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    Summary
    EudraCT Number:2015-004986-99
    Sponsor's Protocol Code Number:NBTCS02
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2016-09-06
    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 ConcernedUK - MHRA
    A.2EudraCT number2015-004986-99
    A.3Full title of the trial
    A Randomized, Double-Blind, Parallel Group, Placebo-Controlled Study Investigating the Optimal Dose Regimen, Efficacy, and Safety of Adding Oral Cysteamine in Adult Patients with Cystic Fibrosis (CF) Being Treated for an Exacerbation of CF-associated Lung Disease.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to identify whether it is safe, if it works, and how much and how often cysteamine should be given to adult patients with Cystic Fibrosis (CF) who are being treated for a worsening of CF associated lung disease.
    A.4.1Sponsor's protocol code numberNBTCS02
    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 SponsorNovaBiotics, Ltd
    B.1.3.4CountryUnited Kingdom
    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 supportNovaBiotics, Ltd.
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovaBiotics, Ltd.
    B.5.2Functional name of contact pointDeborah O'Neil
    B.5.3 Address:
    B.5.3.1Street AddressCruickshank Building
    B.5.3.2Town/ cityCraibstone, Aberdeen
    B.5.3.3Post codeAB21 9TR
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number441224711377
    B.5.6E-mailDeborah@novabiotics.co.uk
    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 numberEMA/OD/46/11 EU/3/11/928
    D.3 Description of the IMP
    D.3.1Product namecysteamine bitartrate
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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 placeboCapsule, hard
    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
    exacerbation of Cystic Fibrosis
    E.1.1.1Medical condition in easily understood language
    worsening of Cystic Fibrosis
    E.1.1.2Therapeutic area Diseases [C] - Bacterial Infections and Mycoses [C01]
    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 determine the optimal dose and frequency of cysteamine in exacerbations of CF-associated lung disease
    - to determine the best questionnaire for evaluation of clinical benefit arising from use of cysteamine in exacerbations of CF-associated lung disease
    - to determine the effects of treatment with cysteamine on safety parameters
    E.2.2Secondary objectives of the trial
    to determine the effects of treatment with cysteamine on an exacerbation of CF-associated lung disease for each of the following:
    - sputum IL8 and neutrophil elastase levels
    - forced expiratory volume in the first second (FEV1)
    - weight
    - C-reactive protein (CRP)
    - blood leukocyte count
    - assessment of blood and sputum cysteamine levels
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. CF-associated lung disease with documented history of chronic infection with Gram-negative organism(s)
    2. Established patient of the Principal Investigator's CF Multi-Disciplinary team
    3. Age equal or greater than 18 years
    4. Weight equal or more than 40 Kg
    5. FEV1 more than 30% of predicted within the 6 months prior to study exacerbation
    6. At baseline visit: experiencing a new exacerbation of CF associated lung disease requiring treatment that includes an aminoglycoside antibiotic
    7) Females of childbearing potential will be included if they are
    either sexually inactive (sexually abstinent for 14 days prior to the
    first study drug dose continuing through 28 days after the last
    study drug dose, or using one of the following highly effective
    contraceptive (i.e. results in <1% failure rate when used
    consistently and correctly) methods in this trial:
    a. intrauterine device (IUD);
    b. surgical sterilization of the partner (vasectomy for 6 months
    minimum);
    c. combined (estrogen or progestogen containing) hormonal
    contraception associated with the inhibition of ovulation
    (either oral, intravaginal, or transdermal);
    d. progestogen only hormonal contraception associated with the
    inhibition of ovulation (either oral, injectable, or
    implantable);
    e. intrauterine hormone releasing system (IUS);
    f. bilateral tubal occlusion.
    Females of childbearing potential agree to remain sexually inactive or to keep the same birth control method for at least 28 days following
    the last dose.
    9) A female of non-childbearing potential must have undergone one
    of the following sterilization procedures at least 6 months prior to
    the first study drug dose:
    a. hysteroscopic sterilization;
    b. bilateral tubal ligation or bilateral salpingectomy;
    c. hysterectomy;
    d. bilateral oophorectomy;
    or be postmenopausal with amenorrhea for at least 1 year prior
    to the first study drug dose and follicle stimulating hormone
    (FSH) serum levels consistent with postmenopausal status.
    10) A non-vasectomized male subject agrees to use a condom with
    spermicide or abstain from sexual intercourse during the study
    until 90 days beyond the last dose of study medication and the
    female partner agrees to comply with inclusion 7 or 9. For a
    vasectomized male who has had his vasectomy 6 months or more
    prior to study start, it is required that they use a condom during
    sexual intercourse. A male who has been vasectomized less than 6
    months prior to study start must follow the same restrictions as a
    non-vasectomized male.
    11) If male, agrees not to donate sperm from the first study drug dose until 90 days after dosing.
    12) Willing and able to comply with all protocol requirements and
    procedures, including induction of sputum, if necessary
    13) Willing and able to provide signed and dated informed consent
    E.4Principal exclusion criteria
    1. Hypersensitive to cysteamine or to any of the excipients
    2. Hypersensitive to penicillamine
    3. Transplant recipient
    E.5 End points
    E.5.1Primary end point(s)
    - Change from baseline in patient health-related questionnaires (CFRSD-CRISS, Jarad and Sequeiros Smptom Score Questionnaire
    - Change from baseline in sputum bacterial load of (a) total CFU per ml and per mg and (b) gram negative CFU per ml and per mg at Day 7, Day 14 and Day 21 following a CF exacerbation
    - Change from baseline in sputum IL8 and neutrophil elastase levels at Day 7, Day 14 and Day 21 following a CF exacerbation
    - Change from baseline to Day 7, Day 14 and Day 21 in FEV1, weight, CRP, blood leucocyte count and CFQ-R
    - Assessment of blood and sputum cysteamine levels at Day 14
    - Patient Global Assessment of Exacerbation outcome
    E.5.1.1Timepoint(s) of evaluation of this end point
    See section E.5.1. Primary End Points
    E.5.2Secondary end point(s)
    See section E.5.1 Primary end points
    E.5.2.1Timepoint(s) of evaluation of this end point
    See section E.5.1 Primary End Points
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) 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 No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 trial6
    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 concerned20
    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 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
    Italy
    Netherlands
    United Kingdom
    United States
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months6
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 120
    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 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 state60
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 120
    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)
    Patient may be offered the oppertunity to participate in NovaBiotics' compassionate use program.
    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 Decision2016-10-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-09-22
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2019-01-11
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