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    Summary
    EudraCT Number:2007-000724-40
    Sponsor's Protocol Code Number:PTC124-GD-006-CF
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2007-04-27
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2007-000724-40
    A.3Full title of the trial
    A Phase 2 Study of PTC124 as an Oral Treatment for Nonsense-Mutation-Mediated Cystic Fibrosis
    A.4.1Sponsor's protocol code numberPTC124-GD-006-CF
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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/3/05/277
    D.3 Description of the IMP
    D.3.2Product code PTC124
    D.3.4Pharmaceutical form Oral powder
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codePTC124
    D.3.10 Strength
    D.3.10.1Concentration unit % (W/W) percent weight/weight
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 Yes
    D.3.11.13.1Other medicinal product typePTC124 is a Biopharmaceutical Classification System Case 2 compound [FDA 1997] PTC124 is a 1,2,4-oxadiazole linked to 2 ring structures: fluorobenzene and benzoic acid C15H9FN2O3.
    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
    Cystic Fibrosis
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 9.1
    E.1.2Level LLT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    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 whether PTC124 safely provides pharmacological activity as evaluated by TEPD assessment of chloride conductance
    E.2.2Secondary objectives of the trial
    • To assess PTC124 effects on additional TEPD measures of ion channel activity
    • To evaluate PTC124 effects on CFTR protein and CFTR mRNA in nasal mucosa
    • To assess inflammatory markers in sputum and serum
    • To characterize changes in pulmonary function
    • To evaluate changes in patient weight
    • To determine compliance with PTC124 therapy
    • To further characterize the safety profile of PTC124 in patients with CF
    • To further evaluate the PK profile of PTC124 in patients with CF
    • To assess changes in disease-related symptoms over the course of treatment
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Diagnosis of CF based on documented evidence of a conclusively abnormal sweat test (sweat chloride >35 mEq/liter by pilocarpine iontophoresis). Note: A patient does not need to have a repeat sweat test solely for enrollment into this study if documentation of an abnormal sweat test is already available.
    2. Abnormal nasal epithelial TEPD total chloride conductance (a less electrically negative value than 5 mV for Δchloride-free+isoproterenol). Note: Even if a patient has past documentation of an abnormal TEPD, the TEPD must be repeated as part of the screening procedures and must be abnormal (taken as a mean of both nostrils if values for both nostrils are available). A patient requiring inhaled tobramycin (eg, TOBI®) should have the baseline TEPD assessed while receiving a course of inhaled tobramycin.
    3. Presence of a mutation in both alleles of the cftr gene with a premature stop (nonsense) mutation in at least one of the alleles of the gene (ie, patient should be either heterozygous or homozygous for stop mutation).
    4. Documentation that a blood sample has been drawn for reconfirmation of the presence of a nonsense mutation in the cftr gene. Note: A patient who has documentation of a nonsense mutation need not wait for confirmatory results to start study therapy as long as a blood sample has been drawn for reconfirmation.
    5. Age >/=6 and </=18 yrs of age.
    6. Body weight 25 kg.
    7. FEV1 40% of predicted for age, gender, and height (Knudson standards) [Knudson 1983].
    8. Oxygen saturation (as measured by pulse oximetry) 92% on room air.
    9. Willingness of male and female patients, if not surgically sterile, to abstain from sexual intercourse or employ a barrier or medical method of contraception during the study drug administration and follow-up periods.
    10. Negative pregnancy test (for females of childbearing potential).
    11. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures (including TEPD measurements, clinical laboratory tests, pulmonary function tests, and PK sampling), and study restrictions.
    12. Ability to provide written informed consent and/or assent.
    13. Evidence of signed and dated informed consent document (by the patient or a legal guardian) indicating that the patient and/or the legal guardian has been informed of all pertinent aspects of the trial.
    E.4Principal exclusion criteria
    1. Prior exposure to PTC124.
    2. Prior or ongoing medical condition (eg, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect the safety of the patient, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
    3. Ongoing acute illness including acute upper or lower respiratory infections within 2 weeks before start of study treatment.
    4. History of major complications of lung disease (including recent massive hemoptysis or pneumothorax) within 2 months prior to start of study treatment.
    5. Abnormalities on screening chest x-ray suggesting clinically significant active pulmonary disease other than CF, or new, significant abnormalities such as atelectasis or pleural effusion which may be indicative of clinically significant active pulmonary involvement secondary to CF.
    6. Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test.
    7. Hemoglobin <10 g/dL.
    8. Serum albumin <2.5 g/dL.
    9. Abnormal liver function (serum total bilirubin > the upper limit of normal, or serum ALT, AST, or GGT >2.0 times the upper limit of normal). Note: A patient who are taking ursodiol tablets (eg, URSO®) may be enrolled as long as these liver function test values are within the designated limits.
    10. Abnormal renal function (serum creatinine >1.5 times upper limit of normal).
    11. Pregnancy or breast-feeding.
    12. History of solid organ or hematological transplantation.
    13. Exposure to another investigational drug within 14 days prior to start of study treatment.
    14. Ongoing participation in any other therapeutic clinical trial.
    15. Ongoing use of thiazolidinedione peroxisome proliferator-activated receptor gamma
    (PPAR γ) agonists, eg, rosiglitazone (Avandia® or equivalent) or pioglitazone (Actos® or equivalent).
    16. Requirement for treatment with intranasal or inhaled medications (including use of cromolyn, ipratropium bromide, phenylephrine, or oxymetazoline) within 7 days prior to start of study treatment. Note: A patient may receive inhaled β-agonist therapy as clinically necessary for acute pulmonary exacerbations during the study, although, if medically appropriate, an attempt should be made to avoid such use.
    17. Requirement for treatment with systemic or inhaled corticosteroids within 7 days prior to start of study treatment. Note: A patient may receive inhaled corticosteroids as clinically necessary for acute pulmonary exacerbations during the study, although, if medically appropriate, an attempt should be made to avoid their use.
    18. Use of or requirement for inhaled gentamicin or amikacin within 14 days prior to start of study treatment or during study treatment. Note: A patient may receive inhaled tobramycin (eg, TOBI®) before and during the study but use of inhaled tobramycin should not be changed or initiated during the study.
    19. Requirement for systemic aminoglycoside antibiotics within 14 days prior to start of study treatment. Note: A patient may receive systemic antibiotics as clinically necessary for acute pulmonary exacerbations during the study, although, if medically appropriate, an attempt should be made to avoid systemic aminoglycoside antibiotics.
    Note: A patient may receive inhaled dornase alpha (Pulmozyme®) or bronchodilators before and during the study.
    E.5 End points
    E.5.1Primary end point(s)
    • Changes in total nasal chloride conductance as assessed by TEPD, with assessment of mean changes in TEPD by dose level and the proportion of patients with a chloride conductance response by dose level; a chloride conductance response is defined as at least a -5 mV (or more negative) improvement in TEPD following sequential perfusion with a chloride-free amiloride, 100 M, solution and the same solution with isoproterenol, 10 M, for 3 minutes each (Δchloride-free+isoproterenol)
    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 Yes
    E.6.10Pharmacogenetic Yes
    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 Information not present in EudraCT
    E.7.1.2Bioequivalence study Information not present in EudraCT
    E.7.1.3Other Information not present in EudraCT
    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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over Yes
    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 Yes
    E.8.2.3.1Comparator description
    PTC124 in another concentration
    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 No
    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 Information not present in EudraCT
    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
    The last vist of the last subject undergoing the trial.
    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 months0
    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 months0
    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.1.1.1In 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.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    F.1.3Elderly (>=65 years) No
    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 Information not present in EudraCT
    F.4 Planned number of subjects to be included
    F.4.1In the member state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 30
    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 Decision2007-05-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2007-09-27
    P. End of Trial
    P.End of Trial StatusCompleted
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