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    Summary
    EudraCT Number:2011-004214-42
    Sponsor's Protocol Code Number:RETIRD02
    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:2012-05-03
    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 ConcernedGermany - BfArM
    A.2EudraCT number2011-004214-42
    A.3Full title of the trial
    An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' vision
    A.4.1Sponsor's protocol code numberRETIRD02
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01521793
    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 SponsorQLT Inc.
    B.1.3.4CountryCanada
    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 supportQLT Inc.
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPSI Co Ltd.
    B.5.2Functional name of contact pointRegulatory Affairs
    B.5.3 Address:
    B.5.3.1Street Address19/21 Dostoyevsky St.
    B.5.3.2Town/ citySaint Petersburg
    B.5.3.3Post code191119
    B.5.3.4CountryRussian Federation
    B.5.4Telephone number7812320 3820
    B.5.5Fax number7812320 3850
    B.5.6E-mailRAEurope@psi-cro.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 Yes
    D.2.5.1Orphan drug designation numberLCA:EU/3/11/861 RP:EU/3/11/865
    D.3 Description of the IMP
    D.3.2Product code QLT091001
    D.3.4Pharmaceutical form Oral solution
    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.3.9.1CAS number 29584-22-3
    D.3.9.2Current sponsor codeQLT091001
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Leber Congenital Amaurosis
    Retinitis pigmentosa
    E.1.1.1Medical condition in easily understood language
    Inherited ophthalmology disorder
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10038914
    E.1.2Term Retinitis pigmentosa
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10070667
    E.1.2Term Leber's congenital amaurosis
    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
    • To evaluate the safety of up to 3 additional courses of oral QLT091001 administered once daily for 7 days in subjects with LCA or RP caused by RPE65 or LRAT gene mutations who have been treated previously with a single 7-day course of QLT091001 in Study RET IRD 01
    • To evaluate whether up to 3 additional courses of oral QLT091001 administered once daily for 7 days can maintain or improve visual function in subjects with LCA or RP caused by RPE65 or LRAT gene mutations who have been treated previously with a single 7-day course of QLT091001 in Study RET IRD 01
    E.2.2Secondary objectives of the trial
    Not applicable.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects with LCA or RP due to RPE65 or LRAT deficiency who received a 7-day treatment course of QLT091001 and completed the Day 30 visit in Study RET IRD 01.

    2. Subjects who meet any one of the following criteria at least 1 month after the start of the 7-day treatment course in Study RET IRD 01:
    a. No increase in GVF (in at least 1 eye):i.e. follow-up GVF increased ≤20% from baseline or
    b. Decrease in GVF (in at least 1 eye): i.e. follow-up GVF decreased below the highest previous response by >20% or
    c. Considered a reasonable candidate for treatment, as determined by the Investigator based on regression or lack of response in other parameters of visual function (e.g., subjective reports of changes in color vision or adaptation to low light) but who do not meet the other (GVF) criteria for study entry

    3. Subjects who are girls or women of child-bearing potential must not be pregnant or lactating, must have negative serum pregnancy tests (≥25 mIU/mL sensitivity) at Screening (i.e., ≥19 days before Day -1, and on Day -1) and must have been practicing 2 adequate methods of birth control or complete abstinence for at least 2 months prior to study dosing. Adequate methods of birth control include (1) use of oral contraceptives (excluding low-dose oral formulation), implantable or injectable contraceptives, or an intrauterine device (IUD), with an additional barrier method (diaphragm with spermicidal gel OR condoms with spermicide); (2) a double-barrier method (diaphragm with spermicidal gel AND condoms with spermicide); (3) partner vasectomy; and (4) total abstinence. (Women are considered postmenopausal after 1 complete year of amenorrhea).

    4. Subjects who are boys or men must (1) agree to completely abstain from sexual intercourse, (2) have had a vasectomy, (3) use a barrier method (condoms) with spermicide and, if his partner is not using oral contraceptives, implantable or injectable contraceptives, or an IUD (as described in the criterion above), ensure that she is using a diaphragm with spermicidal gel during sexual intercourse, during the treatment phase of the study and for 2 months after finishing the last dose of study drug in Treatment Course 3.

    5. Subjects who provide informed consent and, if applicable, assent, for the study. The parent or guardian must sign an approved informed consent form for the study for subjects younger than the age of majority.
    Note that use of the term "subject" throughout this protocol may also include the parent/guardian of subjects younger than the age of majority, as appropriate.

    6. Subjects who are willing to comply with the protocol.
    E.4Principal exclusion criteria
    1. Subjects with any clinically important abnormal physical finding at Screening as determined by the Investigator.

    2. Subjects who have taken any prescription or investigational oral retinoid medication (e.g., Accutane/Roaccutane® or Soriatane/Neotigason®) excluding study medication QLT091001 within 6 months of Day 0 and subjects who did not tolerate their previous oral retinoid medication will be excluded regardless of the time of last exposure.

    3. Subjects with liver failure, cirrhosis, hepatitis, uncontrolled thyroid disease or chronic hyperlipidemia.

    4. Subjects with a hypersensitivity to retinoids or hypervitaminosis A syndrome

    5. Subjects with any of the following findings at Screening:
    - Blood pressure reading upon repeated measurement (i.e., 2 measurements) taken in a sitting or supine position of the following (by age group).
    5-9 years: 120/80 mmHg or higher
    10-13 years: 132/83 mmHg or higher
    14-17 years: 140/90 mmHg or higher
    18 years and older: 150/95 mmHg or higher
    - Resting heart rate upon repeated measurement of the following:
    5-9 years: <70 bpm or >130 bpm
    10-13 years: <45 bpm or >105 bpm
    14-17 years: <40 bpm or >100 bpm
    18 years and older: <40 bpm or >100 bpm
    - ALT, AST or fasting triglycerides >3 times the upper limit of the clinical laboratory value normal range (upon repeated measurement)
    - Thyroid stimulating hormone (TSH) outside the clinical laboratory value normal range (upon repeated measurement); other thyroid function tests to be evaluated by Investigator to exclude subjects with uncontrolled thyroid disease
    - Serum retinol clinical laboratory value above 98 μg/dL
    - Subjects who, in the Investigator’s opinion, have any severe acute or chronic medical condition, psychiatric condition, or laboratory abnormality that may increase the risk associated with study participation or administration of study treatment, or interfere with the interpretation of study results.

    6. Subjects with a marked QTc prolongation (>450 milliseconds [ms]) upon repeated demonstration (e.g., 2 of 3 assessments) at screening or baseline, which is considered to be clinically significant by the Investigator.

    7. Subjects with a history of risk factors for torsade de pointes (TdP) (e.g., heart failure, hypokalemia, history or family history of Long QT Syndrome), and Wolff-Parkinson-White (WPW) syndrome.

    8. Subjects who have taken any supplements containing ≥10,000 IU vitamin A within 60 days of Screening.

    9. Subjects who are actively participating in an experimental therapy study (other than RET IRD 01) or who have received another experimental therapy (in addition to QLT091001) within 60 days of Day 0.

    10. Subjects with a documented and known allergy to soy
    E.5 End points
    E.5.1Primary end point(s)
    Assessment of safety
    Assessment of vision
    E.5.1.1Timepoint(s) of evaluation of this end point
    For Treatment Course 1:
    Vision assessments will be conducted at screening, baseline, Day 7/8, 14/15, 30; Months 2, 4, 6.
    General safety assessments will be conducted at screening, baseline, Day 0-7/8, 14/15, 30; Months 2, 4, 6.
    For Treatment Courses 2 and 3:
    Vision assessments will be conducted at baseline, Day 7/8, 14/15, 30; Months 2, 4, 6.
    General safety assessments will be conducted at baseline, Day 0-7/8, 14/15, 30; Months 2, 4, 6.
    E.5.2Secondary end point(s)
    not applicable
    E.5.2.1Timepoint(s) of evaluation of this end point
    not applicable
    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 No
    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) Yes
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other Yes
    E.7.1.3.1Other trial type description
    Phase 1b Continuation Study
    E.7.2Therapeutic exploratory (Phase II) No
    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 Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    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.8.3 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 EEA3
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Germany
    Netherlands
    United Kingdom
    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 years3
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months9
    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.1.1Number of subjects for this age range: 11
    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.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    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 state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 13
    F.4.2.2In the whole clinical trial 31
    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)
    N/A - expected normal treatment
    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 Decision2012-08-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-06-26
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-05-15
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