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    Summary
    EudraCT Number:2009-017882-42
    Sponsor's Protocol Code Number:RP103-03
    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:2010-06-25
    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 number2009-017882-42
    A.3Full title of the trial
    A Randomized, Crossover, Pharmacokinetic and Pharmacodynamic
    Study to Determine the Safety and Efficacy of Cysteamine Bitartrate
    Delayed-release Capsules (RP103), Compared to Cystagon® in Patients
    with Nephropathic Cystinosis
    A.4.1Sponsor's protocol code numberRP103-03
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorRaptor Therapeutics 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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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.3.1Product nameCysteamine bitartrate (INN: mercaptamine bitartrate
    D.3.2Product code RP103
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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, 75
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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 RoleComparator
    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 Cystagon
    D.2.1.1.2Name of the Marketing Authorisation holderOrphan Europe
    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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPOral use
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 12.1
    E.1.2Level LLT
    E.1.2Classification code 10011777
    E.1.2Term Cystinosis
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate that at steady-state, patients receiving every 6 hour
    (Q6H) treatment of Cystagon® can maintain a comparable depletion
    of white blood cell (WBC) cystine levels after receiving every 12
    hour (Q12H) treatment regimen of RP103
    E.2.2Secondary objectives of the trial
    To assess safety and tolerability of RP103.
    To assess the steady-state pharmacokinetics (PK) and
    pharmacodynamics (PD) of RP103 compared to Cystagon®.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Male or female subject with a documented diagnosis of
    nephropathic cystinosis.
    2. Subject must be on a stable dose of Cystagon® sufficient to
    maintain their white blood cell (WBC) cystine level at ≤ 1.0
    nmol/half-cystine/mg protein.
    3. Subject must be able to swallow their typically administered
    Cystagon® capsule with the capsule intact.
    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. Subject’s age < 6 years old or subjects weight < 21 kg.
    2. 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.
    3. 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.
    4. Subjects receiving any form of cysteamine medication through a
    gastric tube.
    5. Subjects who are receiving maintenance dialysis or who have
    had a kidney transplant.
    6. Subjects who are on an active kidney transplant list or who are
    planning to receive a kidney transplant within 3 months of
    Screening.
    7. Subjects with known hypersensitivity to cysteamine and
    penicillamine.
    8. Female subjects who are nursing, planning a pregnancy, known
    or suspected to be pregnant, or with a positive serum pregnancy
    screen.
    9. Subjects who have a made a blood donation within 30 days of
    Screening.
    10. 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)
    Comparable depletion of steady-state cysteamine-trough WBC
    cystine levels (Days 5, 6 and 7 of Week 6 (Period 1) and Week 9
    (Period 2) between Cystagon® and RP103. If the one-sided test
    of non-inferiority, conducted at the nominal level of 0.02104, is
    rejected at a non-inferiority margin of 0.3, we will conclude that
    RP103 is non-inferior to Cystagon® with an overall significance
    level of 0.025.
    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 Yes
    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 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 Yes
    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.8.2.3.1Comparator description
    Cystagon
    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 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
    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
    last patient last visit
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months3
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 12
    F.4.2.2In the whole clinical trial 20
    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 Opinion2010-10-19
    P. End of Trial
    P.End of Trial StatusCompleted
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