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   7272   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:2017-001223-49
    Sponsor's Protocol Code Number:PTC124-GD-041DMD
    National Competent Authority:Bulgarian Drug Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-10-11
    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 ConcernedBulgarian Drug Agency
    A.2EudraCT number2017-001223-49
    A.3Full title of the trial
    A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to evaluate the safety and efficacy of Ataluren versus placebo in people who have specific genetic disease Duchenne Muscular Dystrophy
    A.4.1Sponsor's protocol code numberPTC124-GD-041DMD
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01826487
    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 SponsorPTC 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 supportPTC Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedpace Inc
    B.5.2Functional name of contact pointWendy Van den Branden
    B.5.3 Address:
    B.5.3.1Street AddressTechnologielaan 11
    B.5.3.2Town/ cityLeuven
    B.5.3.3Post codeB-3001
    B.5.3.4CountryBelgium
    B.5.4Telephone number+32 (0)16 39 18 70 2703
    B.5.5Fax number+32(0)78 48 03 51
    B.5.6E-mailregsubmissions@medpace.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 Yes
    D.2.1.1.1Trade name Translarna 125 mg granules for oral suspension
    D.2.1.1.2Name of the Marketing Authorisation holderPTC Therapeutics International Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/05/278
    D.3 Description of the IMP
    D.3.1Product nameataluren
    D.3.2Product code PTC124
    D.3.4Pharmaceutical form Granules in sachet
    D.3.4.1Specific paediatric formulation No
    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 INNATALUREN
    D.3.9.1CAS number 775304-57-9
    D.3.9.2Current sponsor codePTC124
    D.3.9.4EV Substance CodeSUB89249
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number125
    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 Yes
    D.2.1.1.1Trade name Translarna 250 mg granules for oral suspension
    D.2.1.1.2Name of the Marketing Authorisation holderPTC Therapeutics International Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/05/278
    D.3 Description of the IMP
    D.3.1Product nameataluren
    D.3.2Product code PTC124
    D.3.4Pharmaceutical form Granules in sachet
    D.3.4.1Specific paediatric formulation No
    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 INNATALUREN
    D.3.9.1CAS number 775304-57-9
    D.3.9.2Current sponsor codePTC124
    D.3.9.4EV Substance CodeSUB89249
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number250
    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 Yes
    D.2.1.1.1Trade name Translarna 1000 mg granules for oral suspension
    D.2.1.1.2Name of the Marketing Authorisation holderPTC Therapeutics International Limited
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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/3/05/278
    D.3 Description of the IMP
    D.3.1Product nameataluren
    D.3.2Product code PTC124
    D.3.4Pharmaceutical form Granules in sachet
    D.3.4.1Specific paediatric formulation No
    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 INNATALUREN
    D.3.9.1CAS number 775304-57-9
    D.3.9.2Current sponsor codePTC124
    D.3.9.4EV Substance CodeSUB89249
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1000
    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 placeboGranules in sachet
    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
    Nonsense Mutation Duchenne Muscular Dystrophy
    E.1.1.1Medical condition in easily understood language
    Duchenne Muscular Dystrophy
    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 determine the effect of ataluren on ambulation and endurance as assessed by the 6-minute walk test (6MWT)
    E.2.2Secondary objectives of the trial
    • To determine the effects of ataluren on ambulation and burst activity as assessed by timed function tests
    • To determine the effects of ataluren on lower-limb muscle function as assessed by the North Star Ambulatory Assessment (NSAA)
    • To assess the safety profile of ataluren
    Evaluate the correlation between plasma concentration of ataluren and functional outcomes
    ‚óŹ Evaluate the plasma pharmacokinetic (PK) profile of ataluren
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    indicating that the subject (and/or his parent/legal guardian) has been
    informed of all pertinent aspects of the trial. Note: If the study
    candidate is considered a child under local regulation, a parent or legal
    guardian must provide written consent prior to initiation of study
    screening procedures and the study candidate may be required to
    provide written assent. The rules of the responsible institutional review
    board/ethics committee (IRB/EC) regarding whether one or both
    parents must provide consent and the appropriate ages for obtaining
    consent and assent from the subject should be followed.
    2. Male sex.
    3. Age ≥5 years. When Version 3 is implemented or approximately 270
    subjects have enrolled in the study, only subjects aged ≥7 to ≤16 who
    meet the mITT criteria will be eligible to enroll.
    4. Phenotypic evidence of DMD based on the onset of characteristic clinical symptoms or signs (eg, proximal muscle weakness, waddling
    gait, and Gowers' maneuver) by 6 years of age and an elevated serum
    creatine kinase (CK). Medical documentation of phenotypic evidence of
    DMD needs to be provided upon request by the PTC Therapeutics medical
    monitor.
    5. Documentation of the presence of a nonsense point mutation in the
    dystrophin gene as determined by gene sequencing. Note: Review and
    approval of documentation by sponsor or designee is required prior to
    enrollment.
    6. Use of systemic corticosteroids (prednisone/prednisolone or
    deflazacort) for a minimum of 12 months immediately prior to start of
    study treatment, with no significant change in dosage or dosing regimen
    for a minimum of 3 months immediately prior to start of study treatment
    and a reasonable expectation that dosage and dosing regimen will not
    change significantly for the duration of the study. Note: Daily, every
    other day, high-dose weekend, and intermittent regimens permitted
    only. The doses recommended or required are shown in Table 1.
    Increases in corticosteroid dose to adjust for increases in body weight
    will not exclude a subject from participation.
    7. 6MWD ≥150 meters at screening, baseline Day 1, and baseline Day 2.
    When Version 3 is implemented or approximately 270 subjects have enrolled
    in the study, only subjects who meet the mITT criteria, 6MWD ≥ 300 meters at,
    Baseline Day 1 or Day 2, and time to stand from supine ≥ 5 seconds at Baseline Day 1,
    will be eligible to enroll. Note: Personal assistance or use of assistive devices for ambulation
    (eg, short leg braces, long leg braces, or walkers) will not be permitted during the 6MWT.
    8. Results of the two Baseline 6MWD results must be determined as
    valid and results of the Day 2 Baseline 6MWD must be within 20% of the
    Day 1 Baseline 6MWD.
    9. Baseline 6MWD (maximum of valid Day 1 and Day 2 values) must be
    no more than a 20% reduction from the valid Screening 6MWD.
    10. Ability to perform timed function tests (run/walk 10 meters, climb
    4 stairs, descend 4 stairs, stand from supine) within 30 seconds at
    screening and baseline.
    11. In subjects who are sexually active, willingness to abstain from
    sexual intercourse or employ a barrier or medical method of
    contraception during the study drug administration and 4 week followup
    period.
    12. Willingness and ability to comply with scheduled visits, drug
    administration plan, study procedures, laboratory tests, and study
    restrictions. Note: Psychological, social, familial, or geographical factors
    that might preclude adequate study participation (in particular, the
    ability to satisfactorily perform the 6MWT) should be considered
    E.4Principal exclusion criteria
    1. Any change (initiation, change in type of drug, dose modification, schedule modification, interruption, discontinuation, or reinitiation) in prophylaxis/treatment for cardiomyopathy within 1 month prior to start of study treatment.
    2. Ongoing intravenous (IV) aminoglycoside or IV vancomycin therapy.
    3. Prior or ongoing therapy with ataluren.
    4. Known hypersensitivity to any of the ingredients or excipients of the study drug [eg, refined polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, colloidal silica, magnesium stearate].
    5. Exposure to another investigational drug within 6 months prior to start of study treatment, or ongoing participation in any interventional clinical trial. Note: This does not apply to patients receiving deflazacort through an expanded access program.
    6. History of major surgical procedure within 12 weeks prior to start of study treatment, or expectation of major surgical procedure (eg, scoliosis surgery) during the 72-week placebo controlled treatment period.
    7. Ongoing immunosuppressive therapy (other than corticosteroids).
    8. Requirement for daytime ventilator assistance or any use of invasive mechanical ventilation via tracheostomy. Note: Evening non-invasive mechanical ventilation such as use of bi level positive airway pressure (Bi-PAP) therapy is allowed.
    9. Uncontrolled clinical symptoms and signs of congestive heart failure (American College of Cardiology/American Heart Association Stage C or Stage D) [Hunt 2001].
    10. Elevated serum creatinine or cystatin C at screening. Note: If the initial test result is abnormal, it is permissible to re-test serum creatinine or cystatin C and randomize the subject if the re test result is normal.
    11. Positive for hepatitis B core antibody or hepatitis C antibody at screening.
    12. Prior or ongoing medical condition (eg, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings (eg, lower-limb injury that may affect 6MWT performance), ECG findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint of this study is slope of change in 6-minute walk distance (6MWD) over 72 weeks.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening, every 12 weeks for double-blind period, every 24 weeks for open-label period
    E.5.2Secondary end point(s)
    Double-Blind Treatment Period:
    • Change from baseline to Week 72 in 6MWD
    • Composite of average change in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs at Week 72
    • Change from baseline to Week 72 in time to run/walk 10 meters
    • Change from baseline to Week 72 in time to climb 4 stairs
    • Change from baseline to Week 72 in time to descend 4 stairs
    • Change from baseline to Week 72 in NSAA total score
    • Time to loss of ambulation over 72 weeks
    • Time to loss of stair-climbing over 72 weeks
    • Time to loss of stair-descending over 72 weeks
    • Risk of loss of NSAA items over 72 weeks
    • Ataluren safety profile characterized by type, frequency, severity, and relationship to study drug of any adverse events (AEs), or of abnormalities of laboratory tests, vital signs, physical examinations, or electrocardiograms (ECGs)

    Open-Label Treatment Period:
    • Slope of change in 6MWD over 144 weeks
    • Change from baseline to Week 144 in 6MWD
    • Composite of average change in times to run/walk 10 meters, climb 4 stairs, and descend 4 stairs at Week 144
    • Change from baseline to Week 144 in time to run/walk 10 meters
    • Change from baseline to Week 144 in time to climb 4 stairs
    • Change from baseline to Week 144 in time to descend 4 stairs
    • Change from baseline to Week 144 in NSAA total score
    • Time to loss of ambulation over 144 weeks
    • Time to loss of stair-climbing over 144 weeks
    • Time to loss of stair-descending over 144 weeks
    • Risk of loss of NSAA items over 144 weeks
    • Ataluren safety profile characterized by type, frequency, severity, and relationship to study drug of any AEs, or of abnormalities of laboratory tests, vital signs, physical examinations, or ECGs
    Plasma PK at pre-morning dose and 2 hours post-morning dose at Week 144
    E.5.2.1Timepoint(s) of evaluation of this end point
    Screening, every 12 weeks for double-blind period, every 24 weeks for open-label period
    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 No
    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) No
    E.7.1.1First administration to humans No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 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 concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Argentina
    Australia
    Brazil
    Bulgaria
    Canada
    China
    Hong Kong
    India
    Japan
    Korea, Republic of
    Malaysia
    Mexico
    Poland
    Russian Federation
    Taiwan
    Thailand
    Turkey
    United States
    Jordan
    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 years3
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days
    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 days25
    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: 340
    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: 292
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 48
    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 state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 19
    F.4.2.2In the whole clinical trial 340
    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)
    This study may be further extended by amendment until either ataluren becomes commercially available or the clinical development of ataluren in DMD is discontinued.
    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 Decision2017-12-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-01-16
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-07-21
    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