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    EudraCT Number:2014-000350-11
    Sponsor's Protocol Code Number:2032/MUL
    National Competent Authority:Germany - BfArM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-03-10
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedGermany - BfArM
    A.2EudraCT number2014-000350-11
    A.3Full title of the trial
    An open label, randomized study to investigate the safety of weekly pentosan polysulfate injections in adult patients with Mucopolysaccharidosis Type I receiving enzyme replacement therapy.
    Eine offene, randomisierte Studie zur Untersuchung der Sicherheit von wöchentlichen Pentosanpolysulfatinjektionen bei erwachsenen Patienten mit Mukopolysaccharidose Typ I, die eine Enzymersatztherapie erhalten.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment of patients, who have Mucopolysaccharidosis Type I, receiving pentosan polysulfate subcutaneous injections weekly
    Behandlung von Patienten, die an Mukopolysaccharidose Typ I erkrankt sind, mit einer wöchentlichen Injektion Pentosanpolysulfat
    A.4.1Sponsor's protocol code number2032/MUL
    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 SponsorMultiplex Pharma Holdings LLC
    B.1.3.4CountryUnited States
    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 supportMultiplex Pharma Holdings LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationbene pharmaChem GmbH & Co. KG
    B.5.2Functional name of contact pointKlaus Maag, MD
    B.5.3 Address:
    B.5.3.1Street AddressBayerwaldstr. 7-9
    B.5.3.2Town/ cityGeretsried
    B.5.3.3Post code82538
    B.5.4Telephone number+4981719825302
    B.5.5Fax number+4981719825350
    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. name Pentosan Polysulfat SP 54® injection solution
    D. of the Marketing Authorisation holderbene-Arzneimittel 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 namePentosan Polysulphate SP54®
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 37319-17-8
    D.3.9.3Other descriptive namePentosan Polysulphate SP54
    D.3.9.4EV Substance CodeSUB14801MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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 Yes
    D. medicinal product typePentosan polysulfate sodium (PPS), is a polysaccharide extracted from beech wood and esterified with sulphuric acid. PPS belongs to the polyanions having high electric chargedensity.
    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
    Mucopolysaccharidosis type I (alpha-L-Iduronidase deficiency)
    E.1.1.1Medical condition in easily understood language
    Mucopolysaccharidosis type I (absence/insufficient levels of the enzyme alpha-L-iduronidase)
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10056888
    E.1.2Term Mucopolysaccharidosis IS
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10056887
    E.1.2Term Mucopolysaccharidosis IH/S
    E.1.2System Organ Class 100000004850
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10028094
    E.1.2Term Mucopolysaccharidosis IH
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To establish the safety of weekly subcutaneous injections of 1 mg/kg and 2 mg/kg body weight Pentosan Polysulphate SP54® in the treatment of Mucopolysaccharidosis Type I.
    E.2.2Secondary objectives of the trial
    To investigate the effect of pentosan polysulfate (PPS) on:
    - 6 minute walk test
    - pulmonary function
    - grip test
    - quality of life
    - pain intensity
    - range of motion
    - inflammatory biomarkers
    - glycosaminoglycan (GAG)
    - CRP (C-reactive protein) and ESR (erythrocyte sedimentation rate)
    - measurement of pentosan polysulfate
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female.
    2. Age ≥18 years.
    3.Diagnosed as having Mucopolysaccharidosis Type I (alpha-L-Iduronidase deficiency) and have been treated with enzyme replacement therapy (ERT) for a minimum of 24 months.
    4. Joint pain and/or reduced joint range of motion at time of enrolment according to the investigator.
    5. Written informed consent upon enrolment.
    E.4Principal exclusion criteria
    1. History of hypersensitivity to pentosan polysulfate or any of the excipients.
    2. Known allergic reaction to heparin or pentosan polysulfate e.g. heparin induced thrombocytopenia.
    3. Clinically relevant concomitant severe condition (with the exception of signs and symptoms relating to MPSI) e.g. gastrointestinal (including gastrointestinal ulcerations, polyps, or diverticula), renal, hepatic (ALT and/or AST >10 times upper limit of reference range), pancreatic, pulmonary, cardiovascular (including endocarditis lenta), aneurysm, cerebral hemorrhage, hematological (including thrombocytopenia, hemophilia, hemorrhagic diathesis), endocrinological, suspected tumors with risk of bleeding, neurological, or psychiatric conditions in the opinion of the investigator.
    4. Planned invasive procedure other than blood sampling.
    5. History of or planned bone marrow transplant.
    6. Clinically relevant abnormal laboratory test results including liver enzymes, thrombocytopenia, prolonged clotting time.
    7. Intake of anti-inflammatory agents e.g. corticosteroids, non-steroidal anti-inflammatory drugs (NSAIDs) other than ibuprofen or paracetamol within 10 half lives (t½) of the drug prior to first dosing and throughout the study.
    8. Intake of anti-thrombotic agents e.g. heparin, tissue plasminogen activator (t-PA), streptokinase, aspirin within 10 half lives (t½) of the drug prior to first dosing and throughout the study. Coumarin anticoagulants are permitted.
    9. Known history or presence of alcohol or substance abuse.
    10. Known hepatitis B or C or human immunodeficiency virus (HIV) or syphilis seropositivity. Note: testing for hepatitis and HIV will be performed as part of the screening procedures.
    11. For females: pregnancy or lactation.
    12. For females of childbearing potential: not willing and able to use a contraceptive method, which the investigator considers reliable. Reliable methods for women are hormonal contraceptives, surgical intervention (e.g. tubal ligation), intrauterine device (IUD) and sexual abstinence. Women must use a reliable method from 6 weeks before the first administration of study medication until 3 weeks after the last administration of the study medication.
    13. Participation in another clinical study involving a new chemical entity within 3 months and for studies involving biologicals 6 months prior to enrolment in this study.
    14. The subject has received vaccination(s) within 1 month prior to enrollment, or is unwilling to postpone vaccinations for the duration of the study.
    15. Subjects who are unable to comply with the requirements of the study or who in the opinion of the investigator should not participate in the study.
    E.5 End points
    E.5.1Primary end point(s)
    1. aPTT and INR
    2. Platelet count (thrombocytes)
    3. Clinical laboratory parameters: sodium, potassium, calcium, glucose, urea, creatinine, total bilirubin, AST(SGOT), ALT (SGPT), alkaline phosphatase (ALP), gamma glutamyl transpeptidase (GGT), creatinine kinase (CK), total protein, Troponin I and C-reactive protein (CRP) and erythrocyte sedimentation rate (ESR)
    4. Hematology parameters: hemoglobin, red blood cell count (erythrocytes), hematocrit, total white blood cell count (leucocytes)
    5. Adverse events and concomitant medication documented
    6. Vital signs (pulse rate, blood pressure, body temperature) documented
    7. General medical examination
    8. ECG
    E.5.1.1Timepoint(s) of evaluation of this end point
    Ad 1.: at screening, and pre-dose (immediately before start of ERT iv infusion) and 2 hours after subcutaneous injection and at Day 2 and 1 week (Week 25) and 3 weeks (Week 27) after the last dose of PPS.
    Ad 2.: at screening, pre-dose at every visit and at Day 2 and 1 week and 3 weeks after the last dose of PPS.
    Ad 3.: at screening, baseline, Week 12 and Week 25.
    Ad 4.: at screening, Week 12 and Week 25.
    Ad 5.: at every visit.
    Ad 6.: at every visit.
    Ad 7.: at screening and at Week 25.
    Ad 8.: at screening and at Week 25.
    E.5.2Secondary end point(s)
    1. Change from baseline in cytokines TNF-alpha, IL-1 and glycosaminoglycan (GAG)
    2. Change from baseline in 6 minute walk test
    3. Change from baseline in pulmonary function tests measured in the morning
    4. Change from baseline in Grip Test
    5. Change from baseline in CRP (C-reactive protein) and ESR (erythrocyte sedimentation rate)
    6. Change from baseline in SF 36 Quality of Life
    7. Change from baseline in pain assessment measured using Brief Pain Inventory
    8. Change from baseline in range of motion measured by gait analysis
    9. Pentosan polysulfate plasma concentrations measured pre-dose and 2 hours post-dose
    E.5.2.1Timepoint(s) of evaluation of this end point
    Ad. 1. - 8.: at Week 12 and at Week 25.
    Ad 9.: at Day 1, Week 2, Week 6, Week 10, Week 14, Week 18, Week 24 and once at Day 2 and Week 27.
    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. 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 Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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) Yes
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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.8.5The 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
    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 months2
    E.8.9.1In the Member State concerned days
    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: 6
    F.1.3Elderly (>=65 years) No
    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 state6
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 6
    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)
    Subjects, who benefit from the study drug treatment, can continue with the treatment at the investigator’s discretion as long as the subject benefits from treatment. bene pharmaChem will provide Pentosan Polysulphate SP54® to subjects who wish to continue treatment at no cost to the subject.
    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 Decision2014-05-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-05-28
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-04-27
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