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    Summary
    EudraCT Number:2017-003260-12
    Sponsor's Protocol Code Number:NTMT-03-B
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2017-10-09
    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 ConcernedGermany - PEI
    A.2EudraCT number2017-003260-12
    A.3Full title of the trial
    A Phase 3 Multicenter Randomized, Sham-Controlled Study to Determine the Safety and Efficacy of NT-501 in Macular Telangiectasia type 2
    Eine multizentrische randomerisierte, Scheineingriff-kontrollierte Phase-III-Studie zur Bestimmung der Sicherheit und Wirksamkeit von NT-501 bei makulärer Teleangiektasie Typ 2
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A phase III clinical trial to determine the safety and effectiveness of
    NT-501, an implant in the eye shown to reduce the rate of progression of Macular Telangiectasia type 2
    A.3.2Name or abbreviated title of the trial where available
    NTMT-03-B
    A.4.1Sponsor's protocol code numberNTMT-03-B
    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 SponsorNeurotech Pharmaceuticals Inc.
    B.1.3.4CountryUnited States
    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 supportNeurotech Pharmaceuticals Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationNeurotech Pharmaceuticals Inc
    B.5.2Functional name of contact pointJenni Bursell
    B.5.3 Address:
    B.5.3.1Street Address900 Highland Corporate Drive, Suite 101
    B.5.3.2Town/ cityCumberland RI
    B.5.3.3Post code02864
    B.5.3.4CountryUnited States
    B.5.4Telephone number+14014952388
    B.5.5Fax number+14013333881
    B.5.6E-mailj.bursell@neurotechusa.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 numberEMA/OD/160/11
    D.3 Description of the IMP
    D.3.1Product nameNT-501
    D.3.2Product code NT501.6A.02
    D.3.4Pharmaceutical form Implant
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntraocular use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCiliary Neurotrophic factor (CNTF)
    D.3.9.2Current sponsor codeNT501.6A.02
    D.3.10 Strength
    D.3.10.1Concentration unit ng nanogram(s)
    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 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 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) Yes
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Yes
    D.3.11.3.5.1CAT classification and reference numberEMA/CAT/570019/2012
    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
    Macular Telangiectasia type 2
    E.1.1.1Medical condition in easily understood language
    Macular Telangiectasia (MacTel) is a disease affecting the macula, causing loss of central vision. MacTel develops when there are problems with the tiny blood vessels around the fovea.
    E.1.1.2Therapeutic area Body processes [G] - Ocular Physiological Phenomena [G14]
    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
    The overall objective of this study is to evaluate the efficacy and safety of NT-501 for the treatment of MacTel.

    Primary objective:
    • To determine the rate of change in the ellipsoid zone (EZ; inner segment/outer segment [IS/OS]) area loss over 24 months, as measured by study eye spectral-domain optical coherence tomography (SD-OCT) in participants with MacTel
    E.2.2Secondary objectives of the trial
    The secondary objective of this study is to evaluate the safety of NT-501 in patients with MacTel Type 2.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    To participate in this study, the potential participant and at least 1 of their eyes must meet all of the following criteria:

    1. Participant must have at least 1 study eye with a positive diagnosis of MacTel with evidence of fluorescein leakage typical of MacTel and at least one of the other features that include hyperpigmentation that is outside of a 500 micron radius from the center of the fovea, retinal opacification, crystalline deposits, right-angle vessels, or inner/outer lamellar cavities
    2. Participant must have an IS/OS PR break and EZ (area of IS/OS loss) as measured by SD OCT between 0.16 and 2.00 mm2
    3. Participant’s best-corrected visual acuity (BCVA) is 54-letter score or better (20/80 or better) as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) chart at screening
    4. Participant must have steady fixation in the foveal or parafoveal area and sufficiently clear media for good quality photographs
    5. Participant must be greater than 21 years of age or less than 80 years of age at screening
    6. Participant must be able to provide written informed consent to participate in the study, in accordance with the International Conference on Harmonisation Good Clinical Practices guidelines, and local regulations, before initiating any study related procedures
    7. Women of childbearing potential must agree to use highly effective contraception (Germany and France only)
    E.4Principal exclusion criteria
    To participate in this study, the potential participant must not meet any of the following criteria. The ocular exclusion criteria are related to the study eye (unless indicated for either eye):

    1. Participant is medically unable to comply with study procedures or follow-up visits
    2. Participant received intravitreal steroid therapy for non neovascular MacTel within the last 3 months
    3. Participant has ever received intravitreal anti-vascular endothelial growth factor (VEGF) therapy in the study eye OR has, within the past 3 months, received intravitreal anti VEGF therapy in the fellow eye at randomization
    4. Participant has evidence of ocular disease other than MacTel that, in the judgment of the examining physician, may confound the diagnosis, procedures, or outcome of the study (eg, glaucoma, severe nonproliferative or proliferative diabetic retinopathy, uveitis)
    5. Participant has a chronic requirement (eg, ≥ 4 weeks at a time) for ocular medications and/or has a diagnosed disease that, in the judgment of the examining physician, may be vision threatening or may affect the primary outcome (artificial tears are permitted)
    6. Participant has evidence of intraretinal neovascularization or subretinal neovascularization (SRNV), as evidenced by hemorrhage, hard exudate, subretinal fluid or intraretinal fluid in either eye
    7. Participant has evidence of central serous chorio-retinopathy in either eye
    8. Participant has evidence of pathologic myopia in either eye
    9. Participant has significant corneal or media opacities in either eye
    10. Participant has had a vitrectomy, penetrating keratoplasty, trabeculectomy, or trabeculoplasty
    11. Participant has any of the following lens opacities: cortical opacity > standard 3, posterior subcapsular opacity > standard 2, or a nuclear opacity > standard 3 as measured on the Age Related Eye Disease Study (AREDS) clinical lens grading system
    12. Participant has undergone lens removal in the previous 3 months or YAG laser within 4 weeks
    13. Participant was a participant in any other clinical trial of an intervention (drug or device) within the last 6 months
    14. Participant is on chemotherapy
    15. Participant is pregnant or breastfeeding
    16. Participant has a history of malignancy that would compromise the 24-month study survival
    17. Participant with a history of ocular herpes virus in either eye
    18. Participant has, in the opinion of the investigator, any physical or mental condition that would increase the risk of participation in the study or may interfere with the study procedures, evaluations, and outcome assessments
    19. Participant has evidence of intraretinal hyperreflectivity by OCT
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint will be the rate of change in the EZ (IS/OS) area loss over 24 months, as measured by study eye SD-OCT in participants with MacTel
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline, day 1, week 1 and 1, 3, 6, 12, 16, 20, and 24 months
    E.5.2Secondary end point(s)
    Secondary efficacy end point:

    - Mean change in aggregate sensitivity of microperimetry within the EZ line break area through 24 months
    - Mean change in reading speed from baseline through 24 months
    - Mean change in the National Eye Institute Visual Function Questionnaire (NEI-VFQ-25) near activities subscale score from baseline through 24 months

    Secondary Safety Endpoint:
    - Number and proportion of participants with a loss in BCVA of 15 or more letters from baseline in the study eye using the Early Treatment Diabetic Retinopathy Study (ETDRS) distance chart
    Number and proportion of participants with at least 1 treatment emergent serious adverse event
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline, day 1, week 1 and 1, 3, 6, 12, 16, 20, and 24 months
    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) 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 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.8.2.3.1Comparator description
    sham implant
    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 concerned3
    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
    Australia
    United States
    France
    United Kingdom
    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: 56
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 56
    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 state56
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 56
    F.4.2.2In the whole clinical trial 112
    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)
    Upon completion of the 24 month observation all participants will be followed for safety and disease progression in a nested substudy of the existing Natural History and Observation and Registry Study of Macular Telangiectasia Type 2 (NHOR).
    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 Decision2018-04-30
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-02
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-08-03
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