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    Summary
    EudraCT Number:2022-000237-17
    Sponsor's Protocol Code Number:NL-80249
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2022-06-03
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2022-000237-17
    A.3Full title of the trial
    Prospective clinical trial to evaluate the efficacy of acetazolamide for the treatment of cystoid macular edema in inherited retinal dystrophies: the CAR trial
    Prospectieve klinische studie om de werkzaamheid van acetazolamide te evalueren voor de behandeling van cystoïd macula-oedeem bij erfelijke retinale dystrofieën: de CAR studie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    The CAR trial for nherited retinal dystrophies
    De CAR studie voor erfelijke retinale dystrofieën
    A.3.2Name or abbreviated title of the trial where available
    The CAR trial
    de CAR studie
    A.4.1Sponsor's protocol code numberNL-80249
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    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 Yes
    D.2.1.1.1Trade name Diamox
    D.2.1.1.2Name of the Marketing Authorisation holderAmdipharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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.3.4Pharmaceutical form
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNAcetazolamide
    D.3.9.1CAS number 59-66-5
    D.3.9.4EV Substance CodeSUB05219MIG
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    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) 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 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 Yes
    D.3.11.13.1Other medicinal product typea sulphonamide derivative
    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
    Cystoid Macular Edema in inherited retinal dystrophies
    Cystoid Macula-oedeem in erfelijke retinale dystrofieën
    E.1.1.1Medical condition in easily understood language
    accumulation of fluid in cyst-like spaces (cystoid macular edema) in the retina
    ophoping van vocht in cyste-achtige ruimten (cystoïde macula-oedeem) in het netvlies.
    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
    Our study objective is to evaluate the efficacy of acetazolamide for the treatment of cystoid macula edema in inherited retinal dystrophies in anticipation of future clinical trials.
    Het primaire doel van dit onderzoek is om de werkzaamheid van acetazolamide te evalueren voor de behandeling van cystoïde macula-oedeem bij erfelijke retinale dystrofieën in afwachting van toekomstige klinische onderzoeken.
    E.2.2Secondary objectives of the trial
    Secondary study endpoints include the following compared within the treated group and compared to control group:
    1) To determine the optimal acetazolamide dose for maximum effect on CME and minimal side effects
    2) To determine the intra- and inter individual variability in such treatment– and side effects
    3) To determine the proportion of IRD patients with CME in which acetazolamide treatment is able to completely resolve CME for a spectrum of different IRD-associated genes
    Secundaire onderzoekvariabelen omvatten de volgende, vergeleken binnen de behandelde groep met de controlegroep:
    1) De optimale dosis acetazolamide bepalen voor een maximaal effect op CME en minimale bijwerkingen
    2) De intra- en interindividuele variabiliteit en bijwerkingen bepalen
    3) Om het percentage IRD-patiënten met CME te bepalen waarbij behandeling met acetazolamide CME volledig kan oplossen voor een spectrum van verschillende IRD-geassocieerde genen
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Eligible patients received a clinical diagnosis of IRD, and underwent at least one clinical examination, in combination with one of the following prerequisites:
    • IRD associated with causal genetic variant(s) (in e.g., USH2A, CRB1, RHO, RP1, RP2, RPGR, PRPF31, or RS1 gene)
    • CME involving the fovea confirmed on spectral-domain optical coherence tomography (OCT)
    In aanmerking komende patiënten hebben een klinische diagnose van IRD en ondergingen ten minste één klinisch onderzoek, in combinatie met een van de volgende voorwaarden:
    • IRD geassocieerd met causale genetische variant(en) (in bijv. USH2A-, CRB1-, RHO-, RP1-, RP2- RPGR-, PRPF31- of RS1-gen)
    • CME met betrekking tot de fovea bevestigd op spectraaldomein optische coherentietomografie (OCT)
    E.4Principal exclusion criteria
    A potential subject who meets any of the following criteria will be excluded from participation in this study:
    • Eyes will be excluded when the visual dysfunction is also significantly associated with other ocular diseases besides the IRDs (e.g., glaucoma, perforating trauma).
    • Patients treated with loop diuretics
    • Severe hepatic impairment
    • Severe renal insufficiency
    • Sodium and Potassium Depletion
    • Addison's disease
    • Hyperchloremic Acidosis
    • Cor pulmonale
    • Chronic non-congestive angle-closure glaucoma
    • The use of Acetazolamide
    Een potentiële proefpersoon die aan een van de volgende criteria voldoet, wordt uitgesloten van deelname aan dit onderzoek:
    • Ogen worden uitgesloten wanneer de visuele disfunctie ook significant geassocieerd is met andere oogziekten dan de IRD's (bijv. glaucoom, perforerend trauma).
    • Patiënten behandeld met lisdiuretica
    • Ernstige leverfunctiestoornis
    • Ernstige nierinsufficiëntie
    • Natrium- en kaliumdepletie
    • De ziekte van Addison
    • Hyperchloremische acidose
    • Cor pulmonale
    • Chronisch niet-congestief geslotenhoekglaucoom
    • Het gebruik van Acetazolamide
    E.5 End points
    E.5.1Primary end point(s)
    The main endpoint is the to determine the effective dosage, the outcome of the treatment, and the effect on the visual acuity. As such, these findings will have immediate impact on translational scientific progress, by applying cutting-edge multidisciplinary technology to facilitate patient identification and selection for novel treatments.
    Het belangrijkste eindpunt is het bepalen van de effectieve dosering, het resultaat van de behandeling en het effect op de gezichtsscherpte. Als zodanig zullen deze bevindingen een onmiddellijke impact hebben op de wetenschappelijke vooruitgang door de toepassing van geavanceerde multidisciplinaire technologie om de identificatie en selectie van patiënten voor nieuwe behandelingen te vergemakkelijken.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Investigator-initiated, single-center, prospective, experimental study consisting of seven visits at 2, 4, 8, 12, 16, and 32 weeks after baseline evaluation visit. During each visit participants will perform several ophthalmological measurements
    Het voorgestelde onderzoek is investigator-initiated, monocenter, prospectieve, experimenteel onderzoek, bestaande uit zeven bezoeken op 2, 4, 12, 16 en 32 weken na het baseline-evaluatiebezoek. Tijdens elk bezoek zullen de deelnemers verschillende oogheelkundige metingen uitvoeren.
    E.5.2Secondary end point(s)
    is there rebound after halving the dosis?
    is er een rebound na het halveren van de dosis?
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 16
    week 16
    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 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) 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 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 Yes
    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
    geen behandeling
    no treatment
    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 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
    laaste bezoek is het eind van de studie
    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 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 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) No
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 6
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 state18
    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
    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 Decision2022-12-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-05-31
    P. End of Trial
    P.End of Trial StatusOngoing
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