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    Summary
    EudraCT Number:2017-002447-15
    Sponsor's Protocol Code Number:SPIMM-301
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-10-16
    Trial results View 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 number2017-002447-15
    A.3Full title of the trial
    A Phase 3 Randomized, Double-Blind, Parallel-Group, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Daily Subcutaneous Injections of Elamipretide in Subjects with Primary Mitochondrial Myopathy Followed by an Open-Label Treatment Extension
    Sperimentazione di fase 3, randomizzata, in doppio cieco, a gruppi paralleli, controllata con placebo per valutare l’efficacia e la sicurezza di iniezioni sottocutanee giornaliere di elamipretide in soggetti con miopatia mitocondriale primaria seguita da un’estensione del trattamento in aperto
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to assess the efficacy, safety and tolerability of daily subcutaneous Injections of Elamipretide in subjects with Primary Mitochondrial Myopathy
    Uno studio per valutare l'efficacia, la sicurezza e la tollerabilità di iniezioni sottocutanee quotidiane di elamipretide in soggetti con miopatia mitocondriale primaria
    A.4.1Sponsor's protocol code numberSPIMM-301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT03323749
    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 SponsorStealth BioTherapeutics 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 supportStealth BioTherapeutics Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationStealth BioTherapeutics Inc.
    B.5.2Functional name of contact pointDirector, Clinical Operations
    B.5.3 Address:
    B.5.3.1Street Address275 Grove Street, 3-107
    B.5.3.2Town/ cityNewton
    B.5.3.3Post codeMA 02466
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1617600-6888
    B.5.5Fax number+1617600-6884
    B.5.6E-mailJoseph.Covino@stealthbt.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameElamipretide
    D.3.2Product code MTP-131
    D.3.4Pharmaceutical form Solution for injection in dose-dispenser cartridge
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNELAMIPRETIDE
    D.3.9.1CAS number 736992-21-5
    D.3.9.2Current sponsor codeMTP-131
    D.3.9.3Other descriptive nameSS-31; SBT-031, SPI-31, Bendavia™
    D.3.9.4EV Substance CodeSUB187808
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number80
    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 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection in dose-dispenser cartridge
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Primary Mitochondrial Myopathy
    Miopatia Mitocondriale Primaria
    E.1.1.1Medical condition in easily understood language
    Primary Mitochondrial Myopathy
    Miopatia Mitocondriale Primaria
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10027710
    E.1.2Term Mitochondrial myopathy
    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 PART 1 is to evaluate the effect of single daily SC doses of 40 mg elamipretide administered with the elamipretide delivery system for 24 weeks on the:
    • Distance Walked on the 6MWT
    • Total Fatigue on the Primary Mitochondrial Myopathy Symptom Assessment (PMMSA)

    The PART 2 objective is to assess the long-term safety and tolerability of single daily SC doses of 40 mg elamipretide administered with the elamipretide delivery system for up to 144 weeks.
    L’obiettivo primario della PARTE 1 è valutare l’effetto di singole dosi SC giornaliere di elamipretide da 40 mg somministrate con l’ausilio del sistema di erogazione di elamipretide per 24 settimane su:
     Distanza percorsa a piedi nel test dei 6 minuti di cammino (6MWT)
     Affaticamento totale misurato mediante la Valutazione dei sintomi della miopatia mitocondriale primaria (PMMSA)
    L’obiettivo primario della PARTE 2 è valutare la sicurezza e la tollerabilità a lungo termine di singole dosi SC giornaliere di elamipretide da 40 mg somministrate con l’ausilio del sistema di erogazione di elamipretide per un massimo di 144 settimane.
    E.2.2Secondary objectives of the trial
    Secondary objectives of PART 1 are:
    • To evaluate the effect of single daily SC doses of 40 mg elamipretide administered with the elamipretide delivery system for 24 weeks as measured by changes in the:
    - Fatigue During Activities on the PMMSA
    - Neuro-QoL Fatigue
    - Most bothersome symptom on the PMMSA
    • To evaluate the safety and tolerability of single daily SC doses of 40 mg elamipretide administered with the elamipretide delivery system for 24 weeks.
    Gli obiettivi secondari della PARTE 1 sono:
     Valutare l’effetto di singole dosi SC giornaliere di elamipretide da 40 mg somministrate con l’ausilio del sistema di erogazione di elamipretide per 24 settimane misurato in base alle variazioni di:
     Affaticamento durante le attività alla PMMSA
     Scala di valutazione della qualità della vita nei disturbi neurologici (Neuro-QoL) relativa all’affaticamento
     Sintomo più fastidioso alla PMMSA
     Valutare la sicurezza e la tollerabilità di singole dosi SC giornaliere di elamipretide da 40 mg somministrate con l’ausilio del sistema di erogazione di elamipretide per 24 settimane
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Willing and able to provide a signed informed consent form (ICF) prior to participation in any trial-related procedures.
    2. Agrees and is able to adhere to the trial requirements for the length of the trial, including the use of the elamipretide delivery system.
    3. Subject is ≥16 and ≤80 years of age
    4. Enrolled (signed ICF) in SPIMM-300.
    5. Diagnosed with PMM in the opinion of the Investigator, consisting of:
    a. Molecular genetic abnormality of the mitochondrial respiratory chain, and
    b. Subject reported symptoms (i.e., exercise intolerance, fatigue, muscle weakness) or physical examination findings of myopathy that are the predominant symptoms of the subject’s mitochondrial respiratory chain disorder.
    6. The subject’s molecular genetic abnormality is consistent with PMM as confirmed by the Adjudication Committee in SPIMM-300.
    7. Women of childbearing potential must agree to use 1 of the following methods of birth control from the date they sign the ICF until trial completion:
    a. Abstinence, when it is in line with the preferred and usual lifestyle of the subject. Subject agrees to use a highly effective method of contraception should they become sexually active.
    b. Relationships with male partners who have been surgically sterilized by vasectomy (the vasectomy procedure must have been conducted at least 60 days prior to the Screening Visit).
    c. Barrier method (e.g., condom or occlusive cap) with spermicidal foam/gel/film/cream AND either hormonal contraception (oral, implanted, or
    injectable) or an intrauterine device or system.
    Note: Non-childbearing potential is defined as surgical sterilization (e.g., bilateral oophorectomy, hysterectomy, or tubal ligation) or postmenopausal (defined as permanent cessation of menstruation for at least 12 consecutive months prior to the Screening Visit).
    8. Male subjects with female partners of child-bearing potential must be willing to use a highly effective method of contraception from the date they sign the ICF until trial completion.

    PART 2 Subject Continuation Criteria
    A subject must meet all of the following PART 2 Continuation Criteria at the Week 24 Visit in SPIMM-301 to be eligible for PART 2:
    1. Subjects must continue to be able and willing to adhere to the trial requirements.
    2. Subject is appropriate to continue in PART 2 (i.e. subject was compliant in SPIMM-301), in the opinion of the Investigator.
    3. Subject has not had a serious adverse event (SAE)/serious adverse device effect (SADE) attributed to the elamipretide delivery system.
    4. Subject has not permanently discontinued the elamipretide delivery system.
    1. Disponibilità e capacità di fornire un modulo di consenso informato (ICF) firmato prima della partecipazione a qualsiasi procedura correlata alla sperimentazione.
    2. Accetta ed è in grado di aderire ai requisiti della sperimentazione per la durata della stessa, compreso l’uso del sistema di erogazione di elamipretide.
    3. Il soggetto ha un’età ≥16 e ≤80 anni.
    4. Il soggetto è arruolato (ICF firmato) in SPIMM-300.
    5. Il soggetto ha ricevuto una diagnosi di PMM secondo il parere dello sperimentatore, costituita da:
    a. anomalia genetica molecolare della catena respiratoria mitocondriale e
    b. sintomi riferiti dal soggetto (ovvero, intolleranza all’esercizio fisico, affaticamento, debolezza muscolare) o riscontri di miopatia all’esame obiettivo che rappresentano i sintomi predominanti del disturbo a carico della catena respiratoria mitocondriale del soggetto.
    6. L’anomalia genetica molecolare del soggetto è coerente con la PMM, secondo quanto confermato dal Comitato di validazione in SPIMM-300.
    7. Le donne in età fertile devono acconsentire a usare uno dei seguenti metodi contraccettivi dalla data in cui firmano l’ICF al completamento della sperimentazione:
    a. Astinenza, se in linea con lo stile di vita preferito e abituale del soggetto. Il soggetto acconsente a usare un metodo contraccettivo altamente efficace qualora dovesse diventare sessualmente attivo.
    b. Rapporti con partner di sesso maschile che sono stati resi sterili chirurgicamente mediante vasectomia (la procedura di vasectomia deve essersi conclusa almeno 60 giorni prima della visita di screening).
    c. Metodo barriera (es. preservativo o cappuccio occlusivo) con schiuma/gel/crema spermicida E contraccezione ormonale (orale, impiantabile o iniettabile) o un dispositivo o sistema intrauterino.
    Nota: le donne non in età fertile sono quelle donne che si sono sottoposte a sterilizzazione chirurgica (es. ooforectomia bilaterale, isterectomia o legatura tubarica) o in età post-menopausale (definita come l’interruzione permanente del ciclo mestruale da almeno 12 mesi consecutivi prima della visita di screening).
    8. I soggetti di sesso maschile con compagne in età fertile devono essere disposti a usare un metodo contraccettivo altamente efficace dalla data in cui firmano l’ICF al completamento della sperimentazione.
    Criteri per il proseguimento nella PARTE 2
    Un soggetto deve soddisfare tutti i seguenti Criteri per il proseguimento nella PARTE 2 alla Visita della Settimana 24 nella PARTE 1 per essere idoneo alla PARTE 2:
    1. Il soggetto deve continuare ad essere in grado di e disposto ad attenersi ai requisiti della sperimentazione.
    Il soggetto è adatto a continuare nella PARTE 2 (ovvero, ha seguito i requisiti previsti dalla PARTE 1), secondo il parere dello sperimentatore.
    Il soggetto non ha manifestato un evento avverso grave (SAE)/effetto avverso grave del dispositivo (SADE) attribuito al sistema di erogazione di elamipretide.
    Il soggetto non ha interrotto in maniera permanente l’uso del sistema di erogazione di elamipretide.
    E.4Principal exclusion criteria
    1. Subject has myopathic signs and/or symptoms due to a neuropathic process (i.e. cerebellar dysfunctions and neuropathies) or a gait problem that would interfere with the 6MWT, in the opinion of the Investigator.
    2. Female subjects who are pregnant, planning to become pregnant, or breastfeeding/lactating.
    3. Walks < 100 meters or > 450 meters during the 6MWT at either the Screening Visit OR Baseline Visit.
    4. At Screening, the estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2, using the Modification of Diet in Renal Disease (MDRD) Study equation.
    5. Subject has undergone an in-patient hospitalization within the 30 days prior to the Baseline Visit or has a planned hospitalization or a surgical procedure during the trial.
    6. Subject has clinically significant cardiac disease or prior interventional procedure and/or respiratory disease (medical history or current clinical findings) within 3 months of the Baseline Visit, in the opinion of the Investigator.
    7. Subject has QTc elongation (using the correction factor utilized at the clinical site) defined as a QTc >450 msec in male subjects and >480 msec in female subjects.
    Note: At the initial electrocardiogram (ECG), if QTc exceeds these parameters, the ECG may be repeated 2 more times (during the same visit), and the average of the 3 QTc values used to determine the subject’s eligibility.
    8. ECG evidence of acute ischemia, atrial fibrillation, or active conduction system abnormalities with the exception of any of the following:
    a. First degree AV-block
    b. Second degree AV-block Type 1 (Mobitz Type 1 / Wenckebach type)
    c. Right bundle branch block
    9. Subject has severe vision impairment that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements
    10. Subject has a seizure disorder that, in the opinion of the Investigator, may interfere with their ability to complete all trial requirements.
    11. Active malignancy or any other cancer from which the subject has been disease-free for < 2 years.
    12. Subject has a solid organ transplant and/or is currently receiving treatment with therapy for immunosuppression, in the opinion of the Investigator.
    13. Subject has been previously diagnosed with human immunodeficiency virus (HIV), hepatitis B, or hepatitis C infection.
    14. Subject has a history of a systemic eosinophilic illness and/or an eosinophil count >1,000 cells x106/L at the Screening Visit.
    15. Subject is currently participating or has participated in an interventional clinical trial (i.e., investigational product or device, stem cell therapy, gene therapy) within 30 days of the Baseline Visit; or is currently enrolled in a non-interventional clinical trial (except for SPIMM-300) at the Baseline Visit which, in the opinion of the Investigator, may be potentially confounding to the results of the current trial (e.g. exercise therapy trial).
    16. Subject has previously received elamipretide (MTP-131), for any reason.
    17. Subject has a history of active substance abuse during the year before the Baseline Visit, in the opinion of the Investigator.
    18. Subject has any prior or current medical condition that, in the judgment of the Investigator, would prevent the subject from safely participating in and/or completing all trial requirements.
    1. Il soggetto presenta segni e/o sintomi miopatici dovuti a un processo neuropatico (ovvero, disfunzioni cerebellari e neuropatie) o un problema di andatura che, secondo il parere dello sperimentatore, interferirebbero con il 6MWT.
    2. Soggetti di sesso femminile in stato di gravidanza, che prevedono di avviare una gravidanza o che allattano al seno.
    3. Il soggetto percorre a piedi <100 metri o >450 metri durante il 6MWT alla visita di screening OPPURE alla visita basale.
    4. Allo screening, la velocità di filtrazione glomerulare stimata (eGFR) è <30 ml/min/1,73 m2, usando l’equazione di studio della Modifica della dieta nella malattia renale (MDRD).
    5. Il soggetto si è sottoposto a un ricovero ospedaliero nei 30 giorni precedenti la visita basale o ha programmato di ricoverarsi o sottoporsi a una procedura chirurgica durante la sperimentazione.
    6. Il soggetto presenta una malattia cardiaca clinicamente significativa o una precedente procedura interventistica e/o malattia respiratoria (anamnesi medica o risultati clinici attuali) entro 3 mesi dalla visita basale, secondo il parere dello sperimentatore.
    7. Il soggetto presenta prolungamento del QTc (utilizzando il fattore di correzione adottato dal centro clinico) definito come un QTc >450 msec nei soggetti di sesso maschile e >480 msec nei soggetti di sesso femminile.
    a. Nota: all’elettrocardiogramma (ECG) iniziale, se il QTc eccede questi parametri, l’ECG può essere ripetuto altre 2 volte (durante la stessa visita) e la media dei 3 valori di QTc può essere usata per determinare l’idoneità del soggetto.
    8. Evidenza all’ECG di ischemia acuta, fibrillazione atriale o anomalie attive nel sistema di conduzione, esclusa una qualsiasi tra le seguenti condizioni:
    a. Blocco atrio-ventricolare (AV) di primo grado
    b. Blocco AV tipo 1 di secondo grado (Mobitz tipo 1/tipo Wenckebach)
    c. Blocco di branca destra
    9. Il soggetto presenta una grave compromissione della vista che, secondo il parere dello sperimentatore, potrebbe interferire con la sua capacità di completare tutti i requisiti della sperimentazione.
    10. Il soggetto presenta un disturbo da crisi convulsive che, secondo il parere dello sperimentatore, potrebbe interferire con la sua capacità di completare tutti i requisiti della sperimentazione.
    11. Tumore maligno attivo o qualsiasi altra forma tumorale per cui il soggetto sia rimasto libero da malattia per meno di 2 anni.
    12. Il soggetto ha subìto un trapianto di organo solido e/o attualmente sta ricevendo un trattamento con terapia immunosoppressiva, secondo il parere dello sperimentatore.
    13. Al soggetto è stata diagnosticata l’infezione da virus dell’immunodeficienza umana (HIV), epatite B o epatite C.
    14. Il soggetto presenta un’anamnesi di malattia eosinofila sistemica e/o una conta eosinofila >1.000 cellule x106/l alla visita di screening.
    15. Il soggetto sta attualmente partecipando o ha partecipato a una sperimentazione clinica interventistica (ovvero, con un prodotto o dispositivo sperimentale, terapia con cellule staminali, terapia genica) entro 30 giorni dalla visita basale; oppure è attualmente arruolato in una sperimentazione clinica non interventistica (esclusa SPIMM-300) alla visita basale che, secondo il parere dello sperimentatore, potrebbe potenzialmente rappresentare un fattore di confusione con i risultati dell’attuale sperimentazione (per es., sperimentazione con terapia dell’esercizio).
    16. Il soggetto ha ricevuto in precedenza elamipretide (MTP-131) per un qualsiasi motivo.
    17. Il soggetto presenta un’anamnesi di abuso di principio attivo durante l’anno precedente la visita basale, secondo il parere dello sperimentatore.
    18. Il soggetto presenta qualsiasi condizione medica attuale o pregressa che, a giudizio dello sperimentatore, gli impedirebbe di partecipare e/o completare in modo sicuro tutte le procedure richieste dalla sperimentazione.
    E.5 End points
    E.5.1Primary end point(s)
    The primary (efficacy) endpoints are:
    • Distance walked (meters) during the 6MWT
    • Total Fatigue score on the PMMSA

    Efficacy will only be assessed for PART 1.
    Endpoint primari (efficacia)
     Distanza percorsa (metri) durante il 6MWT
     Punteggio totale relativo all’affaticamento alla PMMSA
    L’efficacia sarà valutata soltanto per la PARTE 1.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The primary time point is at Week 24.
    il timepoint primario è alla settimana 24.
    E.5.2Secondary end point(s)
    The secondary endpoints are:
    • Fatigue During Activities score on the PMMSA
    • Neuro-QoL Fatigue score
    • Most bothersome symptom score on the PMMSA
    Endpoint secondari sono:
     Punteggio relativo all’affaticamento durante le attività alla PMMSA
     Punteggio della Neuro-QoL relativa all’affaticamento
     Punteggio relativo al sintomo più fastidioso alla PMMSA
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 24 weeks
    24 settimane
    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 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 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 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA12
    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
    Canada
    Denmark
    Germany
    Hungary
    Italy
    United Kingdom
    United States
    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
    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 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: 194
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 2
    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 state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 80
    F.4.2.2In the whole clinical trial 202
    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
    Nessuno
    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 Decision2019-01-04
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-01-17
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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