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    Summary
    EudraCT Number:2019-003395-39
    Sponsor's Protocol Code Number:MRX-503
    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:2021-05-24
    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 number2019-003395-39
    A.3Full title of the trial
    MRX-503: An Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in the Treatment of Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC)
    Studio di estensione in aperto per valutare l’efficacia e la sicurezza a lungo termine di Maralixibat nel trattamento di soggetti con Colestasi Intraepatica Progressiva Familiare (PFIC).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Extension Study to Evaluate the Long-term Safety and Efficacy of Maralixibat in Subjects with Progressive Familial Intrahepatic Cholestasis (PFIC).
    Studio di estensione per valutare la sicurezza e l'efficacia a lungo termine di Maralixibat in soggetti con colestasi intraepatica familiare progressiva (PFIC).
    A.3.2Name or abbreviated title of the trial where available
    Not Applicable
    Non applicabile
    A.4.1Sponsor's protocol code numberMRX-503
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/409/2019
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMirum 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 supportMirum Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMirum Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointChief Scientific Officer
    B.5.3 Address:
    B.5.3.1Street Address950 Tower Lane, Suite 300
    B.5.3.2Town/ cityFoster City California
    B.5.3.3Post codeCA 94404
    B.5.3.4CountryUnited States
    B.5.4Telephone number16506674085
    B.5.5Fax number16506674085
    B.5.6E-mailmedinfo@mirumpharma.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/3/13/1216
    D.3 Description of the IMP
    D.3.1Product nameMaralixibat
    D.3.2Product code [Maralixibat]
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 INNMaralixibat cloruro
    D.3.9.1CAS number 228113-66-4
    D.3.9.2Current sponsor codeMaralixibat
    D.3.9.4EV Substance CodeSUB195693
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 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.IMP: 2
    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/3/13/1216
    D.3 Description of the IMP
    D.3.1Product nameMaralixibat
    D.3.2Product code [Maralixibat]
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 INNMaralixibat cloruro
    D.3.9.1CAS number 228113-66-4
    D.3.9.2Current sponsor codeMaralixibat
    D.3.9.4EV Substance CodeSUB195693
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 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.IMP: 3
    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/3/13/1216
    D.3 Description of the IMP
    D.3.1Product nameMaralixibat
    D.3.2Product code [Maralixibat]
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 INNMaralixibat cloruro
    D.3.9.1CAS number 228113-66-4
    D.3.9.2Current sponsor codeMaralixibat
    D.3.9.4EV Substance CodeSUB195693
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number15
    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 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.IMP: 4
    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/3/13/1216
    D.3 Description of the IMP
    D.3.1Product nameMaralixibat
    D.3.2Product code [Maralixibat]
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    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 INNMaralixibat cloruro
    D.3.9.1CAS number 228113-66-4
    D.3.9.2Current sponsor codeMaralixibat
    D.3.9.4EV Substance CodeSUB195693
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    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 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 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
    In patients with progressive familial intrahepatic cholestasis (PFIC),
    impairment of the egress of bile acids from the liver leads to cholestasis,
    hepatocellular injury and damage, and progressive liver disease that
    may ultimately lead to the need for liver transplantation. Itch is a
    common symptom associated with cholestasis, it can occur at all stages
    of cholestatic liver disease, with or without jaundice.
    Nei pazienti con colestasi intraepatica familiare progressiva (PFIC),
    la compromissione della fuoriuscita degli acidi biliari dal fegato porta alla colestasi,
    a danno epatocellulare e a danno e malattia epatica progressiva che
    può infine portare alla necessità di un trapianto di fegato. Il prurito è a
    sintomo comune associato a colestasi e può verificarsi in tutte le fasi
    di malattia epatica colestatica, con o senza ittero.
    E.1.1.1Medical condition in easily understood language
    PFIC is a long-term debilitating and life-threatening disease
    due to liver and heart problems.
    PFIC è una malattia a lungo termine debilitante e potenzialmente letale a causa di problemi al fegato ed al cuore
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10076033
    E.1.2Term Progressive familial intrahepatic cholestasis
    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 evaluate the long-term safety and tolerability of maralixibat.
    L'obiettivo principale dello studio è valutare la sicurezza e la tollerabilità a lungo termine di maralixibat.
    E.2.2Secondary objectives of the trial
    To evaluate the long-term efficacy of maralixibat, including the maintenance of severity and frequency of pruritus as well as serum bile acids (sBA) over time and growth in the primary cohort
    Valutare l'efficacia a lungo termine di maralixibat, incluso il mantenimento della gravità e della frequenza del prurito, nonché degli acidi biliari sierici (sBA) nel tempo e la crescita nella coorte primaria
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Provide informed consent and assent (as applicable) per Institutional Review Board/Ethics Committee.
    - Completion of study MRX-502; treatment interruption between MRX-502 and MRX-503 should be avoided. Subjects who do not complete the study MRX-503 Baseline Visit (Day 0) on the same day as the study MRX 502 EOT Visit will be considered for participation in study MRX-503 only after discussion with the Medical Monitor.
    - Males and females of non-childbearing potential. Males and non-pregnant, non lactating females of childbearing potential who are sexually active must agree to use acceptable contraception during the study through 30 days after the last dose of maralixibat.
    - Females of childbearing potential must have a negative urine pregnancy test at the Baseline Visit (Day 0).
    - Access to email or telephone for scheduled remote visits.
    - Ability to read and understand the questionnaires (both caregivers and subjects above the age of assent).
    - Access to consistent caregiver(s) during the study.
    - Subject and caregiver willingness to comply with all study visits and requirements.
    - Fornire il consenso informato e il consenso (ove applicabile) come da Comitato etico.
    - Completamento dello studio MRX-502; l'interruzione del trattamento tra MRX-502 e MRX-503 deve essere evitata. I soggetti che non completano lo studio MRX-503 Baseline Visit (giorno 0) lo stesso giorno dello studio MRX 502 End Of Treatment Visit, verranno considerati per la partecipazione allo studio MRX-503 solo dopo aver discusso con il Medical Monitor.
    - Maschi e femmine in età fertile. I maschi e le femmine non in gravidanza, che non allattano, in età fertile che sono sessualmente attivi, devono concordare di utilizzare una contraccezione accettabile durante lo studio fino a 30 giorni dopo l'ultima dose di maralixibat.
    - Le femmine in età fertile devono essere negative al test di gravidanza sulle urine alla visita basale (giorno 0).
    - Accesso alla posta elettronica o al telefono per le visite remote programmate.
    - Capacità di leggere e comprendere i questionari (sia i caregiver che i soggetti al di sopra dell'età dell' assenso).
    - Accesso a caregiver coerenti durante lo studio.
    - Disponibilità del soggetto e del caregiver a rispettare tutte le visite e i requisiti dello studio.
    E.4Principal exclusion criteria
    - Any female who is pregnant or lactating or who is planning to become pregnant.
    - Administration of prohibited medication between the MRX-502 EOT visit and the MRX 503 Baseline Visit (Day 0).
    - History of non-compliance in study MRX-502, non-adherence to medical regimens, unreliability, mental instability, or incompetence that could compromise the validity of informed consent or lead to non-adherence with the study protocol based on Investigator judgment.
    - Experienced an adverse event (AE) or serious adverse event (SAE) related to maralixibat during the MRX-502 study that led to permanent discontinuation of the subject from maralixibat.
    - Any other conditions or laboratory abnormalities that, in the opinion of the Investigator or Sponsor Medical Monitor, may compromise the safety of the subject, or interfere with the subject participating in or completing the study.
    - Cognitive impairment of the subject or caregiver that would, in the opinion of the Investigator, preclude appropriate understanding of study information and compliance with study procedures.
    - Qualsiasi donna incinta o in allattamento o che sta pianificando una gravidanza.
    - Somministrazione di farmaci vietati tra la visita EOT MRX-502 e la visita basale MRX 503 (Giorno 0).
    - Storia di non conformità nello studio MRX-502, non aderenza a regimi medici, inaffidabilità, instabilità mentale o incompetenza che potrebbero compromettere la validità del consenso informato o portare a non aderenza al protocollo di studio, basato sul giudizio dello sperimentatore.
    - Durante lo studio MRX-502 il soggetto ha sperimentato un evento avverso (AE) o un evento avverso serio (SAE) correlato al maralixibat che ha portato all'interruzione permanente del soggetto dal trattamento con maralixibat.
    - Qualsiasi altra condizione o anomalia da laboratorio che, secondo l'opinione dell'Investigatore o del Medical Monitor dello Sponsor, possa compromettere la sicurezza del soggetto o interferire con il soggetto che partecipa o completa lo studio.
    - Compromissione cognitiva del soggetto o del caregiver che, a giudizio dell'Investigatore, impedirebbe un'adeguata comprensione delle informazioni sullo studio e il rispetto delle procedure di studio.
    E.5 End points
    E.5.1Primary end point(s)
    - Incidence of treatment-emergent adverse events (TEAEs) during the study by system organ class (SOC) and preferred term (serious, non-serious, related, and non-related) (primary objective for the primary cohort and for all cohorts combined)
    - Safety laboratory parameters (clinical laboratory tests [hematology, chemistry, urinalysis, lipid-soluble vitamins, and related measures)] over time (primary objective for the primary cohort and for all cohorts combined)
    - Incidenza di eventi avversi emergenti dal trattamento (TEAE) durante lo studio per classe di organo e sistema (SOC) e termine preferito (grave, non grave, correlato e non correlato) (obiettivo primario per la coorte primaria e per tutte le coorti combinate )
    - Parametri di laboratorio di sicurezza (test clinici di laboratorio [ematologia, chimica, analisi delle urine, vitamine liposolubili e misure correlate)] nel tempo (obiettivo primario per la coorte primaria e per tutte le coorti combinate)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Safety analyses will be conducted using data from the Safety Population. Safety variables include TEAEs and clinical laboratory values. No formal inferential analyses will be conducted for safety variables, unless otherwise noted. Analyses will be conducted separately on the primary cohort and all cohorts combined.
    Le analisi di sicurezza saranno condotte utilizzando i dati della popolazione di sicurezza. Le variabili di sicurezza includono TEAE e valori clinici di laboratorio. Nessuna analisi inferenziale formale sarà condotta per variabili di sicurezza, se non diversamente indicato. Le analisi saranno condotte separatamente sulla coorte primaria e su tutte le coorti combinate.
    E.5.2Secondary end point(s)
    - Mean change from baseline over time in the average morning Observer reported Itch Reported Outcome Instrument (ItchRO(Obs))™ severity score (secondary objective in the primary cohort and exploratory objective in the supplemental cohort and any subgroup[s])
    - Mean change from baseline over time in the average morning ItchRO(Obs) frequency score (secondary objective in the primary cohort and exploratory objective in the supplemental cohort and any subgroup[s])
    - Maintenance of treatment effect based on the average morning ItchRO(Obs) severity scores over time (secondary objective in the primary cohort and exploratory objective in the supplemental cohort and any subgroup[s])
    - Mean change from baseline over time in sBA levels (secondary objective in the primary cohort and exploratory objective in the supplemental cohort and any subgroup[s])
    - Mean change from baseline over time in height and weight z-scores (secondary objective in the primary cohort and exploratory objective in the supplemental cohort and any subgroup[s])
    - Proportion of responders over time (secondary objective in the primary cohort and exploratory objective in the supplemental cohort and any subgroup[s])
    - Variazione media rispetto al basale nel tempo nella mattinata media dell' Osservatore ha riportato il punteggio di gravità Itch Reported Outcome Instrument (ItchRO (Obs)) ™ (obiettivo secondario nella coorte primaria e obiettivo esplorativo nella coorte supplementare e qualsiasi sottogruppo)
    - Variazione media rispetto al basale nel tempo del punteggio di frequenza ItchRO (Obs) medio mattutino (obiettivo secondario nella coorte primaria e obiettivo esplorativo nella coorte supplementare e qualsiasi sottogruppo [s])
    - Mantenimento dell'effetto del trattamento in base ai punteggi medi di gravità ItchRO (Obs) mattutini nel tempo (obiettivo secondario nella coorte primaria e obiettivo esplorativo nella coorte supplementare e qualsiasi sottogruppo / i)
    - Variazione media rispetto al basale nel tempo dei livelli di SBA (obiettivo secondario nella coorte primaria e obiettivo esplorativo nella coorte supplementare ed eventuali sottogruppi)
    - Variazione media rispetto al basale nel tempo in altezza e peso z-score (obiettivo secondario nella coorte primaria e obiettivo esplorativo nella coorte supplementare ed eventuali sottogruppi)
    - Proporzione di responder nel tempo (obiettivo secondario nella coorte primaria e obiettivo esplorativo nella coorte supplementare e qualsiasi sottogruppo / i)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Secondary and exploratory endpoints will be analyzed as described in the supplemental cohort in the Safety Population, overall and by treatment sequence. Additional subgroupings of the supplemental cohort may also be explored depending on the sample sizes.
    Gli endpoint secondari ed esplorativi saranno analizzati come descritto nella coorte supplementare nella popolazione di sicurezza, in generale e per sequenza di trattamento. Ulteriori sottogruppi della coorte supplementare possono anche essere esplorati a seconda delle dimensioni del campione.
    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 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 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 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 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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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
    Brazil
    Canada
    Colombia
    Lebanon
    Mexico
    Singapore
    Turkey
    United States
    Austria
    Belgium
    France
    Germany
    Hungary
    Italy
    Poland
    United Kingdom
    Argentina
    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
    LPLV
    LPLV
    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 months0
    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 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 Yes
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 5
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 4
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 1
    F.1.3Elderly (>=65 years) No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    All subjects from 502 study can rollover to 503 extension study after signed ICF. Assent & parent/caregiver consent will be obtained for children not
    able to provide assent personally. Subjects signed assent at age 17 may turn to 18 during the study
    Tutti i soggetti dello studio 502 possono passare al studio di estensione 503 dopo aver firmato il Consenso Informato. L'assenso e il consenso dei genitori / caregiver saranno ottenuti per i bambini non
    in grado di fornire assenso personalmente. Gli
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 13
    F.4.2.2In the whole clinical trial 30
    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 protocol MRX-503, patients will be provided with opportunity to continue treatment in another extension study MRX-800.
    Al completamento del protocollo MRX-503, i pazienti avranno l'opportunità di continuare il trattamento in un altro studio di estensione MRX-800.
    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 Decision2020-05-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-08
    P. End of Trial
    P.End of Trial StatusOngoing
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