into confirmatory Part 3

    Part 3
    To further evaluate efficacy of BIO101 versus placebo and external controls on strength and respiratory function of participants according to their initial disease status
    To evaluate the safety and tolerability of BIO101 versus placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Part 1
    1. Genetically confirmed DMD
    2. Age 12 and above at screening
    3. Non-ambulant patients (The non-ambulatory patient is defined as non-ambulant for at least 6 months with inability to walk 10 meters without assistance, by another person or device)
    4. FVC ≤80% and >45% of predicted value based on most recent assessment noted in the patient's medical record and subsequently confirmed at Screening and at Baseline and who, in the opinion of the investigator are in the respiratory function decline phase.
    5. Stable cardiac function, defined as:
    • No clinically important ECG abnormalities.
    • Echocardiography left ventricular ejection fraction (LVEF) at screening >50%.
    6. If clinically indicated, approved concomitant treatment for DMD is allowed (e.g., Eteplirsen [Exondys 51™], Golodirsen [Vyondys 53™], Ataluren [Translarna™])
    7. On or off corticosteroid therapy
    • If on steroids, including but not limited to Prednisone/ Prednisolone and Deflazacort [Emflaza®]:
    o At a minimum dose of 0.3 mg/Kg/day prednisone equivalent
    o Deflazacort approximately 0.9 mg/Kg/Day
    o As long as the treatment is stable (i.e., unchanged and with no significant side effects) for at least 3 months.
    o As long as the treatment is stable (i.e., unchanged and with no significant side effects) for at least 3 months.

    • Participants who were treated with corticosteroids and stopped (for any reason) will wait for 3 months until they can be eligible for inclusion.
    • Steroid treatment is expected to remain stable during study, except for adjustment for weight.
    8. Participants on antihypertensive, lipid lowering, and/or thyroid replacement therapies, carnitine, creatineare eligible only if they are on a stable dose for at least 1 month prior to screening.
    9. Stable hepatic function:
    • Gamma-glutamyl transferase (GGT) ≤ upper limit of normal (ULN)
    • Total bilirubin < 2×ULN
    • GLDH < 2×ULN
    • Alkaline phosphatase ≤ULN
    • Serum albumin ≥ lower limit of normal (LLN)
    10. Ability to provide reliable and reproducible repeat PEF within 15% of the first assessment (i.e. Baseline vs. Screening)
    11. Ability to provide reliable and reproducible repeat FVC within 15% of the first assessment (i.e. Baseline vs. Screening)
    12. Willing and able to participate and comply with all study procedures.
    • For participants aged <18: Parent or legal guardians will sign an informed consent prior to start of screening procedures and patient will sign an informed ascent.
    • For participants aged 18 and about – patient will sign an informed consent.
    13. Must be accompanied, during the whole study activities, by a parent, guardian or a caregiver, who is 18 years or older.
    14. Sexually active participants must use double contraception (e.g. the male uses a condom and the female uses an oral contraceptive pills)
    15. For France only: Being affiliated with a European Social Security.
    E.4Principal exclusion criteria
    Participants will not be eligible to participate in any part of this study (Part 1, Part 2, or Part 3) if one or more of the following is/are met:
    1. Any serious medical/surgical or psychiatric condition/illness that in the opinion of the Investigator would jeopardize participant’s safety or would interfere with the study assessments/results.
    2. History or active signs or symptoms of gallbladder/biliary disease (e.g., previous episodes of cholestasis/biliary tract obstruction, cholelithiasis, cholecystitis). Of note, history of cholecystectomy and no active biliary signs or symptoms, is not an exclusion criterion.
    3. Any known allergies to products likely to be used in the study (e.g., antiseptics, anesthetics), known hypersensitivity to any of the ingredients, or excipients of the study drug.
    4.Participation in other investigational study within 30 days or 5 half lives (whichever is longer) prior to randomization.
    5. Patients undergoing Idebenone (Raxone ®)
    6. Intellectual disability or behavioral problem such that he cannot comply with study procedures.
    7. Patients who require daytime invasive ventilatory assistance(defined as use of any assisted ventilation while awake).
    8. Renal disease requiring dialysis, or known renal insufficiency (moderate or severe reduction of eGFR≤30 ml/min/1.73 m2, based on Cockroft & Gault formula)
    9. Asthma, bronchiolitis/COPD, bronchiectasis, emphysema, pneumonia or the presence of any other non-DMD respiratory illness that affects PEF.
    E.5 End points
    E.5.1Primary end point(s)
    Efficacy:
    Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF % predictive) at Week 48 (assessed by hospital-based spirometry measurements)

    E.5.1.1Timepoint(s) of evaluation of this end point
    During the trial
    E.5.2Secondary end point(s)
    Efficacy:
    Change From Baseline in Forced Vital Capacity (FVC% predictive) at Week 48 (assessed by hospital-based spirometry measurements)

    Safety:
    Type, frequency, intensity, timing, and relationship to study drug of:
    • Adverse events (AEs), discontinuations due to AEs, serious AEs (SAEs), deaths
    • 12-lead triplicate electrocardiograms (ECGs)
    • Local Clinical laboratory tests
    • Physical examination and vital signs
    • Anthropometry

    Pharmacokinetics:
    • Non-compartmental PK parameters including maximum concentration (Cmax), time to Cmax (tmax), area under the curve (AUC) and other parameters for participants at Part 1:
    o On Day 1 (pre-treatment)
    o On Day 7, 14 and 56
    • Population PK parameters including apparent clearance and volume of distribution (for participants of parts 2 and Part 3)
    E.5.2.1Timepoint(s) of evaluation of this end point
    During this trial
    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 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) 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
    3 part adaptive seamless Phase 1-3 study
    E.7.2Therapeutic exploratory (Phase II) No
    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 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 EEA2
    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
    The end of the study is defined as the date of the last visit of the last participant in the study.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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 Yes
    F.1.1Number of subjects for this age range: 36
    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: 36
    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 Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    The study population will consist of participants who are male and at least 12 years of age who have been diagnosed with genotypically confirmed DMD.
    F.3.3.7Others Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 260
    F.4.2.2In the whole clinical trial 260
    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 Decision2020-03-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-03-27
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA