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    Summary
    EudraCT Number:2010-018365-34
    Sponsor's Protocol Code Number:RP103-04
    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:2010-07-06
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2010-018365-34
    A.3Full title of the trial
    A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Nephropathic Cystinosis
    A.3.2Name or abbreviated title of the trial where available
    RP103-04
    A.4.1Sponsor's protocol code numberRP103-04
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01733316
    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 SponsorHorizon Pharma USA, Inc.
    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 supportHorizon Pharma Ireland Limited
    B.4.2CountryIreland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHorizon Pharma USA, Inc.
    B.5.2Functional name of contact pointSenior Director, Regulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address150 South Saunders Road
    B.5.3.2Town/ cityForest Lake, IL
    B.5.3.3Post code60045
    B.5.3.4CountryUnited States
    B.5.4Telephone number+14154086284
    B.5.6E-mailskumar@horizonpharma.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 Yes
    D.2.1.1.1Trade name PROCYSBI
    D.2.1.1.2Name of the Marketing Authorisation holderChiesi Orphan B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/10/778
    D.3 Description of the IMP
    D.3.1Product nameCysteamine bitartrate (INN: mercaptamine bitartrate )
    D.3.2Product code RP103
    D.3.4Pharmaceutical form Gastro-resistant 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 INNmercaptamine bitartrate
    D.3.9.1CAS number CAS 27761-19
    D.3.9.2Current sponsor codeRP103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number25
    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.IMP: 2
    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 PROCYSBI
    D.2.1.1.2Name of the Marketing Authorisation holderChiesi Orphan B.V.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation numberEU/3/10/778
    D.3 Description of the IMP
    D.3.1Product nameCysteamine bitartrate (INN: mercaptamine bitartrate )
    D.3.2Product code RP103
    D.3.4Pharmaceutical form Gastro-resistant 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 INNmercaptamine bitartrate
    D.3.9.1CAS number CAS 27761-19
    D.3.9.2Current sponsor codeRP103
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Cystinosis
    E.1.1.1Medical condition in easily understood language
    Cystinosis
    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 20.0
    E.1.2Level PT
    E.1.2Classification code 10011777
    E.1.2Term Cystinosis
    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
    Primary Objective:
    • To assess safety and tolerability of long-term repeat dosing of RP103 in patients with nephropathic cystinosis.
    E.2.2Secondary objectives of the trial
    Secondary Objectives:

    • To assess the steady-state pharmacokinetics (PK) and pharmacodynamics (PD) of RP103.
    • To assess the long term quality of life using either PedsQL™ or SF-36® instruments.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    Optional PK Substudy Visit for Subjects 6 Years Old or Younger:
    The optional PK Substudy Visit may take place during a regularly scheduled RP103-04 Quarterly Visit, or it may take place as a separately scheduled visit. PK samples will be collected within 15 minutes pre- and 0.5, 1, 2, 3, 4, 6, 8, 10 and 12 hours post-RP103 morning dose. PD samples will be collected at 0.5 and 3 hours post-RP103 morning dose. During the PK Substudy Visit, blood samples will be collected at the following timepoints:
    PK Blood Samples:
    Time 0 (within 15 minutes prior to the morning RP103 dose)
    0.5 hour (30 minutes) post-dose
    1 hour post-dose
    2 hours post-dose
    3 hours post-dose
    4 hours post-dose
    6 hours post-dose
    8 hours post-dose
    10 hours post-dose
    12 hours post-dose (within 15 minutes prior to the evening RP103 dose)
    PD Blood Sample:
    0.5 hour (30 minutes) after the morning RP103 dose
    3 hours post-dose
    E.3Principal inclusion criteria
    1. Male and female subjects must have completed the last visit of Study RP103-03 and be willing to continue with RP103 treatment.
    OR for patients who did not complete the RP103-03 study:
    2. Male and female subject with a documented diagnosis of cystinosis.
    3. Subject must be on a stable dose of Cystagon® at least 21 days prior to Screening.
    4. Within the last 6 months, no clinically significant change from normal in liver function tests [i.e., 1.5 times ULN for ALT and AST, and/or 1.5 times ULN for total bilirubin] and renal function [i.e., estimated GFR (corrected for body surface area)] at Screening as determined by the Investigator.
    5. Subject must have an estimated GFR (corrected for body surface area) > 30 mL/minute/1.73 m2.
    6. Sexually active female subjects of childbearing potential (i.e., not surgically sterile [tubal ligation, hysterectomy, or bilateral oophorectomy] or at least 2 years naturally postmenopausal) must agree to utilize the same acceptable form of contraception from Screening through completion of the Study. The acceptable forms of contraception for this Study include hormonal contraceptives (oral, implant, transdermal patch, or injection) at a stable dose for at least 3 months prior to Screening, barrier (spermicidal condom, diaphragm with spermicide), IUD, or a partner who has been vasectomized for at least 6 months. For pre-pubescent children, a documented attestation of abstinence from their parent or guardian will be acceptable.
    7. Subject must be willing and able to comply with the Study restrictions and requirements.
    8. Subject or their parent or guardian must provide written informed consent and assent (where applicable) prior to participation in the Study.
    E.4Principal exclusion criteria
    1. Patients enrolled in the previous Study RP103-03 who did not complete their last scheduled study visit or who do not wish to continue on treatment with RP103.
    AND for patients who did not complete the RP103-03 study:
    2. Subjects less than 1 year old
    3. Subjects with current history of the following conditions or any other health issues that make it, in the opinion of the Investigator, unsafe for them to participate:
    • Inflammatory bowel disease (if currently active) or prior resection of small intestine;
    • Heart disease (e.g., myocardial infarction, heart failure, unstable arrhythmias, or poorly controlled hypertension) 90 days prior to Screening;
    • Active bleeding disorder 90 days prior to Screening;
    • History of malignant disease within the last 2 years.
    4. Subject with a hemoglobin level of < 10 g/dL at Screening or, in the opinion of the Investigator, a hemoglobin level that would make it unsafe for the subject to participate.
    5. Subjects with known hypersensitivity to cysteamine and penicillamine.
    6. Female subjects who are nursing, planning a pregnancy, known or suspected to be pregnant, or with a positive serum pregnancy screen.
    7. Subjects who, in the opinion of the Investigator, are not able or willing to comply with the protocol.
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints:
    • The safety profile of RP103 will be investigated by safety reviews conducted by the Investigator at or prior to each Monthly or Quarterly visit and through adverse event report monitoring (including attribution of treatment-emergent adverse events and serious adverse events [SAEs]), physical examination (including basic neurological and skin assessment findings), vital signs, ECG and clinical laboratory testing as outlined in the Schedule of Events.

    Pharmacokinetic Endpoint:
    • The trough plasma cysteamine concentration will be measured from samples collected 0.5 hour post dose at each Study visit.

    Pharmacodynamic Endpoint:
    •The WBC cystine content will be measured from samples collected 0.5 hour post dose at each Study visit.

    Exploratory Endpoints:
    • The longitudinal change in the use of concomitant gastric acid reduction therapies, VAS swallowing difficulty and PedsQL™ or SF-36® questionnaire scores while being administered RP103.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Monthly Study Visits are only for patients who completed Study RP103-03 and will begin within 1 month (± 7 days) after study entry. Each patient will complete a minimum of 6 consecutive Monthly visits.
    Quarterly Study Visits will commence for each patient ≥ 1 month and < 4 months after the patient has completed at least 6 consecutive Monthly visits.
    Each Quarterly visit will take place by the middle of the first month (± 7 days) of each calendar quarter (i.e., January 15, April 15, July 15 and October 15).
    Depending on enrollment timing, up to 6 Quarterly visits may occur over this 3 year study
    E.5.2Secondary end point(s)
    To assess the steady-state pharmacokinetics (PK) and pharmacodynamics(PD) of RP103.
    To assess the long term quality of life using either PedsQL™ or SF-36® instruments.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Monthly Study Visits are only for patients who completed Study RP103-03 and will begin within 1 month (± 7 days) after study entry. Each patient will complete a minimum of 6 consecutive Monthly visits.
    Quarterly Study Visits will commence for each patient ≥ 1 month and < 4 months after the patient has completed at least 6 consecutive Monthly visits.
    Each Quarterly visit will take place by the middle of the first month (± 7 days) of each calendar quarter (i.e., January 15, April 15, July 15 and October 15).
    Depending on enrollment timing, up to 6 Quarterly visits may occur over this 3 year study
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.2.4Number of treatment arms in the trial1
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA5
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    France
    United States
    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 years6
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months6
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.2Adults (18-64 years) Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 60
    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 Decision2010-07-22
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-12-14
    P. End of Trial
    P.End of Trial StatusCompleted
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