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   43861   clinical trials with a EudraCT protocol, of which   7284   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:2016-000342-60
    Sponsor's Protocol Code Number:190280
    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:2019-02-06
    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 number2016-000342-60
    A.3Full title of the trial
    Fatigue in Sarcoidosis - A feasibility study investigating the treatment of fatigue in stable sarcoidosis patients using methylphenidate
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Fatigue and Sarcoidosis: Treatment with Methylphenidate
    A.3.2Name or abbreviated title of the trial where available
    Fatigue in Sarcoidosis: Treatment with Methylphenidate
    A.4.1Sponsor's protocol code number190280
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02643732
    A.5.3WHO Universal Trial Reference Number (UTRN)U1111-1180-0191
    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 SponsorNorfolk and Norwich University Hospital
    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 supportNational Institute of Health Research
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNorfolk and Norwich University Hospital NHS Foundation Trust
    B.5.2Functional name of contact pointLisa Chalkley
    B.5.3 Address:
    B.5.3.1Street AddressResearch Services Manager, R&D Department, Norfolk and Norwich University Hospital
    B.5.3.2Town/ cityNorwich
    B.5.3.3Post codeNR4 7UY
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number01603286611
    B.5.5Fax number01603289800
    B.5.6E-maillisa.chalkley@nnuh.nhs.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 Methylphenidate
    D.2.1.1.2Name of the Marketing Authorisation holderLABORATORIOS RUBIÓ S A, BARCELONA, E-08755, SPAIN
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameMethylphenidate 10mg tablets
    D.3.2Product code NA
    D.3.4Pharmaceutical form Tablet
    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 INNMethylphenidate
    D.3.9.1CAS number 113-45-1
    D.3.9.2Current sponsor codeNone
    D.3.9.3Other descriptive nameNone
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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
    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
    Sarcoidosis-associated fatigue (Patients with stable sarcoidosis and chronic fatigue)
    E.1.1.1Medical condition in easily understood language
    Sarcoidosis - a multi-system disease caused by deposits of immune system cells around the body, and which frequently leads to chronic disease despite appropriate treatment of the disease process.
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10039486
    E.1.2Term Sarcoidosis
    E.1.2System Organ Class 10021428 - Immune system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objectives for this trial is to determine whether it is feasible to perform a suitably large randomised controlled trial investigating whether methylphenidate is a clinically effective treatment for fatigue in sarcoidosis, and how best to design this trial to successfully answer this question.

    As a result, the main research questions are:
    (1) What is the recruitment rate of participants/how quickly can we recruit people into the trial? (How many studies might need to be involved in a future trial?)
    (2) How many patients with sarcoidosis are excluded from the study and for what reason? (as above)
    (3) How many people who are included in the study manage to complete the study? (How many participants extra do we need to ensure statistical power is maintained allowing for drop-outs)
    (4) Why are potential participants excluded from the study, and why do participants withdraw/drop-out of the study? (as above, as well as generalisability of the results – is the population i
    E.2.2Secondary objectives of the trial
    Although as a feasibility study there are no primary and secondary objectives (purely questions of feasibility), data collected will be analysed for changes in the following over the 24 weeks of the trial:
    (1) Fatigue (using a validated fatigue score, the fatigue assessment scale (FAS))
    (2) Disease-related health status (Kings' Sarcoidosis Questionnaire(KSQ))
    (3) Anxiety and Depression (Hospital Anxiety and Depression Scale (HADS))
    (4) Generic Quality of Life (EQ5D and SF36 questionnaires)
    (5) Physical capability (incremental shuttle walk test, a measured walking test performed in hospital to determine the distance able to be walked during a fixed time)
    (6) Lung function (breathing tests)
    (7) Physical activity over a 7 day period (measured using a wrist-worn accelerometer, a small device the size of a wrist watch that measures how active a person is)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. A proven diagnosis of sarcoidosis – this is defined as either a biopsy-proven disease (non-caseating granulomas from a tissue biopsy), or a diagnosis of sarcoidosis agreed by an interstitial lung disease multidisciplinary team (ILD MDT) meeting.
    2. Stable disease (treatment unchanged for 6 weeks, without anticipation of change in treatment during trial period)
    3. Able to give informed consent.
    4. Fatigue Assessment Sscale (FAS) score greater than 21 units (defined cut off for significant fatigue present)
    5. In patients on warfarin therapy – Willing to consent to increased frequency of monitoring
    E.4Principal exclusion criteria
    1. Evidence of co-existing obstructive sleep apnoea. Patients screened with a “STOP-Bang” questionnaire score of greater than 4 must undertake overnight oximetry; they are only excluded if this shows a desaturation index of more than 15 events per hour on overnight oximetry. Below this participants are eligible for inclusion.
    2. Documented history of significant cardiac disease (including cardiac sarcoid) OR associated disease which would increase risk of underlying coronary artery disease (cerebrovascular disease, previous stroke or peripheral vascular disease). Definitively treated cardiac disease e.g. previous myocardial infarction treated with stents or coronary artery bypass grafting with no ongoing symptoms is permitted.
    3. Hyperthyroidism evidenced by abnormal screening thyroid function tests (TSH outside normal range of 0.35 – 3.50 mU/L or T4 outside normal range of 8 – 21 pmol/L).
    4. History of seizures, excluding febrile convulsions whilst an infant.
    5. Abnormal electrocardiogram (ECG) with evidence of arrhythmia (except first degree heart block which has been stable for 3 months).
    6. Concomitant therapy with the following drugs:
    a. Tricyclic antidepressants
    b. Monoamine oxidase inhibitors
    c. Tramadol or buprenorphine
    d. Levodopa
    e. Haloperidol and atypical antipsychotics
    7. Glaucoma or raised intra-ocular pressure for any reason.
    8. Patients with established liver disease defined as Child-Pugh class B or C.
    9. Documented medical history of psychiatric disorders (excluding depression)
    10. History of drug-dependence or addiction at any time
    11. Female participant who is pregnant, lactating or planning pregnancy during the course of the trial
    12. Female patient of childbearing potential unable or unwilling to take two acceptable forms of contraception (see below)
    13. Receiving an investigational drug or biological agent within 6 weeks (or 5 times the half-life if this is longer) prior to study entry.

    Exclusion criteria on pregnancy
    Female patients of childbearing potential and unable or unwilling to take two of the following acceptable methods of contraception for the duration of their treatment will be excluded from participating in this trial:
    • Established use of oral, injected or implanted hormonal methods of contraception
    • Barrier methods of contraception (condom or occlusive cap with spermicide – please note, use of spermicide without a form of barrier contraception is not an acceptable form of contraception)
    • Absolute and continuous abstinence. Periodic abstinence (calendar, ovulation, symptothermal, post-ovulation) or withdrawal are not acceptable methods of contraception

    For the purposes of this trial the definition of a woman of childbearing potential is a sexually mature woman (i.e. has experienced menstruation) who has not been postmenopausal for 12 consecutive months (i.e. who has had menses at any time in the last 12 months without an alternative medical cause).
    E.5 End points
    E.5.1Primary end point(s)
    This is a feasibility study - the outcome measures regarding this are viewed with equal importance and are therefore not divided into primary and secondary objectives.

    The assessments of feasibility are:
    (1) What is the recruitment rate of participants? (How many studies might need to be involved in a future trial?)
    (2) How many patients with sarcoidosis are excluded from the study and for what reason? (as above)
    (3) What is the retention rate of participants within the study? (How many participants extra do we need to ensure statistical power is maintained allowing for drop-outs)
    (4) Why are potential participants excluded from the study, and why do participants withdraw/drop-out of the study? (as above, as well as generalisability of the results – is the population included and continuing the trial representative of sarcoidosis cohorts?)
    (5) How often are patients suffering side effects from the medication and how many participants discontinuing the medication during the study trial? (see assessment of safety below)
    (6) Is the effect of methylphenidate sustained over the whole 24 weeks of the trial? (does the future trial need to be as long? Is 8-12 weeks an acceptable study length?)
    (7) Do participants complete assessments during the trial? (Can we capture the data that we need? - and which of the assessments collect the data that we need)
    (8) Will participants wear accelerometer devices on their wrist (like a wrist watch) to measure their activity levels and can adequate data be reliably obtained from these devices? (will enough patients remember to use the accelerometers for the required minimum four days out of the seven day period?)
    (9) Acceptability of number of study visits and assessments
    (10) Acceptability of randomisation to active drug or placebo.
    (11) Acceptability of receiving a stimulant medication.
    (12) Estimate of size of effect of the medication (to plan future sample size)
    E.5.1.1Timepoint(s) of evaluation of this end point
    The evaluations of feasibility are made at 2,4,6,12,18 and 24 weeks.
    E.5.2Secondary end point(s)
    The secondary end-points of the trial are exploratory analyses of the questionnaires and exercise assessments performed during the trial. Specifically, these are:

    1. Difference in change in fatigue scores (measured by FAS and FACIT-F)
    2. Difference in change in disease-specific health-related quality of life questionnaire (Kings' Sarcoidosis Questionnaire - KSQ)
    3. Difference in change in depression and anxiety scores (Hospital Anxiety and Depression Scale - HADS)
    4. Difference in change in generic quality of life scores (EQ5D and SF-36)
    5. Use of health and social care resources (designed for this trial, elements taken from relevant tools within “Database of instruments for resource-use management” (DIRUM)).
    6. Change in physical capacity (measured using Modified Shuttle Walk Test)
    7. Change in level of physical activity (measured using wrist-worn accelerometer)
    8. Patient perceptions of the trial, including experiences of participating in the trial (focus groups)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Participants are followed up at 0,2,4,6,12,18 (postal questionnaires only) and 24 weeks.
    With regards to safety end-points, this data is recorded at each visit.
    For other end-points, data is collected at the following visits:
    FAS: 0,2,4,6,12,18, 24 and 30 weeks
    FACIT-F: 0,2,4,6,12,18,24 and 30 weeks
    HADS: 0,6,12,18,24,30 weeks
    KSQ: 0,6,12,18,24,30 weeks
    EQ5D: 0,6,12,18,24,30 weeks
    SF-36: 0,6,12,18,24,30 weeks
    Cost questionnaires: 0,6,12,18,24 weeks
    Spirometry: 0,12,24 weeks
    Modified shuttle walk test: 0,12,24 weeks
    Accelerometer measure of activity (device worn for 7 day period): -2,12,24 weeks
    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 No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    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
    Feasibility of future trial (efficacy data is also collected but trial not powered for this)
    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.8.3 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.8.4.1Number of sites anticipated in Member State concerned1
    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 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 years1
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days7
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months8
    E.8.9.2In all countries concerned by the trial days7
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    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: 0
    F.1.1.6Adolescents (12-17 years) No
    F.1.1.6.1Number of subjects for this age range: 0
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 5
    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 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 state30
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 30
    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)
    No treatment available after the trial - There is no ability to provide an ongoing prescription of the study medication at the end of the trial.
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2016-04-19
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-06-21
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-07-09
    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-Fri Apr 26 08:53:55 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA