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    Summary
    EudraCT Number:2016-002696-10
    Sponsor's Protocol Code Number:CCPK850X2202
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2017-12-21
    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 ConcernedSweden - MPA
    A.2EudraCT number2016-002696-10
    A.3Full title of the trial
    An open-label first-in-human single ascending dose study to explore the safety, tolerability and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Explore the safety and efficacy of subretinal administration of CPK850 gene therapy in patients with retinitis pigmentosa caused by mutations in the RLBP1 gene
    A.4.1Sponsor's protocol code numberCCPK850X2202
    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 SponsorNovartis Pharma AG
    B.1.3.4CountrySwitzerland
    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 supportNovartis Pharma AG
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNovartis Sverige AB
    B.5.2Functional name of contact pointMedical information
    B.5.3 Address:
    B.5.3.1Street AddressBox 1218
    B.5.3.2Town/ cityKista
    B.5.3.3Post code164 28
    B.5.3.4CountrySweden
    B.5.4Telephone number +46 8 7323200
    B.5.6E-mailmedinfo.se@novartis.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.2Product code CPK850
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubretinal use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNTBD
    D.3.9.2Current sponsor codeCPK850
    D.3.9.4EV Substance CodeSUB190236
    D.3.10 Strength
    D.3.10.1Concentration unit vector genomes (vg)/mL
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number50000000000
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    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
    retinitis pigmentosa caused by biallelic mutations in the RLBP1 gene
    E.1.1.1Medical condition in easily understood language
    RLBP1 retinitis pigmentosa (RLBP1 RP) is an autosomal recessive form of inherited retinitis pigmentosa (RP) caused by biallelic mutations in the retinaldehyde binding protein 1 (RLBP1) gene
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    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.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Safety:
    - To evaluate the ocular and systemic safety of a single dose of CPK850 in patients with RLBP1 retinitis pigmentosa.
    - Ophthalmic safety parameters: best corrected visual acuity (BCVA), intraocular pressure (IOP), slit lamp examination (biomicroscopy), dilated fundus examination, optical coherence tomography (OCT), and color fundus photography (CFP) parameters.
    - Systemic safety parameters: adverse events (AEs), blood chemistry, hematology, urinalysis and vital signs.

    Efficacy:
    To evaluate the recovery of the rod system in eyes treated with CPK850 as compared to pre-treatment assessments.
    - Recovery (pre-bleach minus post-bleach values in log10 units) of dark adaptation (DA) at 1 hour post-bleach.
    - Pre-bleach DA values (in log10 units).
    - Bleaching effect (bleaching minus pre-bleach thresholds in log10 units).
    - Dark adaptation kinetics from pre-bleach to 6 hours post-bleach (in log10 units).
    E.2.2Secondary objectives of the trial
    - To evaluate the recovery of the cone system in eyes treated with CPK850 as compared to pre-treatment assessments.
    - To evaluate the recovery of the rod system in eyes treated with CPK850 as compared to the untreated eye.
    - To evaluate changes from baseline in measures of visual function in eyes treated with CPK850 as compared to pre-treatment assessments as well as to untreated eyes.
    - To evaluate the changes from baseline in the electrical rod and cone system function in eyes treated with CPK850 as compared to pre-treatment assessments as well as to untreated eyes.
    - To evaluate the change from baseline in navigation skills in eyes treated with CPK850 as compared to pre-treatment assessments as well as to untreated eyes.
    - To assess the effects of CPK850 on activities of daily living as compared to pre-treatment assessments

    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    -Written informed consent must be obtained before any
    assessment is performed.
    - Male and female patients aged 18 to 70 years. All female patients must have negative pregnancy test results at the screening visits and just prior to the treatment day.
    - The patients must have sufficiently clear ocular media and adequate pupil dilation to permit fundus photographs of adequate clarity and performance of vitrectomy and subretinal injection.
    - Patients must meet surgical requirements. In addition, patients must weigh at least 40 kg to participate in the study, and must have a body mass index (BMI) <40 kg/m2. BMI = Body weight (kg) / [Height (m)]2.
    - The visual acuity in the study eye at the screening 1 visit should be no better than 60 early treatment diabetic retinopathy study (ETDRS) letters. In Cohort 1, patients with visual acuity from 35 ETDRS letters to Hand Motion visual acuity will be included. For Cohorts 2 and beyond, patients with visual acuity of 60 ETDRS letters to Hand Motion visual acuity will be enrolled. Consideration for inclusion in Cohorts 1 and 2 only may be given to patients with visual acuity as low as Light Perception at the screening 1 visit, if they meet all other inclusion/exclusion criteria and at the agreement of the sponsor and principal investigators.
    - Clinical diagnosis of Bothnia dystrophy, Newfoundland rod-cone dystrophy or other progressive retinitis pigmentosa phenotype with biallelic mutations in the RLBP1 gene verified by Clinical Laboratory Improvement Amendments (CLIA), Good Laboratory Practices (GLP) or equivalent molecular genetics testing.
    - Visible photoreceptor (outer nuclear) and retinal pigment epithelium (RPE) layers on standard OCT scan in the study eye at the screening 1 visit as confirmed by the Central Reading Center.
    - Dark adaptation bleaching effect in the study eye at short wavelength stimulus of > 1.0 log unit at 2 of the 3 baseline measures obtained prior to treatment.
    E.4Principal exclusion criteria
    - Unable or unwilling to meet requirements of the study
    - History of hypersensitivity to the study drug or to drugs of similar classes or to any of the medications required in the perioperative period.
    - Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints; for example: glaucoma (IOP ≥25 mm Hg despite treatment with anti-glaucoma medication or low tension glaucoma), corneal or significant lenticular opacities, retinal vascular occlusion, retinal detachment, macular hole, or choroidal neovascularization of any cause.
    - Any active infection or ocular disease involving ocular adnexa including infectious conjunctivitis, keratitis, scleritis, endophthalmitis, as well as idiopathic or autoimmune-associated uveitis in either eye.
    - Any contraindication to the planned surgery or anesthesia as determined by the treating physician (surgeon, anesthesiologist, primary care physician or designee). Use of systemic anticoagulant therapies during the study, such as warfarin, heparin or similar are to be evaluated by the treating physician as potential exclusions. The use of aspirin is not usually an exclusion criterion unless indicated by the treating physician. Abnormal vital signs and/or Electrocardiograms (ECGs) that suggest potential contraindications for planned study anesthesia are exclusions.
    - Known history of or current clinically significant arrhythmias, myocardial infarction, coronary bypass surgery, or any percutaneous coronary intervention (PCI) within 6 months prior to screening or patients with heart failure New York Heart Association (NYHA) class II-IV at the discretion of the treating physician or cardiologist.
    - Cerebrovascular accident (stroke) within the 12 months prior to screening at the discretion of the treating physician.
    - Complicating systemic diseases or clinically significant abnormal laboratory values. Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter ocular function. Examples are malignancies whose treatment could affect central nervous system function (for example radiation treatment of the orbit; leukemia with central nervous system (CNS)/optic nerve involvement). Also patients with immunocompromising diseases would be excluded since they would have susceptibility to opportunistic infections.
    - Women who are pregnant or nursing (lactating), as well as male and female patients of childbearing potential that are unwilling to use contraception as per study requirements.
    E.5 End points
    E.5.1Primary end point(s)
    -Number of participants with adverse events (AEs), serious adverse events(SAEs) and deaths
    -Number of responders in dark adaptation (A patient is considered a responder if sensitivity recovery values at 1 hour post-bleach are observed to be outside of the patient’s prediction interval at ≥2 consecutive posttreatment
    visits within one year after treatment.)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Number of participants with adverse events (AEs), serious adverse events(SAEs) and deaths: up to year 5
    -Number of responders in dark adaptation (A patient is considered a responder if sensitivity recovery values at 1 hour post-bleach are observed to be outside of the patient’s prediction interval at ≥2 consecutive posttreatment
    visits within one year after treatment.): Screening/baseline up to year 1
    E.5.2Secondary end point(s)
    1 Number of patients with recovery of the cone system
    2 Number of patients with improvement in rod function in the treated eye vs the untreated eye
    3 Change from screening/baseline in Visual field perimetry mean deviation
    4 Change from screening/baseline in Total contrast sensitivity score
    5 Change from screening/baseline in Lightadapted microperimetry sensitivity
    6 Change from screening/baseline in Reading speed
    7 Change from screening/baseline in the local electrical activity of the retina
    8 Change from screening/baseline in eye dominance
    9 Change from screening/baseline in mobility test scores
    10 Change from screening/baseline in the National Eye Institute -Visual function questionnaire 25 (NEI-VFQ 25) composite score
    11 Change from screening/baseline in the low luminance questionnaire (LLQ) responses
    12 Change from screening/baseline in the Functional vision questionnaire (FVQ), as available
    13 Change from screening/baseline in the electrical activity of the retina
    E.5.2.1Timepoint(s) of evaluation of this end point
    1 to 13: screening/baseline up to year 1
    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 No
    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) Yes
    E.7.1.1First administration to humans Yes
    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) Yes
    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 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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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
    LVLS (Last Visit Last Subject)
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days19
    E.8.9.2In all countries concerned by the trial years7
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days19
    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: 18
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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 state21
    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)
    All patients should have a safety follow-up call conducted 30 days after last visit. The information collected is kept as source documentation.
    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 Decision2018-03-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-05-09
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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