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    Summary
    EudraCT Number:2014-001101-40
    Sponsor's Protocol Code Number:13/0417
    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:2015-01-14
    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 number2014-001101-40
    A.3Full title of the trial
    Sclero XIII: A phase II ,double-blind, randomized, placebo-controlled study to investigate pharmacokinetics (PK), safety and efficacy of intravenous factor XIII treatment in patients with systemic sclerosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Investigation of safety and therapeutic effect of Fibrogammin® in Scleroderma
    A.3.2Name or abbreviated title of the trial where available
    Sclero XIII
    A.4.1Sponsor's protocol code number13/0417
    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 College London (UCL)
    B.1.3.4Country
    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 supportCSL Behring GmbH
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversity College London
    B.5.2Functional name of contact pointProf Christopher Denton
    B.5.3 Address:
    B.5.3.1Street AddressClinical Trials,Rhuematology Department
    B.5.3.2Town/ cityHampstead, London, Royal Free Hospital
    B.5.3.3Post codeNW3 2QG
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number02073177544
    B.5.6E-mailc.denton@ucl.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 Fibrogammin® 1250
    D.2.1.1.2Name of the Marketing Authorisation holderCSL Behring GmbH
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 nameFibrogammin® 1250
    D.3.4Pharmaceutical form Powder and solvent for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNblood coagulation factor XIII
    D.3.9.2Current sponsor code13/0417
    D.3.9.4EV Substance CodeAS2
    D.3.10 Strength
    D.3.10.1Concentration unit IU/ml international unit(s)/millilitre
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number62.5
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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 Yes
    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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Systemic Sclerosis
    E.1.1.1Medical condition in easily understood language
    Scleroderma
    E.1.1.2Therapeutic area Diseases [C] - Skin and Connective Tissue Diseases [C17]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10010759
    E.1.2Term Connective tissue disorder NOS
    E.1.2System Organ Class 100000004859
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.1
    E.1.2Level LLT
    E.1.2Classification code 10018124
    E.1.2Term Generalized scleroderma
    E.1.2System Organ Class 100000004859
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    1.To identify any effects of factor XIII treatment on clinical manifestations of Sleroderma (SSc)
    2.To investigate factor XIII safety
    3.To measure individual factor XIII levels in patients with SSc
    4.To measure the effects of factor XIII treatment on factor XIII PK parameters.

    E.2.2Secondary objectives of the trial
    1.To explore effects of factor XIII on thrombospondin expression
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.≥ 18 years
    2.Male and female adults
    3.Subjects with a diagnosis of SSc according to the 2013 ACR EULAR classification criteria.They will be classified according to LeRoy criteria as limited or diffuse subset.
    4.Females of childbearing potential must be willing to use a reliable form of medically acceptable contraception and have a negative pregnancy test
    5.Subjects will have serological status for hepatitis A and B assessed at screening.
    6.Patients who have given their free and informed consent

    E.4Principal exclusion criteria
    1.Females of childbearing potential and males must be willing to use an effective method of contraception (hormonal or barrier method of birth control; abstinence) from the time consent is signed until 6 weeks after treatment discontinuation.
    2.Females of childbearing potential must have a negative pregnancy test within 7 days prior to being registered for trial treatment. NOTE: Subjects are considered not of child bearing potential if they are surgically sterile (i.e. they have undergone a hysterectomy, bilateral tubal ligation, or bilateral oophorectomy) or they are postmenopausal.
    3.Females must not be breastfeeding.
    4.Allergies to excipients of IMP and placebo
    5.Uncontrolled systemic hypertension as evidenced by sitting systolic blood pressure > 160 mmHg or sitting diastolic blood pressure > 100 mmHg.
    6.Portal hypertension or chronic liver disease defined as mild to severe hepatic impairment (Child-Pugh Class A-C). Subjects positive for Hepatitis C with evidence of active viral replication on sensitive PCR testing are also excluded.
    7.Hepatic dysfunction, defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT)  3 times the upper limit of the normal range (× ULN) at the Screening Visit.
    8.Chronic renal insufficiency as defined by a serum creatinine > 2.5 mg/dL or requires dialysis.
    9.Hemoglobin concentration < 10 g/dL at the Screening Visit.
    10.History of left-sided heart disease and/or clinically significant cardiac disease, including but not limited to any of the following:
    11.Aortic or mitral valve disease (stenosis or regurgitation) defined as more
    than minimum aortic insufficiency and more than moderate mitral
    regurgitation; (stenosis or regurgitation>grade 1)Pericardial constriction; Restrictive or congestive cardiomyopathy; Left ventricular ejection fraction  40 % by multiple gated acquisition scan
    12.(MUGA), angiography, or echocardiography; Left ventricular shortening fraction < 22 % by echocardiography; or Symptomatic coronary disease with demonstrable ischemia.
    13.History of malignancies within 5 years of Screening Visit with the exception of localized skin or cervical carcinomas.
    14.Psychiatric, addictive, or other disorder that compromises the ability to give informed consent for participating in this study. This includes subjects with a recent history of abusing alcohol or illicit drugs
    15. Ongoing treatment with hyperbaric oxygen
    16.Exclude PAH patients
    17.Patients who have received IV Iloprost within the last 2 months (1 to 3 months, to discuss)
    18.Patients who have been treated with sypathectomy or toxin botulinum A within the last 3 months
    19.Patients with a recent thrombosis, stroke, pulmonary embolism or myocardial infarction in the last 6 months
    20.Patients with a diagnosis of diabetes mellitus requiring dietary restriction of cardbohydrate.
    21.Patients having low sodium diet on medical advice.
    22.Patients participating in another clinical trial involving an investigational medicinal product.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoints:
    •Skin involvement measured with modified Rodnan skin score
    •Raynaud condition score

    Treatment Failure:
    •Rapid progression of skin involvement defined as more than 8 skin score units and 40% increase.
    •Rapid progression of lung involvement defined as 15% fall in FVC or 20% call in DLco
    •DU evolution to gangrene
    •Autoamputation or surgery for digital ulcer management (amputation)
    •hospitalization

    Safety endpoints

    •Physical examination (including height, weight, BMI, digital ulcer characterization)
    •Adverse events
    •Serious adverse events
    •ECG
    •Vital signs
    •Clinical laboratory parameters
    •Pregnancy
    •Adverse events of special interest: thromboembolic events

    E.5.1.1Timepoint(s) of evaluation of this end point
    This is an early phase exploratory study and so all analysis will be descriptive and examine summary statistics and confidence intervals. Formal significance testing will not be undertaken and this will not be hypothesis testing.
    E.5.2Secondary end point(s)
    Secondary Exploratory Analyses:
    •Pulmonary function
    •Hand function measured with Cochin hand function
    •Quality of life measured with SF36 quality of life questionnaire
    •Prevention of new DU: Number of new DU developed during a 24-week period of treatment
    •Healing of DU: Complete healing of DUs present at baseline; each DU is considered as an entity DU Pain assessment at 4, 8, 12, 16, 24 weeks of treatment: Pain will be assessed by analogue scale for pain (VAS) and Raynaud’s severity (Raynaud’s condition score)
    DU worsening, defined as:
    oOvernight Hospitalization for digital ulcers
    oAddition surgical treatment for digital ulcer in outpatient clinic
    oDigital ulcer infection
    oGangrene and/or amputation
    oNeed of local sympathectomy
    oNeed of toxin botulinum A)
    oNeed of oral or parenteral antibiotic
    oNeed of IV Iloprost : this is considered treatment failure
    E.5.2.1Timepoint(s) of evaluation of this end point
    This is an early phase exploratory study and so all analysis will be descriptive and examine summary statistics and confidence intervals. Formal significance testing will not be undertaken and this will not be hypothesis testing.
    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 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 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) 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 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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    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: 0
    F.1.3Elderly (>=65 years) Yes
    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 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 state26
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 26
    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)
    Factor XIII will not be available for the patients after the study has concluded. Patient will be aware of this at time of consent as it is detailed in the patient information sheet.
    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 Decision2015-02-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-02-18
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2018-09-06
    As of 1.2.2020, the UK is no longer an EU Member State. However, EU law still applies to the UK during the transition period
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