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    Summary
    EudraCT Number:2014-000385-22
    Sponsor's Protocol Code Number:RF-2010-2318561
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2014-05-12
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2014-000385-22
    A.3Full title of the trial
    Nerve Growth Factor eye drops as a novel treatment for vision loss in patients with Retinitis Pigmentosa: from preclinical to
    clinical Phase II trial.
    Nerve Growth Factor in collirio come trattamento innovativo per la perdita visiva nei pazienti con retinite pigmentosa: dalla sperimentazione preclinica allo studio clinico di fase II.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Nerve Growth Factor eye drops as a novel treatment for patients with Retinitis Pigmentosa.
    A.3.2Name or abbreviated title of the trial where available
    NEMO
    A.4.1Sponsor's protocol code numberRF-2010-2318561
    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 SponsorOspedale San Raffaele di Milano
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportItalian Ministry of Health
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOspedale San Raffaele di Milano
    B.5.2Functional name of contact pointCornea and Ocular surface unit
    B.5.3 Address:
    B.5.3.1Street Addressvia olgettina, 60
    B.5.3.2Town/ cityMilan
    B.5.3.3Post code20132
    B.5.3.4CountryItaly
    B.5.6E-mailcorcione.giuditta@hsr.it
    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/3/13I1135
    D.3 Description of the IMP
    D.3.1Product namerecombinant human Nerve Growth Factor (rhNGF)
    D.3.4Pharmaceutical form Eye drops, solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOcular use
    Topical use (Noncurrent)
    Ophthalmic use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNnot yet assigned
    D.3.9.3Other descriptive nameRECOMBINANT HUMAN NERVE GROWTH FACTOR (RHNGF)
    D.3.9.4EV Substance CodeSUB77552
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.18
    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) 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 Yes
    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 placeboEye drops, solution
    D.8.4Route of administration of the placeboOcular use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Retinitis pigmentosa with cystoid macular edema
    E.1.1.1Medical condition in easily understood language
    Retinitis pigmentosa with macular edema
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10054467
    E.1.2Term Macular edema
    E.1.2System Organ Class 100000004853
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    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
    The primary objective of the study is to assess the efficacy of rhNGF eye drops solution administered over 1 month versus a vehicle control in patients with macular edema associated with retinitis pigmentosa
    E.2.2Secondary objectives of the trial
    The secondary objective of this study is to assess safety and tolerability of hNGF eye drops solution administered over 1 month versus a vehicle control in patients with macular edema associated with retinitis pigmentosa
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Patients between 18 and 60 years of age.
    2. Patients with typical orms of RP characterized by the following
    clinical features:
    a) classic fundus appearance (i.e. intraretinal pigment deposits,thinning and atrophy of the RPE in the mid- and far peripheral retina, with relative RPE preservation in the macula, waxy
    pallor of the optic disc, attenuation of the retinal vessels)
    b) reduced and delayed ERG responses
    c) visual field constriction
    and/or atypical forms of RP:
    The atypical form of RP is characterized by early onset (first decade) or syndromic forms of RP (eg paravenous sector pericentral or unilateral RP, Leber congenital amaurosis, Resfum disease, Usher syndrome, Bardet - Biedl, etc. ).
    3. presence of macular edema documented by OCT (macular thickness greater than 250 um) lasting for at least 3 months and not in treatment for at least 1 month;
    4 Best corrected distance visual acuity (BCDVA) between 20/100 (2/10 or 0.1 logMAR) and 20/25 (8/10 or +0.7 logMAR) , near vision lower than second character in either eye.
    5. Absence of other confounding ocular diseases.
    6. Only patients who satisfy all Informed Consent requirements may be
    included in the study. The patient and/or his/her impartial witness must
    read, sign and date the Informed Consent document before any
    studyrelated procedures are performed. The Informed Consent form signed by patients and/or impartial witness must have been approved by the IEC for the current study.
    7. Patients must have the ability and willingness to comply with study
    procedures.
    E.4Principal exclusion criteria
    1. Presence of macular edema associated with diabetes , choroidal neovascularization or after eye surgery.
    2 . Patients with diabetes mellitus
    3 . History of any ocular surgery (including laser or refractive surgical
    procedures) in either eye within the 90 days before study enrolment.
    Ocular surgery will not be allowed during the study treatment period and
    elective ocular surgery procedure.
    4. Evidence of an active ocular infection.
    5. History of uveitis or evidence of intraocular inflammation in either eye.
    6. History or evidence of glaucoma or an IOP greater than or equal 21
    mmHg in either eye at the time of study enrollment
    7. Anterior segment abnormalities or media opacities obscuring the view
    of the posterior pole in either eye.
    8. Treatment with corticosteroids (systemic, periocular or intravitreal)
    ) in either eye within 90 days of study enrollment.
    9. Use of any topical medication other than the study medication for the
    treatment of ocular diseases with the exception of artificial tears during the study period.
    10. Presence or history of any ocular or systemic disorder or condition
    that might significantly limit visual acuity or the visual field, hinder the
    efficacy of the study treatment or its evaluation, could possibly interfere
    with the interpretation of study results, or could be judged by the investigator to be incompatible with the study visit schedule or conduct of trail procedures.
    11. Known hypersensitivity to one of the components of the study or
    procedural medications.
    12. Participation in another clinical study at the same time as the present study or within 90 days of screening/baseline visit.
    13. History of drug, medication or alcohol abuse or addiction.
    14. Females of childbearing potential (those who are not surgically
    sterilized or post-menopausal for at least 1 year) are excluded from participation in the study if they meet any one of the following conditions:
    a. are currently pregnant or,
    b. have a positive result on the urine pregnancy test at the Screening/Baseline Visit or,
    c. intend to become pregnant during the study treatment period or,
    d. are breast-feeding or,
    e. not willing to use highly effective birth control measures, such as:
    Hormonal contraceptives – oral, implanted, transdermal, or injected
    and/or mechanical barrier methods – spermicide in conjunction with a
    barrier such as a condom or diaphragm or IUD during the entire course
    of and 30 days after the study treatment periods.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint will be to evaluate the effects of NGF eye drops on macular thickness in patients with RP associated with macular edema. Macular thickness greater than 250μm will be considered significant. Treatment will be considered effective if it will induce an improvement of at least one of the following parameters: 1) macular thickness less than 250μm; 2) visual acuity of at least 20 EDTRS letters; 3) near visual acuity of at least 1 line
    E.5.1.1Timepoint(s) of evaluation of this end point
    after one month of experimental treatment
    E.5.2Secondary end point(s)
    The secondary endpoints of this study are to evaluate differences between the two treatment groups of the following parameters:
    - Best-corrected visual acuity for distance
    - best-corrected visual acuity for near
    -contrast sensitivity
    - visual-field
    -OCT
    - ERG
    -Quality of Life (NEI-VFQ)
    - Number of drop out for inefficacy
    -number of recurrence of macular edema
    E.5.2.1Timepoint(s) of evaluation of this end point
    secondary endpoints will be evaluated at the end of experimental treatment (1 month) and at 3, 6 and 12 months of follow-up
    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) 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) 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 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 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 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 No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned2
    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 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 years1
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days
    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: 45
    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 state45
    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)
    None
    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 Decision2014-10-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-07-17
    P. End of Trial
    P.End of Trial StatusOngoing
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