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    EudraCT Number:2016-001429-16
    Sponsor's Protocol Code Number:HORA-PDE6B-001
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2017-09-12
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2016-001429-16
    A.3Full title of the trial
    Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients Harboring Mutations in the PDE6B Gene Leading to a Defect in PDE6B Expression.
    Etude de sécurité et d’efficacité après administration unilatérale sous-rétinienne de HORA-PDE6B chez des patients porteurs de mutations du gène PDE6B conduisant à un défaut d’expression de PDE6B
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients Harboring Mutations in the PDE6B Gene Leading to a Defect in PDE6B Expression.
    Etude de sécurité et d’efficacité après administration unilatérale sous-rétinienne de HORA-PDE6B chez des patients porteurs de mutations du gène PDE6B conduisant à un défaut d’expression de PDE6B
    A.4.1Sponsor's protocol code numberHORA-PDE6B-001
    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 SponsorHORAMA SA
    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 supportHORAMA SA
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationHORAMA SA
    B.5.2Functional name of contact pointChief Medical Officer
    B.5.3 Address:
    B.5.3.1Street Address9 rue de l'Eperon
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75006
    B.5.4Telephone number330953 73 68 15
    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/3/13/1142
    D.3 Description of the IMP
    D.3.2Product code HORA-PDE6B (AAV2/5.hPDE6B)
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraocular use
    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) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Yes
    D. cell therapy medicinal product No
    D. therapy medical product Yes
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    Rétinite pigmentaire
    E.1.1.1Medical condition in easily understood language
    Retinitis Pigmentosa
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the safety of a unilateral subretinal administration of HORA-PDE6B in patients harboring mutations in the pde6b gene leading to a defect in PDE6β expression.
    E.2.2Secondary objectives of the trial
    Secondary objectives of this trial are to assess:
    - Efficacy of a unilateral subretinal administration of HORA-PDE6B;
    - Putative humoral and cellular immunological responses following a unilateral subretinal administration of HORA-PDE6b.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients with Retinitis Pigmentosa caused by defect in PDE6B gene without other syndromic manisfestations.
    2. The phenotypic clinical status must be established by:
    a. Disease history,
    b. Presence of characteristic features of retinitis pigmentosa in fundus
    c. Reduction of both rod and cone ERG responses in full field ERG, with predominance of rod involvement, and relative preservation of ERG responses of the fovea in multifocal ERG
    d. Visual acuity ≤ 0.32 for cohort 1 and ≤ 0.63 for cohorts 2 and 3
    3. Each patient will then be genotyped even if a genotyping has already been previously performed.
    4. Each patient will give his/her informed consent, after having been counselled by the investigator, particularly on the high number of visits and on the requirement of hospitalization. For a minor, the investigator will explain the objectives and the course of the clinical trial in order to obtain his/her assent using understandable words appropriate to the age of the child. In total agreement with the decision of the child, the parents will be consecutively asked to sign the informed consent form.
    5. Patients (male or female) with RP aged from 18 years old for the first and second cohorts
    6. Patients (male or female) with RP aged from 6 years old for the third cohort.
    7. Patients still having a residual central retinal function that allows ambulation.
    8. For women with childbearing potential, a negative urine pregnancy test at screening (Visit 1/Day-120 to Day-7) and at visit 2 (Day -1. Women with childbearing potential (i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile) must be using a highlyan effective method of contraception (i.e. combined (estrogen and progestogen containing) hormonal/ progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device, intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomised partner, sexual abstinence. The patient will be requested to continue the contraception for six months after surgery (Visit 9/Day 180)
    9. For males of reproductive potential, agreement to use effective contraception for 6 months after administration of the IMP, for sexual activity that could lead to pregnancy.
    10. Patient affiliated to a health security system
    E.4Principal exclusion criteria
    Exclusion criteria related to co-morbidities
    1. Patients with chronic conditions such as haematological, cardiac, renal diseases.
    2. Patients with, within the past 6 months, a clinically significant cardiac disease on routine clinical examination (history, physical examination), or known congestive heart failure, myocardial infarction, clinically significant valvular heart disease, clinically significant cardiac rhythm or conduction abnormalities, uncontrolled or unstable hypertension.
    3. Patients with pulmonary dysfunction or severe obstructive pulmonary disease that, in the investigator’s judgment, could interfere with the study participation and completion.
    4. Patients with suspected rheumatoid arthritis or any other systemic autoimmune disease.
    5. Patients with active cancer or patients currently undergoing therapy for cancer.
    6. Patients with unstable endocrine disease, including unstable diabetes or thyroid disease.
    7. Patients with alanine transaminase (ALAT), aspartate transaminase (ASAT), or gamma-glutamyl transferase (GGT) > 3x upper limit of normal.
    8. Patients with severe anaemia (haemoglobin < 6 g/dL), leukopenia (while blood cell count <2500/mm3), thrombocytopenia (platelet count < 80.000/mm3), polycytemia (haematocrit> 54% (male) or haematocrit > 49% (female)) or clinically significant coagulopathy.
    Exclusion criteria related to infections or immune-suppression
    9. Patients with known history or clinical diagnosis of herpes zoster or varicella infection within 6 weeks before recruitment or chicken pox exposure within 21 days before recruitment.
    10. Patients with current systemic infection.
    11. Patients with active, extraocular infection requiring the prolonged or chronic use of antimicrobial agents.
    12. Patients with seropositivity for human immunodeficiency virus, Ag-HBS >0, PCR-HCV >0.
    13. Patients using a therapy 3 months before the recruitment that would likely affect immune responses or interfere with trial logistics (steroidal, non-steroidal anti-inflammatory, immunosuppressive and immune-modulatory drugs).
    14. Patients receiving an anti-viral drug treatment 3 months before the recruitment.
    15. Patients receiving a treatment based on a non-specific phosphodiesterase inhibitor 3 months before the recruitment.
    16. Patients participating in another clinical trial with an investigational agent in the 30 days prior to the study participation and/or has not recovered from any reversible effects or side effects prior investigational agent.
    17. Patients enrolled or being enrolled in another gene therapy clinical trial.
    18. Patients with any non-ocular, medically significant co-morbid conditions that impair normal activities, require immunosuppression, or any medical condition that would likely have an impact on the participant’s ability to comply with the study schedule.
    19. Recipients of a solid organ transplant.
    20. Patients currently pregnant or lactating.
    21. Patients with any current or history of substance abuse, psychiatric disorder or a condition that, in the opinion of the investigator, would jeopardize the safety of the subject or impact the validity of the study results.
    Ophthamology-related exclusion criteria
    22. Patients with “monopthalme” (monovisual capacity).
    23. Patients with previous ocular surgery or thermal laser within the past 6 months.
    24. Patients with previous ocular treatment for retinal disease within the past 6 months.
    25. Patients with other ocular pathology, glaucoma, high myopia (> 6 diopters), previous retinal detachment, diabetic retinopathy.
    26. Patients with chronic ocular hypotony (less than 6 mmHg).
    27. Patients with lens opacities or obscured ocular media upon recruitment such reliable evaluation or grading of the posterior segment cannot be performed.
    28. Patients with known serious allergies to the fluorescein dye used in angiography, to the mydriatic, steroidal and non-steroidal eye drops.
    29. Patients who received a vaccination during the month before recruitment and patients who will receive a vaccination within 6 months after recruitment.
    30. Patients who received an anti-acneic medication containing retinoid.
    31. Patients under guardianship, curatorship or legal safeguard.
    E.5 End points
    E.5.1Primary end point(s)
    Primary endpoints will be the assessment of safety parameters : routine ophtalmologic examination, intraocular inflammation, chorioretinal tolerance, questionnaire, vital signs, laboratory measurements
    E.5.1.1Timepoint(s) of evaluation of this end point
    all visits
    E.5.2Secondary end point(s)
    Assessment of improvement of functional tests:
    - A self evaluation of amelioration of quality of life
    - Visual acuity and refraction

    Assessment of improvement of morphologic tests:
    • fundus autofluorescence
    • immunological parameters

    Exploratory endpoints: functional magnetic resonance imaging (fMRI)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Visit 2 and from from visit 6 to the last visit
    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 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) Yes
    E.7.1.1First administration to humans Yes
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E. 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 Yes
    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 Yes
    E. description
    different doses of the same product
    E.8.2.4Number of treatment arms in the trial3
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned 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: 6
    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. of subjects for this age range: 3
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 3
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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 state12
    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)
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2017-07-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-06-20
    P. End of Trial
    P.End of Trial StatusOngoing
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