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    Summary
    EudraCT Number:2015-004986-99
    Sponsor's Protocol Code Number:NBTCS02
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    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 ConcernedItaly - Italian Medicines Agency
    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.
    Studio randomizzato, in doppio cieco, a gruppi paralleli, controllato con placebo per esaminare il regime posologico ottimale, l’efficacia e la sicurezza dell’aggiunta di cisteamina orale in pazienti adulti con fibrosi cistica (FC) in trattamento per un aggravamento della pneumopatia associata alla FC.
    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.
    Uno studio per verificare se la cisteamina è sicura, efficace, e quanta e quanto spesso debba essere somministrata a pazienti adulti con Fibrosi Cistica i quali siano trattati per un peggioramento della malattia polmonare associata.
    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
    Aggravamento della Fibrosi Cistica
    E.1.1.1Medical condition in easily understood language
    worsening of Cystic Fibrosis
    Aggravamento della Fibrosi Cistica
    E.1.1.2Therapeutic area Diseases [C] - Bacterial Infections and Mycoses [C01]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    E.1.2Level PT
    E.1.2Classification code 10070608
    E.1.2Term Infective pulmonary exacerbation of cystic fibrosis
    E.1.2System Organ Class 10021881 - Infections and infestations
    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
    • Determinare la dose e la frequenza ottimali di cisteamina nelle esacerbazioni della malattia respiratoria associate alla Fibrosi Cistica
    • Determinare il questionario migliore per la valutazione del beneficio clinico dato dall’uso di cisteamina nelle esacerbazioni della malattia respiratorio associate alla Fibrosi Cistica
    • Determinare gli effetti del trattamento con cisteamina sui parametric di sicurezza
    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
    • Determinare gli effetti del trattamento con cisteamina sulla esacerbazione della malattia respiratoria associate alla Fibrosi Cistica per ognuno dei seguenti parametri:
    o Livelli in espettorato di IL8 e di elastase in neutrofili
    o FEV1 (Forced Expiratory Volume1) nel primo secondo
    o Peso
    o Proteina C reattiva
    o Conta leucocitaria
    o Valutazione dei livelli di cisteamina nel sangue e nell’espettorato
    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
    1) Pneumopatia associata a FC con anamnesi positiva documentata di infezione cronica da organismi gram-negativi.
    2) Paziente seguito stabilmente dal gruppo multidisciplinare (MDT, Multi Disciplinary Team) per la FC dello sperimentatore principale.
    3) Età ≥ 18 anni.
    4) Peso > 40 kg.
    5) FEV1 > 30% del valore atteso nei 6 mesi precedenti all’aggravamento considerato dallo studio.
    6) Alla visita basale: presenza di un nuovo aggravamento della pneumopatia associata a FC (sulla base della valutazione da parte dello sperimentatore di ≥ 4 sintomi presenti nei criteri di Fuchs) che necessiti di un trattamento comprensivo di un antibiotico aminoglicosidico.
    E.4Principal exclusion criteria
    1. Hypersensitive to cysteamine or to any of the excipients
    2. Hypersensitive to penicillamine
    3. Transplant recipient
    1) Ipersensibilità alla cisteamina o a uno qualunque degli eccipienti.
    2) Ipersensibilità alla penicillamina.
    3) Soggetto trapiantato.
    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
    Variazione rispetto al basale (Giorno 0) nei questionari relativi alla salute del paziente/PROM (CFRSD-CRISS, questionario per il punteggio dei sintomi di Jarad e Sequeiros).
    E.5.1.1Timepoint(s) of evaluation of this end point
    See section E.5.1. Primary End Points
    Giorno 14
    E.5.2Secondary end point(s)
    1. Assessment of blood and sputum cysteamine levels at Day 14
    2. 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
    3 .Change from baseline in sputum IL8 and neutrophil elastase levels at Day 7, Day 14 and Day 21 following a CF exacerbation
    4. Change from baseline to Day 7, Day 14 and Day 21 in FEV1, weight, CRP, blood leucocyte count and CFQ-R
    5. Patient Global Assessment of Exacerbation outcome
    1. Valutazione dei livelli di cisteamina nel sangue e nell’espettorato al Giorno 14.
    2. Variazione rispetto al basale (Giorno 0) della carica batterica nell’espettorato in termini di (a) CFU totali per ml e per mg, e (b) CFU di gram-negativi per ml e per mg al Giorno 7, al Giorno 14 e al Giorno 21 dopo un aggravamento della FC.
    3. Variazione rispetto al basale (Giorno 0) dei livelli di IL8 ed elastasi dei neutrofili nell’espettorato al Giorno 7, al Giorno 14 e al Giorno 21 dopo un aggravamento della FC.
    4. Variazione rispetto al basale (Giorno 0) dei seguenti parametri al Giorno 7, al Giorno 14 e al Giorno 21:FEV1, peso, PCR, conta leucocitaria nel sangue, CFQ-R.
    5. Esito della valutazione globale dell’aggravamento da parte del paziente.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. Day 14
    2. Day 7, 14, 21
    3. Day 7, 14, 21
    4. Day 7, 14, 21
    5. EoS
    1. Giorno 14
    2. Giorno 7, 14, 21
    3. Giorno 7, 14, 21
    4. Giorno 7, 14, 21
    5. End of Study
    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
    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 state12
    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.
    È possibile l’offerta ai pazienti della partecipazione in un programma di uso compassionevole
    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 Decision2016-11-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-01-11
    P. End of Trial
    P.End of Trial StatusOngoing
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    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
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