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    Summary
    EudraCT Number:2014-001638-27
    Sponsor's Protocol Code Number:FFP104-001
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-07-21
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2014-001638-27
    A.3Full title of the trial
    A Phase I/II, Open Label, Multicenter, Pilot Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacodynamics of FFP104 in Subjects Previously Diagnosed with Primary Biliary Cirrhosis (PBC)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A dose finding study of FFP104 for patients with Primary Biliary Cirrhosis (PBC)
    A.3.2Name or abbreviated title of the trial where available
    Pilot Study of FFP104 Dose Escalation in PBC Subjects
    A.4.1Sponsor's protocol code numberFFP104-001
    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 SponsorFast Forward Pharmaceuticals, B.V.
    B.1.3.4CountryNetherlands
    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 supportFast Forward Pharmaceuticals, B.V
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFast Forward Pharmaceuticals, B.V
    B.5.2Functional name of contact pointCEO
    B.5.3 Address:
    B.5.3.1Street AddressYalelaan 46
    B.5.3.2Town/ cityUtrecht
    B.5.3.3Post code3584 CM
    B.5.3.4CountryNetherlands
    B.5.4Telephone number31302534321
    B.5.6E-mailmark@ffpharma.com
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code FFP104
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    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) Yes
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary Biliary Cirrhosis (PBC)
    E.1.1.1Medical condition in easily understood language
    Primary biliary cirrhosis is inflammation and destruction of the bile ducts of the liver, which blocks the flow of bile. This obstruction damages liver cells and leads to scarring called cirrhosis.
    E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 18.1
    E.1.2Level LLT
    E.1.2Classification code 10036680
    E.1.2Term Primary biliary cirrhosis
    E.1.2System Organ Class 100000004871
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of FFP104 in PBC subjects following repeat doses of FFP104.
    E.2.2Secondary objectives of the trial
    • Proportion of subjects with a 10% decrease in ALP from baseline values at 12 weeks after Day 0
    • Proportion of subjects with a 25% and 40% decrease in ALP at Week 4, 8, 12 after Day 0
    • Proportion of subjects with ALP < 1.67 from upper level of normal, an ALP decrease >15% and bilirubin within normal levels at Week 4, 8, 12 and 24 after Day 0
    • Proportion of subjects with a biochemical response: ALP ≤ 3x ULN and AST ≤2 x ULN and bilirubin ≤1mg/dL at Week 4, 8, 12 and 24 after Day 0
    • To evaluate the individual and mean changes in serum ALP, ALT, AST, bilirubin, albumin, and GGT over time after Day 0 at Weeks 2, 4, 8, 12, 16 and 24
    • To evaluate changes from baseline with Mayo Risk Score at 4, 8, 12, and 24 weeks
    • To evaluate changes from baseline in PBC-40 Quality of Life (QoL) at 12 and 24 weeks
    • To evaluate changes from baseline in pruritus using the Visual Analog Scale at 12 and 24 weeks
    • To evaluate changes in the Pharmacokinetics over time from baseline



    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    * Male or Female Age between 18 and 75 years of age inclusive at the time of signing the informed consent.
    * Established diagnosis of PBC according to the EASL criteria (European Association for the Study of the Liver 2009): A diagnosis of PBC can be made with confidence in adult patients with otherwise unexplained elevation of ALP and presence of AMA (≥1:40), and/or AMA type M2. A liver biopsy is not essential for the diagnosis of PBC in these patients, but allows activity and stage of the disease to be assessed.
    * Having a screening ALP serum level between 1.67 and 5 x ULN inclusive.
    * Be on a stable dose of UDCA 12-20 mg/kg/day for at least 3 months prior to screening or intolerant of UDCA in the opinion of the investigator (no UDCA for at least 8 weeks prior to screening).
    E.4Principal exclusion criteria
    1) Laboratory Screening results:
    a) ALT >5x ULN
    b) AST >5x ULN
    c) Total bilirubin >2x ULN. Isolated bilirubin >2x ULN is acceptable if bilirubin is fractionated and direct bilirubin <35%
    d) Total WBC <3000 cells/mm3
    e) Absolute neutrophil count (ANC) <1500 cells/mm3
    f) Platelet count <100,000/mm3
    g) Prothrombin time (international normalized ratio (INR) >1.2
    2) BMI ≥35 or suspected to have relevant nonalcoholic fatty liver disease (NAFLD) as based on the judgment of the Investigator at Screening.
    3) Subject has a history of, or current viral hepatitis B or C (including hepatitis B surface antigen [HBsAg], hepatitis B core antibody and hepatitis C virus antibody [anti-HCV] positivity), or a positive HIV antibody screen at time of Screening.
    4) Recipients of liver or other organ transplantation or anticipated need for orthotropic organ transplantation in one year from Screening as determined by the Mayo Risk Score.
    5) Co-existing liver or biliary diseases, such as primary sclerosing cholangitis, choledocholithiasis, acute or chronic hepatitis, autoimmune hepatitis, alcoholic liver disease, nonalcoholic steatohepatitis (NASH), acute infection of bile duct system or gall bladder, history of gastrointestinal bleeding (secondary to portal hypertension), cholangiocarcinoma diagnosed or suspected liver cancers.
    6) Recurrent variceal hemorrhage, uncontrolled encephalopathy, Child-Pugh Class B/C, Esophageal Varices, or refractory ascites within the previous 6 months of Screening (defined as date informed consent signed).
    7) Have a family history (more than one first degree relative) of multiple thrombotic events or a personal history of any venous or arterial thrombotic event including deep vein thrombosis, stroke, myocardial infarction, pulmonary embolus, or peripheral arterial thromboembolic events.
    8) Prohibited medications 6 months prior to Screening: azathioprine, colchicine, cyclosporine, methotrexate, mycophenolate mofetil, pentoxifylline; fenofibrate or other fibrates; budesonide and other systemic corticosteroids; potentially hepatotoxic drugs (including α-methyl-dopa, sodium valproic acid, isoniazid, or nitrofurantoin).
    9) Prohibited medications 12 months prior to Screening: antibodies or immunotherapy directed against interleukins or other cytokines or chemokines.
    10) Subjects with recurrent bacterial infections (as judged by the Investigator) within 6 months prior to Screening of FFP104), active bacterial, fungal or mycobacterial infections observed during Screening, or any recent episode of infection requiring hospitalizations or treatment with antibiotics (within the 3 months prior to Screening).
    11) History of malignancy, with the exception of resected basal cell carcinoma, squamous cell carcinoma of the skin, or resected cervical atypia or carcinoma in situ.
    12) Immunization with a live vaccine within 4 weeks of Screening, with the exception of influenza vaccine and no planned immunizations within the period of the study.
    13) Known clinically significant cardiac disease (e.g., myocardial infarction or stroke, unstable angina, claudication, etc.), or evidence of a clinically significant electrocardiogram (ECG) abnormality within the previous 12 months prior to Screening.
    14) Subjects with evidence of other serious, significant, acute or chronic medical or psychiatric illness that, in the judgment of the Investigator, could compromise subject safety, limit the subject's ability to complete the study, and/or compromise the objectives of the study.
    15) History of recent (within 12 months of Screening) alcohol or drug abuse.
    16) Use of other immunosuppressive medications 4 weeks prior to Screening.
    17) Active tuberculosis (TB) in the past or currently, suspected TB which is presently receiving treatment or prophylactic therapy, has a positive TB test (as defined by local biological requirements), or any significant abnormality on chest X-ray within 3 months prior to Screening.
    18) Subjects who have planned surgery during the study period or have undergone major surgery within the 3 months prior to Screening.
    19) Known clinically significant allergy or known hypersensitivity to drugs that, in the opinion of the Investigator, may affect the patient's safety.
    20) Known sensitivity to any component of the study drug or previous sensitivity reaction or other clinically significant reaction to intravenous medications or biologic therapy.
    21) Participated in another clinical trial within the past 30 days prior to Screening, or is still within a washout period of a previous clinical trial, or has previously received FFP104 (PG102) or ch5D12 in this or any study.
    E.5 End points
    E.5.1Primary end point(s)
    This is an exploratory study to evaluate the safety and tolerability, pharmacokinetics and efficacy of FFP104. The general strategy of the analysis will be to examine the data summaries for any trends amongst the dose levels. No formal hypothesis testing will be conducted. Exploratory comparisons between dose cohorts can be performed, using non-parametrical statistical methods.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Not relevant
    E.5.2Secondary end point(s)
    Not available
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not relevant
    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 Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial4
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months26
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months26
    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: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Elderly >age 65
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 24
    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)
    None
    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 Decision2014-07-21
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-10-15
    P. End of Trial
    P.End of Trial StatusCompleted
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