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    Summary
    EudraCT Number:2017-001259-29
    Sponsor's Protocol Code Number:UoL001306
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-12-21
    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 ConcernedUK - MHRA
    A.2EudraCT number2017-001259-29
    A.3Full title of the trial
    A PHASE 1B/2A STUDY TO ASSESS THE TOLERABILITY AND ADVERSE EFFECT PROFILE OF CYSTEAMINE (CYSTAGON) IN ADULTS AND CHILDREN WITH HOMOZYGOUS ΔF508 CYSTIC FIBROSIS, IN PATIENTS TAKING EPIGALLOCATECHIN-3-GALLATE (EGCG, EPINERVE) FOOD SUPPLEMENTATION
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to establish the tolerabilty and adverse effects in people with a severe form of Cystic Fibrosi (delta F 508 homozygous) when using a combination of two products - cyteamine (a licensed medicine being used in a new disease) and EGCG (a food supplement).
    A.3.2Name or abbreviated title of the trial where available
    Delta Dose Study
    A.4.1Sponsor's protocol code numberUoL001306
    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 SponsorUniversity of LIverpool
    B.1.3.4CountryUnited Kingdom
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    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 organisationUniversity of Liverpool
    B.5.2Functional name of contact pointDr Dan Hawcutt
    B.5.3 Address:
    B.5.3.1Street AddressUniversity of Liverpool Alder Hey Children's Hospital, Eaton Road
    B.5.3.2Town/ cityLiverpool
    B.5.3.3Post codeL12 2AP
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number0151 228 4811 Ext. 2441
    B.5.6E-maildhawcutt@liverpool.ac.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 Yes
    D.2.1.1.1Trade name Cystagon
    D.2.1.1.2Name of the Marketing Authorisation holderOrphan Europe SARL Immeuble “Le Wilson” 70 avenue du Général de Gaulle F-92800 Puteaux France
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameCystagon
    D.3.4Pharmaceutical form Capsule, hard
    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 INNcysteamine bitartrate
    D.3.9.1CAS number 27761-19-9
    D.3.9.3Other descriptive name2-aminoethanethiol;2,3-dihydroxybutanedioic acid
    D.3.9.4EV Substance CodeAS1
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    DeltaF508 Homozygous Cystic Fibrosis
    E.1.1.1Medical condition in easily understood language
    Cystic Fibrosis (the most common genotype, which causes a particularly severe disease)
    E.1.1.2Therapeutic area Diseases [C] - Respiratory Tract Diseases [C08]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10011762
    E.1.2Term Cystic fibrosis
    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
    In two cohorts of patients (adult and paediatric) with a particular genetic cause of cystic fibrosis (deltaF 508, the most common and one of the most severe types), can patients tolerate an existing medicine (cysteamine) while taking a food supplement (EGCG). The primary outcome measure is discontinuation of the cysteamine. There are well known adverse effects for cysteamine. We also want to capture the suspected ADRs from this medication.
    E.2.2Secondary objectives of the trial
    For a subgroup of patients, we want to measure blood levels of cysteamine, to ensure we are dosing appropriately. In addition, in all the participants, we want to repeat the tests of effectiveness (sweat test), as they have only been done in a small population before, and not in children.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Adult Inclusion Criteria

    1. Patient managed by respiratory team at Leeds General Infirmary
    2. Patient has homozygous ΔF508 Cystic Fibrosis
    3. 18-30 years old at screening visit
    4. Written informed consent obtained from patient. This person must understand the contents of the consent, requirements of the study and have had an opportunity to review questions with a medically trained member of the site study team.
    5. Patient must have a good understanding of the English language, in which the consent forms are available, and understand the requirements for reporting of any AE to the Investigator.
    6. If female, be willing to have a pregnancy test and use one or more of the following methods of highly effective contraception:
    Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
    Oral
    Intravaginal
    Transdermal
    Progestogen-only hormonal contraception associated with inhibition of ovulation
    Oral
    Injectable
    Implantable
    Intrauterine device (IUD)
    Intrauterine hormone-releasing system ( IUS)
    Bilateral tubal occlusion
    Vasectomised partner
    Sexual abstinence
    7. Patient willing and able to comply with protocol including swallowing capsules and venepuncture/cannulation weekly for 6 weeks

    Paediatric Inclusion Criteria

    8. Patient managed by respiratory team at Alder Hey Children’s NHS Foundation Trust
    9. Patient has homozygous ΔF508 Cystic Fibrosis
    10. 5-17 years at screening visit
    11. Written informed consent from parent/legal guardian if patient <16 years or from patient if ≥16 years. This person must understand the contents of the consent, requirements of the study and have had an opportunity to review questions with a medically trained member of the site study team.
    12. Verbal or written age appropriate assent from the patient.
    13. Patient and parent/legal guardian must have a good understanding of the English language, in which the consent/assent forms are available, and understand the requirements for reporting of any AE to the Investigator.
    14. If female, and peri/post pubertal, must be willing to have a pregnancy test and use one or more of the following methods of highly effective contraception:
    Sexual abstinence
    Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation
    Oral
    Intravaginal
    Transdermal
    Progestogen-only hormonal contraception associated with inhibition of ovulation
    Oral
    Injectable
    Implantable
    Intrauterine device (IUD)
    Intrauterine hormone-releasing system ( IUS)
    Bilateral tubal occlusion
    Vasectomised partner
    15. Patient and parent/legal guardian willing and able to comply with protocol including swallowing capsules and venepuncture/cannulation weekly for 6 weeks
    E.4Principal exclusion criteria
    Adult Exclusion Criteria

    1. Use of EGCG, or drinking green tea within 2 weeks of the first dose of study and during study period
    2. Use of Ivacaftor, Lumacaftor or other mutations specific therapy
    3. Drinking black tea within 2 weeks of the first dose of study drugs, and during the study period
    4. Known hypersensitivity to Cysteamine, EGCG, or penicillamine
    5. Known hypersensitivity to the excipients of the study drugs (i.e. microcrystalline cellulose, pregelatinized starch, magnesium stearate/sodium lauryl sulfate, colloidal silicon dioxide, croscarmellose sodium gelatin, titanium dioxide, black ink on hard capsules containing E172).
    6. Recent infective exacerbation of CF. Patient must not have had oral antibiotics within 4 weeks of screening or IV antibiotics within 6 weeks of screening
    7. Any clinically significant acute illness within 4 weeks of the start of dose administration.
    8. Any clinically significant medical condition, other than CF that in the opinion of the Investigator may increase the risk of participating in the study or interfere with the interpretation of safety or efficacy evaluations (e.g., severe reflux, psychiatric condition or behavioural disorder).
    9. Patient on regular itraconazole
    10. Liver function tests deemed too abnormal by a clinician experienced in the management of cystic fibrosis.
    11. Renal function abnormal for age.
    12. Significantly low red cell folate level
    13. Pregnant or breastfeeding
    14. Written consent not obtained.
    15. The patient is deemed unsuitable at the discretion of the investigator.
    16. Patients unable to swallow tablets/capsule by mouth

    Paediatric Exclusion Criteria

    1. Use of EGCG, or drinking green tea within 2 weeks of the first dose of study and during study period
    2. Use of Ivacaftor, Lumacaftor or other mutations specific therapy
    3. Drinking black tea within 2 weeks of the first dose of study drugs, and during the study period
    4. Known hypersensitivity to cysteamine or EGCG
    5. Known hypersensitivity to the excipients of the study drugs (i.e. microcrystalline cellulose, pregelatinized starch, magnesium stearate/sodium lauryl sulfate, colloidal silicon dioxide, croscarmellose sodium gelatin, titanium dioxide, black ink on hard capsules containing E172).
    6. Recent infective exacerbation of CF. Patient must not have had oral antibiotics within 4 weeks of screening or IV antibiotics within 6 weeks of screening
    7. Any clinically significant acute illness within 4 weeks of the start of dose administration.
    8. Any clinically significant medical condition, other than CF that in the opinion of the Investigator may increase the risk of participating in the study or interfere with the interpretation of safety or efficacy evaluations (e.g., severe reflux, psychiatric condition or behavioural disorder).
    9. Patient on regular itraconazole
    10. Liver function tests deemed too abnormal by a clinician experienced in the management of cystic fibrosis.
    11. Renal function abnormal for age.
    12. Significantly low red cell folate level
    13. Pregnant or breastfeeding
    14. Written consent not obtained.
    15. Participant who understands the study (judged on case by case basis, from approximately age 7) declines assent.
    16. The patient is deemed unsuitable at the discretion of the investigator.
    17. Patients unable to swallow tablets/capsule by mouth
    E.5 End points
    E.5.1Primary end point(s)
    1. Discontinuation of the study drug
    2. Adverse effects of cysteamine in homozygous ∆508 cystic fibrosis adult and paediatric patients
    E.5.1.1Timepoint(s) of evaluation of this end point
    The study will last for 6 weeks. This comprises 4 weeks where the cysteamine dose is increased (as per usual dosing in cystinosis) while a single dose of EGCG is administered. On week 5, the participants remain on the dose of cysteamine, but a single dose increase in EGCG is undertaken. In the final week, no medications are used.

    Patients will have weekly review to ensure they are able to take the medications, and to assess for adverse effects of the medications. Safety bloods will also be undertaken weekly.
    E.5.2Secondary end point(s)
    1. Plasma concentration of cysteamine and EGCG at set time points in ∆F508 cystic fibrosis patients.
    2. Change in sweat test chloride result with each dose escalation of cysteamine
    3. Change in sweat test chloride result with cysteamine used in combination with multiple daily dose EGCG
    E.5.2.1Timepoint(s) of evaluation of this end point
    A subset of patients (10 per cohort) will have additional blood taken for PK analysis at their weekly reviews.

    All participants will have a weekly sweat test, as a means to replicate the reduction in sweat chloride seen in adult patients and establish if this occurs in paediatric patients.
    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 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 Yes
    E.6.13.1Other scope of the trial description
    Tolerability
    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 No
    E.7.1.3.1Other trial type description
    Tolerability and Adverse effect
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 concerned2
    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 No
    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 medical review and safety assessment on D42
    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 months8
    E.8.9.1In the Member State concerned days3
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days3
    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: 20
    F.1.1.1In Utero No
    F.1.1.1.1Number of subjects for this age range: 0
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.2.1Number of subjects for this age range: 0
    F.1.1.3Newborns (0-27 days) No
    F.1.1.3.1Number of subjects for this age range: 0
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.4.1Number of subjects for this age range: 0
    F.1.1.5Children (2-11years) No
    F.1.1.5.1Number of subjects for this age range: 10
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) No
    F.1.2.1Number of subjects for this age range: 20
    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 state44
    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)
    We will not be able to continue to supply treatment once the study has finished. Participants will return to standard of care in their respective centre.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    G.4.1Name of Organisation Alder Hey Children's Hospital
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2019-03-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-28
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2020-02-18
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