Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43874   clinical trials with a EudraCT protocol, of which   7294   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2021-001703-32
    Sponsor's Protocol Code Number:CRN00808-08
    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:2022-03-15
    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 number2021-001703-32
    A.3Full title of the trial
    A Randomized, Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Paltusotine in Subjects with Non-pharmacologically Treated Acromegaly
    Uno Studio Randomizzato, Controllato e Multicentrico per Valutare la Sicurezza e l'Efficacia della Paltusotina in Soggetti con Acromegalia non Trattata Farmacologicamente
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study where subjects with non-pharmacologically treated acromegaly will be randomly allocated to receive either paltusotine or placebo.
    Questo è uno studio di fase 3, multicentrico, randomizzato, doppio cieco, controllato con placebo dove i soggetti con acromegalia non trattata farmacologicamente saranno assegnati in modo casuale a ricevere paltusotina o placebo.
    A.3.2Name or abbreviated title of the trial where available
    PATHFNDR-2
    PATHFNDR-2
    A.4.1Sponsor's protocol code numberCRN00808-08
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05192382
    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 SponsorCrinetics 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 supportCrinetics Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCrinetics Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointCrinetics Clinical Trials
    B.5.3 Address:
    B.5.3.1Street Address10222 Barnes Canyon Road, Bldg. 2
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post codeCA 92121
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@crinetics.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 namePaltusotine
    D.3.2Product code [CRN00808]
    D.3.4Pharmaceutical form Tablet
    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 INNPaltusotine (Free base)
    D.3.9.1CAS number 2172870-89-0
    D.3.9.2Current sponsor codeCRN00808
    D.3.9.4EV Substance CodeSUB194845
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboTablet
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Acromegaly
    Acromegalia
    E.1.1.1Medical condition in easily understood language
    Acromegaly is typically caused by a growth hormone (GH) secreting tumor in the pituitary.
    L'Acromegalia è tipicamente causata da un tumore nell'ipofisi che causa la produzione di un ormone della crescita (GH)
    E.1.1.2Therapeutic area Diseases [C] - Hormonal diseases [C19]
    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
    To evaluate the effect of paltusotine versus placebo on IGF-1 response
    Valutare l'effetto di paltusotina rispetto al placebo sulla risposta di IGF-1
    E.2.2Secondary objectives of the trial
    To evaluate the effect of paltusotine versus placebo on IGF-1 level
    To evaluate the effect of paltusotine versus placebo on IGF-1 response
    To evaluate the effect of paltusotine versus placebo on GH response
    To evaluate the effect of paltusotine versus placebo on acromegaly symptoms
    Valutare l'effetto di paltusotina rispetto al placebo sul livello di IGF-1
    Valutare l'effetto di paltusotina rispetto al placebo sulla risposta di IGF-1
    Valutare l'effetto di paltusotina rispetto al placebo sulla risposta del GH
    Valutare l'effetto di paltusotina rispetto al placebo sui sintomi di acromegalia
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Willing and able to provide written informed consent prior to any study-related procedures.
    2. Willing and able to comply with the study procedures as specified in the protocol and comply with the study treatment administration.
    3. Subjects >or =18 years of age at the time of Screening, who have had at least 1 pituitary surgery but have persistent active acromegaly and who fall into 1 of the following 3 groups are eligible to participate in the study.
    • Medically naïve group (Group 1): Those who have not been previously treated with acromegaly medications (including LA-SRLs) who at Screening have IGF >or 1 =1.3×ULN (rounded to 2 significant figures must be >or =1.25) confirmed by the central laboratory test at S1.
    • Previously Treated group (no treatment within previous 4 months) (Group 2): Subjects who have last been treated with acromegaly medications at least 4 months prior to Screening and who have IGF 1>or =1.3×ULN (rounded to 2 significant figures must be >or=1.25) confirmed by the central laboratory test at S1.
    • Washout group (Group 3): Subjects at Screening who are receiving stable treatment (no change in dose for 3 months prior to Screening) with octreotide monotherapy, who have IGF 1 <or=1.0×ULN (rounded to 2 significant figures must be < or =1.04) at Screening Visit 1, and are willing to washout of their medication during the Screening Period.
    4. Previous diagnosis of acromegaly confirmed by the Investigator and approved by the Medical Monitor. This requires evaluable documentation of a pituitary adenoma at least 24 weeks prior to Screening.
    5. If currently using thyroid hormone therapy, the subject should be adequately treated based on clinical status and free thyroxine concentration measured during Screening and on a stable dose of thyroid hormone for at least 8 weeks prior to Screening.
    6. Females who engage in heterosexual intercourse must be of non childbearing potential, defined as either surgically sterile (i.e., hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), OR be postmenopausal with at least 1 year of amenorrhea, OR must agree to use either a highly effective or a clinically acceptable method of contraception from the beginning of Screening until at least 30 days after the last dose of study drug.
    7. If the subject is male, the subject should agree to use a condom when sexually active with a female partner of childbearing potential from Screening until at least 30 days after the last dose of study drug (or be surgically sterile [i.e., vasectomy with documentation]; or remain abstinent [when this is in line with the preferred and usual lifestyle]. Male subjects should also agree to not donate sperm for the duration of the study and until at least 30 days after the last dose of study drug.
    1- Soggetti disposti e in grado di fornire un consenso informato scritto prima di qualsiasi
    procedura relativa allo studio
    2- Disposti e in grado di rispettare le procedure di studio come specificato nel protocollo e rispettino la somministrazione del trattamento in studio.
    3. Soggetti di età >o =18 anni al momento dello Screening, che hanno avuto almeno 1 intervento ipofisario ma con persistente acromegalia attiva e che rientrino in 1 dei seguenti 3 gruppi sono ammessi a partecipare allo studio.
    • Soggeti con acromegalia naïve al trattamento (Gruppo1): Coloro che non sono stati in precedenza trattati con farmaci per l'acromegalia (inclusi LA-SRLs) che allo screening ha IGF 1 >o =1.3×ULN (arrotondato a 2 cifre significative deve essere >o =1.25) confermato dal test di laboratorio centrale in S1.
    • Gruppo precedentemente trattato (nessun trattamento nei 4 mesi precedenti) (Gruppo 2): Soggetti che sono stati trattati per l'ultima volta con farmaci per acromegalia almeno 4 mesi prima dello screening e che hanno IGF 1 >o=1.3×ULN (arrotondato a 2 cifre significative deve essere >o=1.25) confermato da test di laboratorio centrale a S1.
    • Gruppo Washout (Gruppo 3): Soggetti allo Screening che stanno ricevendo
    trattamento stabile (nessuna variazione della dose per 3 mesi prima dello screening)
    con octreotide in monoterapia, che hanno IGF 1 <or =1.0×ULN (arrotondato a 2 cifre significative devono essere < o =1.04) alla visita di screening 1 e sono disposti a
    eliminazione dei farmaci durante il periodo di screening.
    4-Pregressa diagnosi di acromegalia confermata dallo Sperimentatore e approvata dal Medical Monitor. Ciò richiede una documentazione valutabile di un adenoma ipofisario almeno 24 settimane prima dello screening.
    5-Se attualmente sta utilizzando una terapia a base di ormoni tiroidei, il soggetto deve essere adeguatamente trattato sulla base dello stato clinico e della concentrazione di tiroxina libera misurata durante lo screening e con una dose stabile di ormone tiroideo per almeno 8 settimane prima dello screening.
    6-Soggetti femminili sessualmente attivi devono essere potenzialmente non fertili, ossia definiti come sterili chirurgicamente (cioè, isterectomia, salpingectomia bilaterale o ovariectomia bilaterale), o essere in postmenopausa con almeno 1 anno di amenorrea, OPPURE devono accettare di utilizzare un metodo altamente efficace o clinicamente accettabile di contraccezione dall'inizio dello screening fino ad almeno 30 giorni
    dopo l'ultima dose del farmaco in studio.
    7- Se il soggetto è maschio, il soggetto dovrebbe accettare di usare il preservativo quando
    sessualmente attivo con una partner femminile in età fertile dallo Screening fino ad almeno 30 giorni dopo l'ultima dose del farmaco in studio (o essere chirurgicamente sterile [cioè vasectomia con documentazione]; o rimanere astinente [quando questo è in linea con lo stile di vita preferito e abituale]. I soggetti di sesso maschile dovrebbero anche accettare di non donare sperma per la durata dello studio e fino ad almeno 30 giorni dopo l'ultima dose del farmaco in studio.
    E.4Principal exclusion criteria
    -History of ineffectiveness or significant intolerance of octreotide or lanreotide treatment, as determined by the Investigator.
    -History of pituitary radiation therapy within 3 years of Screening.
    -Subjects with adrenal insufficiency, diabetes insipidus, or central hypogonadism who are not receiving adequate hormone replacement therapy at the time of Screening, as determined by the Investigator.
    -High risk pituitary tumor pattern
    -History of major surgery/surgical therapy for any cause within 4 weeks prior to Screening.
    -Diabetes mellitus treated with insulin for less than 6 weeks prior to the study entry, or with change in total daily insulin dose by >15% within 6 weeks prior to Screening.
    -History of unstable angina or acute myocardial infarction within the 12 weeks preceding the Screening Visit or other clinically significant cardiac disease at the time of screening as judged by the Investigator.
    -Known history of hepatitis B or human immunodeficiency virus, or active hepatitis C infection.
    -Active malignant disease within the last 5 years with exception of basal and squamous cell carcinoma of the skin with complete local excision and resected carcinoma in situ of cervix.
    -Concomitant mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study, and/or evidence of poor compliance with medical instructions.
    -Use of the following medications as outlined:
    • Lanreotide (within 16 weeks before Screening)
    • Pasireotide LAR (within 24 weeks prior to Screening),
    • Pegvisomant (within 12 weeks before Screening),
    • Dopamine agonists (within 12 weeks before Screening),
    • Any combination of 2 or more acromegaly medications at Screening
    • Proton pump inhibitors (from start of Screening) until the end of the study
    -Current use of oral estrogen replacement therapy for <12 weeks prior to Screening.
    - Current use of medications that are strong inducers of CYP3A4 within 2 weeks prior to Screening
    - Known allergy or hypersensitivity to any of the test materials or related compounds.
    -Active COVID-19 confirmed or suspected based on clinical symptoms.
    -Symptomatic cholelithiasis.
    - Clinically significant concomitant disease including but not limited to cardiovascular disease, severe renal insufficiency, or significant liver disease (including cirrhosis).
    -Clinically significant abnormal findings during the Screening Period or any other medical condition(s) or laboratory findings that, in the opinion of the Investigator, might jeopardize the subject’s safety or ability to complete the study.
    -Systolic blood pressure >160 mmHg and/or diastolic blood pressure >100 mmHg during Screening.
    -Resting (at least 10 minutes) palpated pulse rate <45 bpm or >105 bpm during Screening. If either of these criteria is met, the assessment should be repeated 2 more times and the last assessment should be used to determine the subject’s eligibility.
    -QT interval corrected using Fridericia’s formula (QTcF) >480 msec (or corrected QT [QTc] interval >500 msec in the presence of complete bundle branch block) or PR interval >240 msec during Screening based on a central reading of an average of 3 ECGs each separated in time by approximately 1 minute after the subject has rested quietly in the supine position for at least 10 minutes without significant stimulation.
    -Female subjects who are pregnant or lactating. Subjects must have a negative pregnancy test during Screening and prior to the first dose of study drug.
    -Known history of, or current alcohol or drug abuse, within the last year.
    - Anamnesi di inefficacia o intolleranza significativa al trattamento con octreotide o lanreotide, come determinato dallo sperimentatore.
    - Anamnesi di radioterapia ipofisaria entro 3 anni dallo screening
    - Soggetti con insufficienza surrenalica, diabete insipido o ipogonadismo che non stanno ricevendo un'adeguata terapia ormonale sostitutiva al momento dello screening, come determinato dallo sperimentatore
    - Modello di tumore ipofisario ad alto rischio
    -Anamnesi di chirurgia maggiore/terapia chirurgica per qualsiasi causa entro 4 settimane prima dello screening.
    -Diabete mellito trattato con insulina per meno di 6 settimane prima dell'ingresso nello studio, o con cambiamento della dose totale giornaliera di insulina >15%
    entro 6 settimane prima dello screening.
    -Anamnesi di angina instabile o infarto miocardico acuto nelle 12 settimane precedenti la visita di screening o altra malattia cardiaca clinicamente significativa al momento dello
    screening, come giudicato dallo sperimentatore.
    -Anamnesi nota di epatite B o HIV, o epatite C attiva.
    -Neoplasia maligna attiva negli ultimi 5 anni con l'eccezione di carcinoma basocellulare e squamocellulare della pelle con locale completo escissione e carcinoma resecato in situ della cervice
    -Condizioni mentali concomitanti che lo rendano incapace di comprendere la natura, la portata e le possibili conseguenze dello studio, e/o prove di scarsa osservanza delle istruzioni mediche.
    -Uso dei seguenti farmaci come descritto:
    • Lanreotide (entro 16 settimane prima dello screening)
    • Pasireotide LAR (entro 24 settimane prima dello screening),
    • Pegvisomant (entro 12 settimane prima dello screening),
    • Agonisti della dopamina (entro 12 settimane prima dello screening),
    • Qualsiasi combinazione di 2 o più farmaci per l'acromegalia allo Screening
    • Inibitori della pompa protonica (dall'inizio dello Screening) fino alla fine dello studio
    - Uso attuale della terapia sostitutiva con estrogeni orali per <12 settimane prima dello screening.
    -Uso attuale di farmaci che sono forti induttori del CYP3A4 nelle 2 settimane prima dello screening
    - Allergia o ipersensibilità nota a uno qualsiasi dei materiali di prova o dei composti correlati.
    -COVID-19 attivo confermato o sospettato in base ai sintomi clinici.
    -Colelitiasi sintomatica.
    - Malattia concomitante clinicamente significativa inclusa, ma non limitata a, malattia cardiovascolare, grave insufficienza renale, o malattia epatica significativa (inclusa cirrosi).
    - Risultati anormali clinicamente significativi durante il periodo di screening o qualsiasi altra condizione medica o risultati di laboratorio che, a parere dello sperimentatore, potrebbero compromettere la sicurezza del soggetto o la la capacità di completare lo studio.
    - Pressione sanguigna sistolica >160 mmHg e/o pressione sanguigna diastolica
    >100 mmHg durante lo screening.
    - Polso palpato a riposo (almeno 10 minuti) <45 bpm o >105 bpm durante lo screening.
    -Intervallo QT corretto utilizzando la formula di Fridericia (QTcF) >480 msec (o intervallo QT [QTc] corretto >500 msec in presenza di completo blocco di branca) o intervallo PR >240 msec durante lo screening basato su una lettura centrale di una media di 3 ECG ciascuno separato nel tempo da circa 1 minuto dopo che il soggetto ha riposato tranquillamente in posizione supina per almeno 10 minuti senza stimolazione significativa.
    -Soggetti femminili in gravidanza o in allattamento.
    -Anamnesi nota di abuso di alcol o sostanze negli ultimi 12 mesi.
    E.5 End points
    E.5.1Primary end point(s)
    Proportion of subjects who achieve biochemical response in IGF-1 (<or=1.0× the upper limit of normal [ULN]) at the End of the Randomized Control Phase (EOR)
    Percentuale di soggetti che raggiungono una risposta biochimica a livello di IGF-1 (minore o uguale a 1.0× il limite superiore della norma [ULN]) alla fine della fase randomizzata e controllata (EOR)
    E.5.1.1Timepoint(s) of evaluation of this end point
    24 weeks for the primary endpoint.
    24 settimane per l'endpoint primario
    E.5.2Secondary end point(s)
    Change from baseline in IGF-1, in units of ULN, to EOR

    Proportion of subjects who achieve IGF-1 <1.3×ULN at EOR
    Proportion of subjects with GH <1 ng/mL at Week 22
    Change from baseline in Total Acromegaly Symptoms Diary (ASD) score to EOR
    variazione dalla baseline all'EOR nell'IGF-1, in unità ULN
    Percentuale di soggetti che raggiungono IGF-1 <1,3×ULN all' EOR
    Percentuale di soggetti con GH <1 ng/mL alla settimana 22
    Cambiamento dalla baseline all'EOR nel punteggio totale del Diario dei sintomi dell'acromegalia (ASD)
    E.5.2.1Timepoint(s) of evaluation of this end point
    24 weeks for the secondary endpoint.
    24 settimane per l'endpoint secondario.
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic Yes
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA31
    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
    Argentina
    Brazil
    China
    India
    Peru
    Russian Federation
    United States
    Bulgaria
    France
    Germany
    Greece
    Hungary
    Italy
    Poland
    Spain
    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
    Last visit of the last subject in the study
    L'ultima visita dell'ultimo soggetto nello studio
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 46
    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 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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 37
    F.4.2.2In the whole clinical trial 98
    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)
    An End of Study (EOS) Visit will occur at Week 124 to collect final safety data and other assessments as detailed in the SOAs. The EOS is defined as the date of the last visit of the last subject in the study.

    Subjects who complete the 24-week RC phase or who meet rescue criteria and complete the 24 week RC phase with rescue medication can continue participation in the OLE phase.
    Una visita di fine studio (EOS) avrà luogo alla settimana 124 per raccogliere i dati di sicurezza finali e altre valutazioni come specificato nelle SOAs. L'EOS è definita come la data dell'ultima visita dell'ultimo soggetto nello studio.
    I soggetti che completano la fase RC di 24 settimane o che soddisfano i criteri di salvataggio e completano la fase RC di 24 settimane con farmaci di salvataggio possono continuare a partecipare alla fase OLE.
    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 Decision2022-03-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-17
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Thu May 16 23:12:39 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA