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    Summary
    EudraCT Number:2019-004547-77
    Sponsor's Protocol Code Number:RN03-CP-0001
    National Competent Authority:UK - MHRA
    Clinical Trial Type:EEA CTA
    Trial Status:GB - no longer in EU/EEA
    Date on which this record was first entered in the EudraCT database:2020-02-26
    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 ConcernedUK - MHRA
    A.2EudraCT number2019-004547-77
    A.3Full title of the trial
    First-in-human Phase I/IIa, Open-Label, Prospective Study of the Safety and Tolerability of Subretinally Transplanted Human Retinal Progenitor Cells (hRPC) in Subjects with Retinitis Pigmentosa (RP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Safety and Tolerability of hRPC in Retinitis Pigmentosa
    A.4.1Sponsor's protocol code numberRN03-CP-0001
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02464436
    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 SponsorReNeuron Ltd
    B.1.3.4CountryUnited Kingdom
    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 supportReNeuron Ltd
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationReNeuron Ltd
    B.5.2Functional name of contact pointRegulatory department
    B.5.3 Address:
    B.5.3.1Street AddressPencoed Business Park
    B.5.3.2Town/ cityPencoed, Bridgend
    B.5.3.3Post codeCF35 5HY
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number02038198400
    B.5.6E-mailRegulatory@reneuron.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/3/13/1140
    D.3 Description of the IMP
    D.3.1Product namehuman Retinal Progenitor Cells Drug Product
    D.3.2Product code hRPC DP
    D.3.4Pharmaceutical form Suspension 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 INNALLOGENEIC NEURAL RETINA-DERIVED HUMAN NEURAL RETINAL PROGENITOR CELLS EX VIVO EXPANDED
    D.3.9.2Current sponsor codeRN03
    D.3.9.3Other descriptive nameALLOGENEIC NEURAL RETINA-DERIVED HUMAN NEURAL RETINAL PROGENITOR CELLS EX VIVO EXPANDED
    D.3.9.4EV Substance CodeSUB197264
    D.3.10 Strength
    D.3.10.1Concentration unit Other
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number40000 to 80000
    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.3.11.3.1Somatic cell therapy medicinal product Yes
    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
    Retinitis Pigmentosa
    E.1.1.1Medical condition in easily understood language
    Retinitis Pigmentosa
    E.1.1.2Therapeutic area Diseases [C] - Eye Diseases [C11]
    MedDRA Classification
    E.1.2 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
    The primary objective of the study is to establish the safety of three different doses of hRPC from a single dose administration.
    E.2.2Secondary objectives of the trial
    The secondary objectives measure changes in visual function in subjects with RP who underwent surgical implantation of hRPC cells.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must satisfy all of the following criteria to be included in the study:
    a) Have ability to give written informed consent as evidenced by signature on the subject consent form
    b) Be adult male or female over 18 years of age
    c) Have clinical diagnosis of RP, based upon one or more of the following: clinical features, medical imaging, electrophysiological measures and genetic testing, if available. Genetic confirmation is not obligatory.
    d) Have Best Corrected ETDRS visual acuity of 35 letters or less (approximately 20/200 or worse) in the study eye for cohorts 1-5; have Best Corrected ETDRS visual acuity of 8 letters (approximately 20/800) to 63 letters (approximately 20/63) in the study eye for cohort 6-8, and Best Corrected ETDRS visual acuity of 8 letters (approximately 20/800) to 68 letters (approximately 20/50) for cohorts 9 and onwards
    e) Be able to complete the entire microperimetry test, and demonstrate adequate fixation and consistency between baseline readings such that the accuracy of both baseline and follow up testing should enable the detection of clinically significant changes in retinal sensitivity.
    f) Be medically able to undergo vitrectomy and subretinal injection.
    g) Have good general health as defined by:
    i. Normal serum chemistry and hematology. Out of normal range laboratory findings deemed not clinically significant (at the discretion of the Investigator) are acceptable.
    ii. No history of malignancy, except non-melanoma skin cancer; pre-malignant conditions and cancer in situ.
    iii. Negative serology for human immunodeficiency virus (HIV), hepatitis B (HBV), hepatitis C (HCV)
    iv. Medically fit enough to undergo surgery which may require general anesthesia as well as medically fit to undergo a short perioperative course of systemic corticosteroid therapy
    v. Free of any other systemic condition that in the opinion of the Investigator may have an impact on the safety of the subject, conduct of study procedures, or integrity of study data (e.g. severe cardiovascular or respiratory disease; poorly controlled diabetes; significant psychiatric impairment)
    h) Females of childbearing potential must have a confirmed negative pregnancy test at Visits 1 and 3 and be willing to use acceptable method of contraception (e.g. oral contraceptive and condom, intra-uterine device (IUD) and condom, diaphragm with spermicide and condom) for the duration of this study
    i) Males must be willing to use to use an acceptable method of contraception (e.g. barrier and spermicide) for the duration of this study; unless they have been surgically sterilized with confirmed azoospermia.
    j) Be willing and able to attend all scheduled clinical assessments, ability to communicate well with the Investigator and to comply with the expectations of the study.
    E.4Principal exclusion criteria
    Subjects must not have any of the following criteria:
    a) Exhibits a difference in ETDRS BCVA of 15 letters of more in either eye between any of the baseline visits.
    b) Exhibits a difference in ETDRS BCVA of 20 or more letters between eyes at the time of any of the screening or baseline visits attributed to asymmetry in the progression of RP.
    c) Presence of ocular disease or ocular media opacity in the study eye, which in the opinion of the Investigator, will preclude an accurate evaluation at any time during the study.
    d) History of any retinal and/or macular disease other than RP (e.g. retinal detachment) that in the opinion of the Investigator may have an impact on the safety of the subject, conduct of study procedures, or integrity of study data. Specifically, subjects in whom significant pre-existing vitreoretinal pathology might influence visual acuity outcomes should be excluded.
    e) Active ocular infection or inflammation, or any history of intraocular inflammation, that would expose subject to risk during or following surgery
    f) Prior vitrectomy in the study eye
    g) A history of amblyopia in the study eye
    h) High myopia (>6 diopters) in the study eye
    i) Cataract surgery in the study eye or ocular surgery in either eye (which in the opinion of the investigator may have an impact on patient safety or the integrity of data from the study eye) during the study or within 3 months prior to treatment.
    j) Participation in any clinical study involving an investigational drug or device within 6 months prior to treatment or 5 half-lives of the drug (whichever is longer) prior to initiation of treatment
    k) Prior stem cell administration or injections to any part of the body (subjects who have received autologous stem cell transplant will be eligible)
    l) Use of systemic immunosuppressive agents (e.g. corticosteroid) in the 6 months prior to treatment or 5 half-lives of the drug (whichever is longer) prior to initiation of treatment (Note: inhaled, intranasal, and/or topical dermatologic steroids are allowed.)
    m) For females, be breastfeeding or planning a pregnancy
    n) Known hypersensitivity to any of ingredients of the excipients
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is safety over the six months after treatment as assessed by the incidence of treatment emergent adverse events and changes from baseline in other safety parameters. Safety measures will be assessed by review of important events, including but not limited to inflammation, complications of the surgical procedure and worsening of vision.
    Anatomical endpoints related to transplant integrity, survival and surgical complications will be measured using:
     Color fundus photography
     Fundus autofluorescence
     Spectral domain-OCT (SD-OCT)
    E.5.1.1Timepoint(s) of evaluation of this end point
    6 months after treatment
    E.5.2Secondary end point(s)
    Secondary safety endpoints:
    There are no specific reference ranges or quantitative changes that are recognised in grading or following RP. Therefore the Principal Investigator will review the data on each of the following tests and assess whether any changes are clinically meaningful and/or may constitute an improvement or adverse event.

    Measures of change in visual function:
    1. Change in visual acuity
    A summary of the ETDRS +/- BRVT BCVA letter score and the change from baseline to end of study (24 months) in the treated eye presented by treatment group.

    2. Change in visual field: Goldmann visual field, microperimetry and FST
    A summary of the perimetry and change from baseline to end of study (24 months) in the treated eye presented by treatment group.

    3. Change in retinal sensitivity in the area overlying the implanted hRPC as compared with untreated retina.
    ERG results and change from baseline to end of study (24 months) summarised descriptively and presented by treatment group.

    Anatomical endpoints relating to retinal function in implant location:
    1. Color Fundus photography
    A qualitative description of the change in retinal appearance of treated and untreated retinal area in the treated eye from baseline to end of study (24 months) presented by treatment group.

    2. Fundus autofluorescence
    A qualitative description of the change in retinal appearance of treated and untreated retinal area from baseline to end of study (24 months) presented by treatment group.

    3. Spectral domain-OCT (SD-OCT)
    Summary of the overall retinal thickness and change from baseline to the end of study (24 months) of treated retina compared with untreated retina in the treated eye by treatment group.

    Summary of the outer nuclear layer thickness and change from baseline to the end of study (24 months) of treated retina compared with untreated retina in the treated eye by treatment group.

    Summary of the ellipsoid zone measurement and change from baseline to the end of study (24 months) of treated retina compared with untreated retina in the treated eye by treatment group, if present.

    Signal strength will be summarised by treatment group and visit as a measure of quality of the OCT parameter estimates.

    Treatment-emergent adverse events (TEAEs) will be summarised for each treatment group. Incidence will be tabulated by MedDRA system organ class (SOC), preferred term (PT), and treatment group. Events will also be summarised by relationship to study drug, severity, seriousness and for AEs leading to study discontinuation.

    Clinical and ocular testing (i.e., laboratory testing, vital signs, physical examinations, ECGs, ocular slit lamp and dilated fundus examination) will be summarised at each visit and presented by treatment group.
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 months after treatment
    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 Yes
    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) 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.8.2.4Number of treatment arms in the trial1
    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 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
    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 years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    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 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: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 13
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 6
    F.4.2.2In the whole clinical trial 33
    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 participants will be monitored for up to 24 months post-treatment with hRPC. There are no further plans for provision of hRPC after patients have ended their participation in the trial.
    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 Decision2020-04-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-06-09
    P. End of Trial
    P.End of Trial StatusGB - no longer in EU/EEA
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