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    Summary
    EudraCT Number:2013-005393-22
    Sponsor's Protocol Code Number:RETIRD04
    National Competent Authority:Denmark - DHMA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2016-09-06
    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 ConcernedDenmark - DHMA
    A.2EudraCT number2013-005393-22
    A.3Full title of the trial
    A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study in subjects with rare inherited eye conditions caused by gene mutations to see if treatment with QLT091001 is safe and works to improve subjects' vision.
    A.4.1Sponsor's protocol code numberRETIRD04
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/023/2014
    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 organisationQLT Inc.
    B.5.2Functional name of contact pointMedical Affairs
    B.5.3 Address:
    B.5.3.1Street Address887 Great Northern Way, Suite 250
    B.5.3.2Town/ cityVancouver, British Columbia
    B.5.3.3Post codeV5T 4T5
    B.5.3.4CountryCanada
    B.5.4Telephone number+1877764 3131
    B.5.5Fax number+1604707 7312
    B.5.6E-mailmedaff@qltinc.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 numberEU/3/11/861 (LCA); EU/3/11/865 (RP)
    D.3 Description of the IMP
    D.3.1Product nameQLT091001
    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.3.8INN - Proposed INNZuretinol Acetate
    D.3.9.3Other descriptive nameQLT091001
    D.3.9.4EV Substance CodeSUB32727
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboOral solution
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT) genes
    E.1.1.1Medical condition in easily understood language
    Inherited retinal disease is a condition caused by gene mutations that lead to severe visual impairment and blindness.
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 19.0
    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 19.0
    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 efficacy, tolerability, pharmacokinetics (PK), and safety of oral QLT091001 in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations
    E.2.2Secondary objectives of the trial
    Not applicable
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female subjects of any race with IRD (phenotypically diagnosed as LCA or RP by an ocular geneticist or ophthalmologist) caused by pathologic autosomal recessive mutation in RPE65 or LRAT (as determined by a fully accredited certified central genotyping laboratory).

    2. Naïve to treatment with QLT091001, gene therapy and surgical implantation of prosthetic retinal chips or sub-retinal injections.

    3. 6 to 40 years of age, inclusive.

    4. Ability to concentrate and fixate adequately to complete visual field evaluations.

    5. In at least one eye and in the same eye:
    • ETDRS HLHC BCVA of better than 1.5 logMAR (≥10 letters at 1 meter); BCVA can be measured a maximum of 3 times between screening (Day -42) and baseline (Day -1) in order to obtain 2 reliable assessments (both assessments must meet the criterion), and

    • Continuous central visual field solid angle of 0.023-1.130 steradians (corresponding visual field diameter of approximately 10-70 degrees) in each of at least 3 isopters on kinetic perimetry (peripheral islands allowed); perimetry can be assessed a maximum of 6 times between screening (Day -42) and baseline (Day -1) in order to obtain 3 reliable assessments (all 3 assessments must meet the criterion)

    6. Pregnancy testing and contraception:
    • Before study treatment: Females 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 a highly effective method of birth control for at least 1 month. Highly effective methods of birth control include:
    -Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal)
    -Progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable)
    -Intrauterine device (IUD)
    -Intrauterine hormone-releasing system (IUS)
    -Bilateral tubal occlusion
    -Vasectomized partner
    -True abstinence, when this is in line with the preferred and usual lifestyle of the subject (periodic abstinence [e.g., calendar, ovulation, symptothermal post-ovulation methods] and withdrawal are not acceptable methods of contraception)
    A woman is considered to be of child-bearing potential unless she meets at least one of the following criteria:
    - Previous bilateral salpingo-oophorectomy or hysterectomy
    - Premature ovarian failure confirmed by a specialist
    - XY genotype, Turner syndrome, uterine agenesis
    - Post-menopausal, defined as 12 consecutive months with no menses without alternative medical cause

    • During the study:
    - Females of child-bearing potential must be willing to receive contraceptive counseling before each treatment course.
    - If the subject is a female under 18 years of age, the legal guardian(s) must agree with the use of contraception.
    - Females of child-bearing potential must practice a highly effective method of birth control (as described above) during the treatment phase of the study and for 2 months after finishing the last dose of study drug. True abstinence during the trial is defined as refraining from heterosexual intercourse during the entire period of risk associated with the study treatments (during the treatment phase and for 2 months after finishing the last dose of study drug); periodic abstinence [e.g., calendar ovulation, symptothermal post-ovulation methods], declaration of abstinence for the duration of the trial, and withdrawal are not acceptable methods of contraception.

    7. Males must either practice true abstinence , or have had a vasectomy (with documented infertility), or agree to use a barrier method (condoms) with spermicide during sexual intercourse (supplemented by one of the highly effective birth control methods defined above for their female partner), during the treatment phase of the study and for 3 months after finishing the last dose of study drug.

    8. Sign an approved informed consent form for the study.

    9. Willing and able to comply with the protocol.
    E.4Principal exclusion criteria
    1. Presence of any concurrent ocular disease that would affect study outcomes (e.g., severe cataracts; subjects can be enrolled 3 months after successful cataract surgery).

    2. Presence of extensive degenerative pigmentary changes throughout the majority of the posterior pole and peripheral retina. This includes both hyper and hypopigmentary (atrophic) changes as determined by the Investigator.

    3. Use of any prescription or investigational oral retinoid medication (e.g., isotretinoin or acitretin) within 6 months of screening.

    4. Intolerance to previous retinoid medication.

    5. Use of any supplements containing ≥10,000 IU vitamin A within 60 days of screening.

    6. Use of any medication that affects bone metabolism within 6 months of screening.

    7. Circulating 25-hydroxy vitamin D (25-OHD) <20 ng/mL.

    8. Use of medications that may interact with alitretinoin, including tetracyclines, ketoconazole, and methotrexate within 60 days of screening.

    9. Use of any medication that prolongs the QTc interval within 60 days of screening.

    10. Prolongation of QTcB intervals as measured in baseline ECG (repeated demonstration [e.g., 2 of 3 assessments]) with average of >450 milliseconds.

    11. History of risk factors for torsade de pointes (e.g., heart failure, hypokalemia, history or family history of Long QT Syndrome), and Wolff-Parkinson-White syndrome.

    12. History of diabetes, chronic hyperlipidemia, pancreatitis, hepatitis, cirrhosis, liver failure, or hypervitaminosis A.

    13. History of idiopathic elevation of intracranial pressure.

    14. Subjects with any of the following findings at screening:
    • Uncontrolled blood pressure upon repeated measurement (i.e., 2 measurements) taken in a sitting or supine position of the following (by age group):
    6-9 years: 120/80 mmHg or higher
    10-13 years: 132/83 mmHg or higher
    14 years and older: 140/90 mmHg or higher

    • Resting heart rate upon repeated measurement of the following (unless the subject has a known and documented consistent lower heart rate):
    6-9 years: <70 bpm or >130 bpm
    10-13 years: <45 bpm or >105 bpm
    14 years and older: <40 bpm or >100 bpm

    • Fasting alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >2 times the upper limit of the clinical laboratory value normal range (upon repeated measurement).
    • Fasting total cholesterol, triglycerides, or LDL >2 times the upper limit of the clinical laboratory value normal range (upon repeated measurement).

    • Thyroid function tests (upon repeated measurement) indicating uncontrolled thyroid disease in the Investigator’s opinion.

    • Serum retinol clinical laboratory value above 98 μg/dL or the upper limit of normal, whichever is higher (upon repeated measurement).

    15. Known and documented allergy to soy.

    16. In the Investigator’s opinion, any severe acute or chronic medical condition, psychiatric condition, physical examination finding 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.

    17. Subjects who are actively participating in an experimental therapy study or who have received another experimental therapy within 60 days of screening.

    18. Subjects who have lumbar spine bone mineral density worse than -2Z.
    E.5 End points
    E.5.1Primary end point(s)
    Percent change from baseline in visual field volume in the study eye
    E.5.1.1Timepoint(s) of evaluation of this end point
    12 months, course 12
    E.5.2Secondary end point(s)
    Secondary Efficacy:
    • Change from baseline in visual field volume in the study eye
    • Visual field volume in the study eye
    • Proportion of subjects with increase in visual field volume in the study eye of at least 10% in Course 12, and proportion of subjects with increase in visual field volume in the study eye of at least 20% in Course 12
    • Change from baseline in HLHC BCVA (ETDRS at 4 meters or 1 meter) in the study eye
    • Change from baseline in LLLC BCVA (ETDRS at 4 meters or 1 meter) in the study eye
    • Patient reported outcomes
    E.5.2.1Timepoint(s) of evaluation of this end point
    12 months, course 12
    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 No
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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) No
    E.8.2.2Placebo Yes
    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 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 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
    Brazil
    Canada
    Denmark
    France
    Germany
    Netherlands
    Switzerland
    United Kingdom
    United States
    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
    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 months8
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months11
    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: 20
    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: 10
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 28
    F.1.3Elderly (>=65 years) No
    F.1.3.1Number of subjects for this age range: 0
    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
    The study will include subjects aged 6-40 years old. Most authorities opine that children below the age of 7 lack the capacity to understand concepts related to the conduct of this study, and thus are considered as incapable of giving assent.
    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.4.2.1In the EEA 24
    F.4.2.2In the whole clinical trial 48
    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)
    Following the end of trial, the sponsor will consider a compassionate use programme for subjects enrolled on this trial if it is considered to be in the subject's best interest.
    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 Decision2016-09-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-11-03
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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