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    Summary
    EudraCT Number:2021-006597-23
    Sponsor's Protocol Code Number:UX143-CL301
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Trial now transitioned
    Date on which this record was first entered in the EudraCT database:2022-09-14
    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 number2021-006597-23
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-controlled, Phase 2/3 Study to Assess the Efficacy and Safety of Setrusumab in Subjects with Osteogenesis Imperfecta
    Studio di fase 2/3 randomizzato, in doppio cieco, controllato con placebo per valutare l’efficacia e la sicurezza di setrusumab in soggetti con osteogenesi imperfetta
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to test product setrusumab in subjects with brittle bone syndrome
    Studio per testare setrusumab in soggetti con la sindrome delle ossa fragili
    A.3.2Name or abbreviated title of the trial where available
    N/A
    N/A
    A.4.1Sponsor's protocol code numberUX143-CL301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05125809
    A.5.4Other Identifiers
    Name:INDNumber:113385
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/241/2018
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorULTRAGENYX PHARMACEUTICAL 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 supportUltragenyx Pharmaceutical Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUltragenyx Pharmaceutical Inc.
    B.5.2Functional name of contact pointUX143-CL301 Global Operations
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato
    B.5.3.3Post codeCA 94949
    B.5.3.4CountryUnited States
    B.5.6E-mailUX143-CL301@ultragenyx.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/3/16/1686
    D.3 Description of the IMP
    D.3.1Product nameSetrusumab
    D.3.2Product code [BPS804, UX143]
    D.3.4Pharmaceutical form Lyophilisate for solution for 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.3.8INN - Proposed INNsetrusumab
    D.3.9.1CAS number 1847394-95-9
    D.3.9.2Current sponsor code.
    D.3.9.3Other descriptive nameUX143, BPS804
    D.3.9.4EV Substance CodeSUB193275
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number160
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    D.3.11.13.1Other medicinal product typeAnticorpo Monoclonale
    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 infusion in administration system
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Osteogenesis imperfecta (OI)
    Osteogenesi imperfetta
    E.1.1.1Medical condition in easily understood language
    Brittle bone syndrome
    Sindrome delle ossa fragili
    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 10031243
    E.1.2Term Osteogenesis imperfecta
    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
    Phase II: Identify a setrusumab dosing strategy in subjects with OI
    Phase III: Evaluate the effect of setrusumab vs placebo on reduction in fracture rate
    Fase II: Identificare una strategia posologica di setrusumab nei soggetti affetti da OI
    Fase III: Valutare l’effetto di setrusumab rispetto al placebo sulla riduzione della percentuale di fratture
    E.2.2Secondary objectives of the trial
    Phase II:
    - Evaluate the pharmacokinetics (PK) of setrusumab doses in subjects with OI
    - Determine the pharmacodynamic (PD) effects of setrusumab on bone formation and turnover markers
    - Evaluate the effect of setrusumab vs placebo on lumbar spine bone mineral density (BMD)
    - Evaluate the safety profile of setrusumab for the treatment of subjects with OI
    - Evaluate the immunogenicity of setrusumab for the treatment of subjects with OI

    Phase III:
    - Evaluate the effect of setrusumab vs placebo on fracture rate reduction
    - Evaluate the effect of setrusumab vs placebo on lumbar spine BMD
    - Evaluate the effect of setrusumab vs placebo on health-related quality of life, including physical health status
    - Assess the safety profile of setrusumab
    - Evaluate the immunogenicity of setrusumab for the treatment of subjects with OI
    Fase II:
    - Valutare la PK delle dosi di setrusumab in soggetti con OI
    - Determinare gli effetti PD di setrusumab sui marcatori di turnover e formazione ossea
    - Valutare l’effetto di setrusumab rispetto al placebo sulla BMD della colonna vertebrale
    - Valutare il profilo di sicurezza di setrusumab per il trattamento di soggetti con OI
    - Valutare l’immunogenicità di setrusumab per il trattamento di soggetti con OI

    Fase III:
    - Valutare l’effetto di setrusumab rispetto al placebo sulla riduzione della percentuale di fratture
    - Valutare l’effetto di setrusumab rispetto al placebo sulla BMD della colonna vertebrale
    - Valutare l’effetto di setrusumab rispetto al placebo sulla qualità della vita correlata alla salute, incluso lo stato di salute fisica
    - Valutare il profilo di sicurezza di setrusumab
    - Valutare l’immunogenicità di setrusumab per il trattamento di soggetti con OI
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Males and females 5 to < 26 years of age at time of informed consent
    2. Confirmed diagnosis of OI Types I, III, or IV as confirmed by identification of genetic mutation in COL1A1 or COL1A2
    3. >= 1 fracture in the past 12 months or >= 2 fractures in the past 24 months
    4. Serum 25-hydroxyvitamin D >= 20 ng/mL at the Screening Visit. If 25-hydroxyvitamin D levels are below the normal range, assuming a subject meets all other eligibility requirements, the subject may be rescreened after a minimum of 14 days of vitamin D supplementation as directed by their treating physician
    5. Willing to not receive bisphosphonate therapy during the study
    6. From the period following informed consent to 60 days after the last dose of study drug, females of childbearing potential and fertile males must consent to use highly effective contraception. If female, agree not to become pregnant. If male, agree not to father a child or donate sperm
    7. Willing and able to provide informed consent for subjects >= 18 years of age, or provide assent (if possible) and have a legally authorized representative provide informed consent, after the nature of the study has been explained and prior to any research-related procedures
    8. Willing to provide access to prior medical records for the collection of historical radiographic data, fracture data, growth data, and disease history
    9. Must, in the opinion of the Investigator, be willing and able to complete all aspects of the study, adhere to the study visit schedule, and comply with the assessments
    1. pazienti di sesso maschile o femminile da 5 a <26 anni di età al momento del consenso informato;
    2. diagnosi confermata di OI di tipo I, III o IV confermata dall’identificazione della mutazione genetica in COL1A1 o COL1A2;
    3. >=1 frattura negli ultimi 12 mesi o >=2 fratture negli ultimi 24 mesi;
    4. 25-idrossivitamina D sierica >=20 ng/ml alla Visita di screening. Se i livelli di 25-idrossivitamina D sono inferiori all’intervallo normale, presumendo che il soggetto soddisfi tutti gli altri requisiti di idoneità, il soggetto può essere riselezionato dopo almeno 14 giorni di integrazione di vitamina D come indicato dal medico curante;
    5. disponibilità a non ricevere terapia con bifosfonati durante lo studio;
    6. dal periodo successivo al consenso informato fino a 60 giorni dopo l’ultima dose del farmaco in studio, le donne in età fertile e gli uomini in grado di procreare devono acconsentire all’utilizzo di metodi contraccettivi altamente efficaci come descritto nell’Appendice 2. Se il soggetto è di sesso femminile, deve acconsentire a non iniziare una gravidanza. Se il soggetto è di sesso maschile, deve acconsentire a non procreare né donare lo sperma;
    7. disponibilità e capacità di fornire il consenso informato per soggetti di età >=18 anni o di fornire l’assenso (se possibile) e di ottenere il consenso informato di un rappresentante legale autorizzato, dopo che è stata spiegata la natura dello studio e prima di qualsiasi procedura correlata alla ricerca;
    8. disponibilità di fornire l’accesso alle precedenti cartelle cliniche per la raccolta dei dati radiografici storici, dati sulle fratture, dati sulla crescita e anamnesi della malattia;
    9. i pazienti devono, secondo il parere dello Sperimentatore, essere disposti e capaci di completare tutti gli aspetti dello studio, aderire al calendario delle visite dello studio e rispettare il piano delle valutazioni.
    E.4Principal exclusion criteria
    1. For Phase 2 subjects only, a history of major bone surgery within the previous 6 months prior to Screening or planned major bone surgery for new hardware placement for the first 3 months of the study
    2. History of skeletal malignancies or bone metastases at any time
    3. History of neural foraminal stenosis (except if due to scoliosis)
    4. History of or uncontrolled concomitant diseases such as hypo/hyperparathyroidism, Paget’s disease, abnormal thyroid function, thyroid disease or other endocrine disorders or conditions that could affect bone metabolism such as Stage IV/V renal disease
    5. Rickets or any skeletal condition (other than OI) leading to long-bone deformities and/or increased risk of fractures
    6. History of stroke, myocardial infarction, TIA, or angina. Investigators should consider whether the potential benefits of treatment outweigh the potential risks in patients with other cardiovascular risk factors such as hypertension, hyperlipidemia, familial hyperlipidemia, family history of premature ischemic cardiovascular disease, smoking, diabetes mellitus, and metabolic syndrome.
    7. Hypocalcemia, defined as serum calcium levels below the age-adjusted normal limits after a >= 4 hour fast
    8. Estimated glomerular filtration rate =< 29 mL/min/1.73 m2
    9. Prior treatment with the following:
    a. Bisphosphonates use prior to Screening within a period that is at least the length of the dosing interval of bisphosphonate use (eg, >= 3 months off bisphosphonates if bisphosphonate dosing was every 3 months). The required time for being off bisphosphonates should not exceed 6 months (eg, patients who were dosed with bisphosphonates every 12 months can enroll if they have not received bisphosphonates within 6 months prior to Screening)
    b. Teriparatide, growth hormone, or other anabolic or anti-resorptive medications within 6 months of Screening
    c. Denosumab within 24 months of Screening
    10. Documented alcohol and/or drug abuse within 12 months prior to dosing or evidence of such abuse as indicated by the laboratory results during the Screening assessments
    11. Presence or history of any condition that, in the view of the Investigator, would interfere with participation, pose undue risk, or would confound interpretation of results
    12. Known hypersensitivity to setrusumab or their excipients that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
    13. History of external radiation
    14. Pregnant or breastfeeding or planning to become pregnant (self or partner) at any time during the study
    15. Use of any investigational product (IP) or investigational medical device within 4 weeks or 5 half-lives of investigational drug (whichever is longer) prior to Screening, or during the study (per discretion of the Investigator in consultation with the Medical Monitor)
    16. Concurrent participation in another clinical study without prior approval from the Investigator in consultation with the Medical Monitor
    1. solo per i soggetti della fase II, un’anamnesi di intervento di chirurgia dei tratti ossei maggiore entro i 2 mesi precedenti lo Screening o intervento di chirurgia dei tratti ossei maggiore programmato per inserimento di nuovo impianto per i primi 3 mesi dello studio;
    2. anamnesi di neoplasie maligne scheletriche o metastasi ossee in qualsiasi momento;
    3. anamnesi di stenosi foraminale neurale (salvo se dovuta a scoliosi);
    4. anamnesi di o malattie concomitanti non controllate come ipo/iperparatiroidismo, malattia di Paget, funzione tiroidea anormale, malattia tiroidea o altri disturbi endocrini o condizioni che potrebbero avere effetti sul metabolismo osseo come malattia renale di stadio IV/V;
    5. rachitismo o qualsiasi patologia scheletrica (diversa dall’OI) che comporta deformità delle ossa lunghe e/o aumento del rischio di fratture;
    6. anamnesi di ictus, infarto miocardico, attacco ischemico transitorio o angina. Gli Sperimentatori devono valutare se i potenziali benefici del trattamento superano i potenziali rischi in pazienti con altri fattori di rischio cardiovascolari come ipertensione, iperlipidemia, iperlipidemia familiare, anamnesi familiare di malattia cardiovascolare ischemica prematura, fumo, diabete mellito e sindrome metabolica;
    7. ipocalcemia, definita come livelli di calcio sierico inferiori ai limiti normali adeguati secondo l’età dopo un digiuno di >=4 ore;
    8. tasso di filtrazione glomerulare stimata =< 29 ml/min/1,73 m2;
    9. trattamento precedente con:
    a. bifosfonati prima dello screening entro un periodo pari ad almeno la durata dell’intervallo di somministrazione dei bifosfonati (es., >=3 mesi senza bifosfonati se la somministrazione di bifosfonati avveniva una volta ogni 3 mesi). Il tempo richiesto di sospensione dei bifosfonati non deve superare i 6 mesi (es., i pazienti che ricevevano i bifosfonati ogni 12 mesi possono arruolarsi se non hanno ricevuto bifosfonati entro i 6 mesi precedenti lo screening);
    b. teriparatide, ormone della crescita o altri farmaci anabolici o anti-rarefacenti entro 6 mesi dallo screening;
    c. denosumab entro 24 mesi dallo screening;
    10. abuso documentato di alcol e/o farmaci entro i 12 mesi precedenti la somministrazione o evidenza di tale abuso indicata dai risultati di laboratorio durante le valutazioni di screening;
    11. presenza o anamnesi di qualsiasi condizione che, secondo il punto di vista dello Sperimentatore, interferirebbe con la partecipazione, creerebbe un rischio inutile o confonderebbe l’interpretazione dei risultati;
    12. nota ipersensibilità a setrusumab o ai suoi eccipienti che, a giudizio dello Sperimentatore, ponga il soggetto a un rischio maggiore di effetti avversi;
    13. anamnesi di radiazione esterna;
    14. gravidanza o allattamento o previsione di iniziare una gravidanza (il soggetto o la partner) in qualsiasi momento durante lo studio;
    15. utilizzo di qualsiasi medicinale sperimentale o dispositivo medico sperimentale entro 4 settimane o 5 emivite del farmaco sperimentale (a seconda di quale periodo è superiore) prima dello screening o durante lo studio (a discrezione dello Sperimentatore in accordo con il Monitor clinico);
    16. partecipazione concomitante a un altro studio clinico senza previa approvazione dello Sperimentatore in accordo con il Monitor clinico.
    E.5 End points
    E.5.1Primary end point(s)
    Phase II: Percent change in serum P1NP from Baseline at Month 1
    Phase III: Annualized rate of all radiographically-confirmed fractures, excluding morphometric vertebral fractures during the double-blind Treatment Period
    Fase II: Cambiamento percentuale del P1NP sierico dal Basale al Mese 1
    Fase III: Percentuale annualizzata di tutte le fratture confermate radiograficamente, escluse le fratture vertebrali morfometrichea, durante il Periodo di trattamento in doppio cieco
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase II: Screening and one month
    Phase III: Annually 12 and 24 months
    Fase II: Screening e 1 mese
    Fase III: Annualmente 12 e 24 mesi
    E.5.2Secondary end point(s)
    Phase II:
    1) Serum setrusumab concentration at scheduled time points
    2)
    • Baseline-corrected AUEC for serum P1NP over a 1- and 2-month timeframe
    • Percent change from Baseline in bone turnover markers (P1NP, CTX, BSAP, OCN) over time
    3)
    • Change from Baseline in DXA lumbar spine BMD z-scores over time
    • Percent change from Baseline in DXA lumbar spine BMD over time
    4) Frequency, severity, and relationship to treatment of TEAEs, SAEs, and AESIs
    5) Incidence of anti-setrusumab binding and neutralizing antibodies at scheduled time points

    Phase III:
    1) Annualized rate of all radiographically-confirmed fractures, including morphometric vertebral fractures, during the double-blind Treatment Period
    2) Change from Baseline in DXA lumbar spine BMD z-score at 18 months (or at 12 months if primary analysis occurs before all subjects have completed 18 months of treatment)
    3) Change from Baseline at 18 months (or at 12 months if primary analysis occurs before all subjects have completed 18 months of treatment) in:
    • PedsQL Physical Functioning Scale and Physical Health Summary Score for subjects < 18 years of age at screening
    • SF-36 PF and BP Domain Scale and PCS for subjects >= 18 years of age at screening
    4) Frequency, severity, and relationship to treatment of TEAEs, SAEs, and AESIs
    5) Incidence of binding and neutralizing anti-setrusumab antibodies at scheduled time points
    Fase II:
    1) Concentrazione sierica di setrusumab nei punti temporali previsti
    2)
    • AUEC corretta al basale per P1NP sierico in un intervallo di 1 e 2 mesi
    • Cambiamento percentuale dal Basale nei marcatori di turnover osseo (P1NP, CTX, BSAP, OCN) nel tempo
    3)
    • Cambiamenti rispetto al Basale degli z-score della BMD della colonna vertebrale con DXA nel tempo
    • Cambiamento percentuale rispetto al Basale della BMD della colonna vertebrale con DXA nel tempo
    4) Frequenza, gravità e relazione con il trattamento di TEAE, SAE e AESI
    5) Incidenza degli anticorpi neutralizzanti e leganti anti-setrusumab nei punti temporali previsti

    Fase III:
    1) Percentuale annualizzata di tutte le fratture confermate radiograficamente, incluse le fratture vertebrali morfometrichea, durante il Periodo di trattamento in doppio cieco
    2) Cambiamento rispetto al Basale degli z-score della BMD della colonna vertebrale con DXA a 18 mesi (o a 12 mesi se l’analisi primaria avviene prima che tutti i soggetti abbiano completato 18 mesi di trattamento)
    3) Cambiamento rispetto al Basale a 18 mesi (o a 12 mesi se l’analisi primaria avviene prima che tutti i soggetti abbiano completato 18 mesi di trattamento) in:
    • scala di funzionamento fisico PedsQL e punteggio riassuntivo della salute fisica per soggetti <18 anni di età allo screening;
    • scala del dominio BP e PF SF-36 e PCS per soggetti >=18 anni di età allo screening.
    4) Frequenza, gravità e relazione con il trattamento di TEAE, SAE e AESI
    5) Incidenza degli anticorpi anti-setrusumab neutralizzanti e leganti nei punti temporali previsti
    E.5.2.1Timepoint(s) of evaluation of this end point
    Phase II: setrusumab concentration is measured at baseline, wk 2,3,4, month 1, day 45, month 2, 3, 6, 12, 18 and 24.

    Phase III: setrusumab concentration is measured at baseline, week 3, months 1, 2, 3, 6, 12, 18 and 24
    - Serum P1NP, CTX, BSAP, OCN at baseline, week 3 and months 1, 2, 3, 6, 12, 18, 24.
    - Anti-setrusumab binding and neutralizing antibodies at screening, week 3 and months 1, 2, 3, 6, 12, 18, 24.
    - DXA at 0, 3,6,12, 18 months and 24 months (final visit).
    - PedsQL Physical Functioning Scale measured at 0, 6, 12, 18 and 24 months/final visit, whichever is sooner
    - Physical Health Summary Score and SF-36 PF and BP Domain Scale and PCS at 18 months (or 12 months)
    - TEAEs, SAEs, and AESIs - all study visits
    Fase II: La concentrazione di setrusumab è misurata al basale, settimana 2,3,4, mese 1, giorno 45, mese 2, 3, 6, 12, 18 e 24.

    Fase III: la concentrazione di setrusumab viene misurata al basale, settimana 3, mesi 1, 2, 3, 6, 12, 18 e 24
    - Siero P1NP, CTX, BSAP, OCN al basale, settimana 3 e mesi 1, 2, 3, 6, 12, 18, 24.
    - Anticorpi leganti e neutralizzanti anti-setrusumab allo screening, settimana 3 e mesi 1, 2, 3, 6, 12, 18, 24.
    - DXA a 0, 3,6,12, 18 mesi e 24 mesi (visita finale).
    - PedsQL Physical Functioning Scale misurata a 0, 6, 12, 18 e 24 mesi/visita finale, a seconda di quale sia la prima
    - Punteggio riepilogativo della salute fisica e scala di dominio SF-36 PF e BP e PCS a 18 mesi (o 12 mesi)
    - TEAE, SAE e AESI - tutte le visite di studio
    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 Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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
    Immunogenicity
    Immunogenicità
    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) Yes
    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 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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial3
    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 EEA8
    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
    Australia
    Canada
    United States
    France
    Germany
    Italy
    Denmark
    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 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) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 124
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 66
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 41
    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 Yes
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Pediatric subjects incapable of giving consent
    Soggetti pediatrici incapaci di dare il consenso
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state27
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 71
    F.4.2.2In the whole clinical trial 231
    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)
    All subjects (Phase 2 and Phase 3) will transition to an open-label Treatment Extension Period after the primary analysis is complete, or once a subject has completed 24 months of treatment in the double-blind Treatment Period, whichever is sooner. All subjects, including those who received placebo in the double-blind Treatment Period, will be treated with setrusumab in the open-label Treatment Extension Period.
    Tutti i soggetti (Fase II e Fase III) passeranno a un periodo di estensione del trattamento in aperto dopo il completamento dell'analisi primaria o una volta che un soggetto ha completato 24 mesi di trattamento nel periodo in doppio cieco, a seconda di quale evento si verifica prima. Tutti i soggetti, compresi quelli che hanno ricevuto placebo nel periodo di trattamento in doppio cieco, saranno trattati con setrusumab nel periodo di estensione del trattamento in aperto.
    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 Decision2022-10-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-02-01
    P. End of Trial
    P.End of Trial StatusTrial now transitioned
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