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    Summary
    EudraCT Number:2020-004494-41
    Sponsor's Protocol Code Number:CLIN2001UCM301
    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:2021-08-30
    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 number2020-004494-41
    A.3Full title of the trial
    Multicenter Phase 3 Pivotal Study to Evaluate the Safety and Efficacy of TOOKAD (padeliporfin) Vascular Targeted Photodynamic Therapy in the Treatment of Low Grade Upper Tract Urothelial Cancer
    Studio multicentrico pivotal di fase 3 per valutare la sicurezza e l’efficacia della terapia fotodinamica vascolare mirata con TOOKAD (padeliporfina) nel trattamento del carcinoma uroteliale del tratto superiore a basso grado
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This is a study that will evaluate the safety and efficacy of TOOKAD® (padeliporfin) Vascular Targeted Photodynamic Therapy (VTP) in the treatment of Low Grade Upper Tract Urothelial Cancer (UTUC).
    Lo studio valuterà la sicurezza e l'efficacia della terapia fotodinamica vascolare mirata (VTP) TOOKAD® (padeliporfin) nel trattamento del carcinoma uroteliale del tratto superiore a basso grado (UTUC).
    A.3.2Name or abbreviated title of the trial where available
    ENdoluminal LIGHT ActivatED Treatment of Upper Tract Urothelial Cancer (ENLIGHTED) Study
    Studio del trattamento del carcinoma uroteriale del tratto superiore attivato dalla luce endoluminal
    A.4.1Sponsor's protocol code numberCLIN2001UCM301
    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 SponsorSTEBA BIOTECH S.A.
    B.1.3.4CountryLuxembourg
    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 supportSteba Biotech, S.A.
    B.4.2CountryLuxembourg
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationSTEBA Biotech S.A.
    B.5.2Functional name of contact pointHead of Global R&D
    B.5.3 Address:
    B.5.3.1Street Address7 place du Théâtre
    B.5.3.2Town/ cityLuxembourg
    B.5.3.3Post codeL-2613
    B.5.3.4CountryLuxembourg
    B.5.4Telephone number00436642034173
    B.5.6E-maild.perry@stebabiotech.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 Yes
    D.2.1.1.1Trade name TOOKAD®
    D.2.1.1.2Name of the Marketing Authorisation holderSteba Biotech S.A.
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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.3.1Product nameTOOKAD®
    D.3.2Product code [PRD5572518]
    D.3.4Pharmaceutical form Powder for solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 886845-72-3
    D.3.9.2Current sponsor codeWST11
    D.3.9.4EV Substance CodeSUB31318
    D.3.10 Strength
    D.3.10.1Concentration unit µg/ml microgram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number9150
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Yes
    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
    Low Grade Upper Tract Urothelial Cancer
    Carcinoma Uroteriale del tratto superiore a basso grado
    E.1.1.1Medical condition in easily understood language
    Urinary tract cancers
    Carcinoma del tratto uroteriale
    E.1.1.2Therapeutic area Diseases [C] - Cancer [C04]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10077840
    E.1.2Term Urothelial cancer of renal pelvis
    E.1.2System Organ Class 100000004864
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To demonstrate the efficacy and durability of effect following TOOKAD (padeliporfin) VTP on low grade UTUC tumors in the calyces, renal pelvis and ureter
    Dimostrare l'efficacia e la durata dell'effetto in seguito al TOOKAD (padeliporfin) VTP sui tumori UTUC di basso grado nei calici, nella pelvi renale e nell'uretere
    E.2.2Secondary objectives of the trial
    -To evaluate TOOKAD (padeliporfin) VTP related safety and tolerability in the treatment of low grade UTUC tumors in the calyces, renal pelvis and ureter
    -To explore a potential link between UTUC cancer genomic markers and treatment related clinical outcomes and disease progression
    -Valutare la sicurezza e la tollerabilità di TOOKAD (padeliporfin) VTP nel trattamento dei tumori UTUC di basso grado nei calici, nella pelvi renale e uretere
    -Esplorare un potenziale legame tra i marcatori genomici del tumore UTUC e risultati clinici legati al trattamento e progressione della malattia
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: To evaluate the pharmacokinetic profile in UTUC patients with moderate hepatic impairment.

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Valutare il profilo farmacocinetico in pazienti UTUC con moderata insufficienza epatica.
    E.3Principal inclusion criteria
    1.Male and female patients 18 years or older
    2.Able to understand and provide written informed consent and willing to comply with all tests and procedures associated with the study
    3.New or recurrent low-grade, non-invasive UTUC disease
    4.Biopsy-proven disease. A concurrence of the central pathology reader will be required for eligibility.
    5.Up to 2 biopsy-proven sites of low-grade involvement with the largest tumor (index tumor) between 5 mm and 15 mm in diameter (as measured by endoscopy), both located in the calyces, renal pelvis or in the ureter of ipsilateral kidney, with an absence of high-grade cells on cytology. (Ureter involvement should be in one anatomical location with no more than 20 mm of contiguous ureteral length).
    6.Karnofsky Performance Status = 50%
    7.Adequate organ function defined at baseline as:
    -ANC =1,000/ µl,
    -Platelets =75,000/ µl, Hb =9 g/dl,
    -INR =2
    -Estimated glomerular filtration rate eGFR =30 ml/min using CKD-EPI Method
    -Total serum bilirubin <3 mg/dL, AST/ALT =5× upper limit of normal
    1.Pazienti maschi e femmine di 18 anni o più
    2. In grado di comprendere e fornire il consenso informato scritto e disposti a rispettare tutti i test e le procedure associate allo studio
    3. Malattia UTUC non invasiva di basso grado, nuova o ricorrente
    4. Malattia biopsia-provata. Un accordo del lettore centrale di patologia sarà richiesto per l'ammissibilità.
    5.Fino a 2 siti biopticamente provati di coinvolgimento di basso grado con il tumore più grande (tumore indice) tra 5 mm e 15 mm di diametro (come misurato dall'endoscopia), entrambi situati nei calici, nella pelvi renale o nell'uretere del rene omolaterale, con assenza di cellule di alto grado all'esame citologico. (Il coinvolgimento dell'uretere deve essere in una sola posizione anatomica con non più di 20 mm di lunghezza ureterale contigua).
    6.Karnofsky Performance Status = 50%
    7.Funzione d'organo adeguata definita al basale come:
    -ANC =1.000/ µl,
    -Piastrine =75.000/ µl, Hb =9 g/dl,
    -INR =2
    -Tasso stimato di filtrazione glomerulare eGFR =30 ml/min usando il metodo CKD-EPI
    Bilirubina sierica totale <3 mg/dl, AST/ALT =5× limite superiore del normale
    E.4Principal exclusion criteria
    1.Current high-grade or muscle invasive (>pT1) urothelial carcinoma of the bladder
    2.Carcinoma in situ (CIS) current or previous in the upper urinary tract
    3.History of invasive T2 or higher urothelial cancer in past 2 years
    4.Participation in another clinical study involving an investigational product within 1 month before study entry
    5.BCG or local chemotherapy treatment in the upper urinary tract within 2 months prior to inclusion
    6.Systemic chemotherapy treatment within 2 months prior to enrollment
    7.Prohibited medication that could not be adjusted or discontinued prior to study treatment
    8.Any other medical or psychiatric co-morbidities, including decompensated heart failure, unstable angina or coronary artery disease, severe pulmonary or liver disease or current heavy smoker that, in the opinion of the study investigator, would make the patient a poor candidate for the study
    9.Pregnant or breast-feeding women Women of childbearing potential (WOCBP) must undergo a negative serum pregnancy test prior to study entry.
    10.Men and women of reproductive potential not willing to observe conventional and effective birth control for the duration of treatment and for 90 days following the last TOOKAD VTP treatment
    1. Carcinoma uroteliale della vescica attuale di alto grado o invasivo (>pT1)
    2. Carcinoma in situ (CIS) attuale o precedente nel tratto urinario superiore
    3. Storia di cancro uroteliale invasivo T2 o superiore negli ultimi 2 anni
    4. Partecipazione a un altro studio clinico che coinvolge un prodotto in fase di sperimentazione entro 1 mese prima dell'ingresso nello studio
    5.Trattamento con BCG o chemioterapia locale nel tratto urinario superiore nei 2 mesi precedenti l'inclusione
    6.Trattamento chemioterapico sistemico entro 2 mesi prima dell'arruolamento
    7.Farmaci proibiti che non potevano essere regolati o sospesi prima del trattamento dello studio
    8.Qualsiasi altra co-morbidità medica o psichiatrica, tra cui insufficienza cardiaca scompensata, angina instabile o malattia coronarica, grave malattia polmonare o epatica o fumatore accanito che, secondo l'opinione del ricercatore dello studio, renderebbe il paziente un cattivo candidato per lo studio
    9. Donne incinte o che allattano Le donne in età fertile (WOCBP) devono sottoporsi ad un test di gravidanza negativo prima dell'inizio dello studio.
    10.Uomini e donne con potenziale riproduttivo non disposti ad osservare un controllo delle nascite convenzionale ed efficace per la durata del trattamento e per i 90 giorni successivi all'ultimo trattamento con TOOKAD VTP
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy outcome is the absence of UTUC tumors in the entire ipsilateral calyces, renal pelvis and ureter on endoscopic evaluation. This outcome will be determined dichotomously as either failure or success in achieving complete response.
    Complete Response will be defined as absence of disease based on:
    •absence of visual tumor on endoscopy
    •no evidence of tumor on biopsy (if feasible)
    •negative urinary cytology by instrumented collection
    Il risultato primario di efficacia è l'assenza di tumori UTUC nell'intero calice omolaterale, nella pelvi renale e nell'uretere alla valutazione endoscopica. Questo risultato sarà determinato dicotomicamente come fallimento o successo nel raggiungimento della risposta completa.
    La risposta completa sarà definita come assenza di malattia basata su:
    -assenza di tumore visibile all'endoscopia
    -nessuna evidenza di tumore alla biopsia (se possibile)
    -citologia urinaria negativa con prelievo strumentale
    E.5.1.1Timepoint(s) of evaluation of this end point
    This endpoint will be evaluated at the time of Primary Response Evaluation (PRE) (28 +/- 3 days post last treatment) during the TOOKAD (padeliporfin) VTP induction treatment phase.
    Questo endpoint sarà valutato al momento della Primary Response Evaluation (PRE) (28 +/- 3 giorni dopo l'ultimo trattamento) durante la fase di trattamento di induzione di TOOKAD (padeliporfin) VTP.
    E.5.2Secondary end point(s)
    1.The duration of response at the entire ipsilateral kidney will be defined as absence of disease in the entire ipsilateral calyces, renal pelvis and ureter
    2.The duration of response at the Treatment Area of the ipsilateral kidney will be defined as absence of disease in the ipsilateral Treatment Area
    3.Overall renal functional outcome
    4.Kidney organ loss or preservation
    5.Pathological evaluation of response performed in kidney tissue of patient that will undergo kidney surgical removal following at least ne TOOKAD VTP treatment
    6.Safety follow up based and recording of adverse events
    1.La durata della risposta all'intero rene omolaterale sarà definita come assenza di malattia in tutto il calice omolaterale, nella pelvi renale e nell'uretere
    2. La durata della risposta nell'area di trattamento del rene omolaterale sarà definita come assenza di malattia nell'area di trattamento omolaterale
    3. Risultato funzionale renale complessivo
    4.Perdita o conservazione dell'organo renale
    5. Valutazione patologica della risposta eseguita nel tessuto renale del paziente che sarà sottoposto a rimozione chirurgica del rene dopo almeno un trattamento TOOKAD VTP
    6.Sicurezza basata sul follow up e sulla registrazione degli eventi avversi
    E.5.2.1Timepoint(s) of evaluation of this end point
    1.Measured at the 3, 6, 9 and 12 months maintenance treatment visit post PRE
    2.Measured at the 3, 6, 9 and 12 months maintenance treatment visit post PRE
    3.Measured at the 6, 12, 18 and 24 months maintenance treatment visit post PRE
    4.Measured at the 3, 6, 9 and 12, 18 and 24 months maintenance treatment visit post PRE
    5.Whenever possible for patients that will undergo kidney surgical removal
    6.At 18 and 24 months post PRE or 6 and 12 months post PRE if the patient has not participated to Maintenance treatment phase
    1. Misurato a 3, 6, 9 e 12 mesi di trattamento di mantenimento post PRE
    2. Misurato a 3, 6, 9 e 12 mesi di trattamento di mantenimento post PRE
    3. Misurato alla visita del trattamento di mantenimento di 6, 12, 18 e 24 mesi post PRE
    4. Misurato al 3, 6, 9 e 12, 18 e 24 mesi di trattamento di mantenimento post PRE
    5. Quando possibile per i pazienti che saranno sottoposti a rimozione chirurgica del rene
    6.A 18 e 24 mesi post PRE o 6 e 12 mesi post PRE se il paziente non ha partecipato alla fase di trattamento di mantenimento
    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 Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    Tumour Genomic
    Genomica del tumore
    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 No
    E.8.1.1Randomised No
    E.8.1.2Open No
    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 Yes
    E.8.1.7.1Other trial design description
    in aperto
    open
    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 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 Yes
    E.8.5.1Number of sites anticipated in the EEA7
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Israel
    United States
    Austria
    France
    Germany
    Italy
    Portugal
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 70
    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 state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 25
    F.4.2.2In the whole clinical trial 100
    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)
    After the trial, the patient care will revert to their physicians
    Dopo la sperimentazione, la cura dei pazienti tornerà ai loro medici
    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 Decision2021-08-23
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-09-28
    P. End of Trial
    P.End of Trial StatusOngoing
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