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    Summary
    EudraCT Number:2021-002244-70
    Sponsor's Protocol Code Number:CLIN-60120-452
    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:2021-11-23
    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 number2021-002244-70
    A.3Full title of the trial
    Rollover Study; Multicentre, Phase III, Open-label Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged >=14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed Study PVO-1A-301 or PVO-1A-202/PVO-1A-204 and May Benefit from Palovarotene Therapy.
    Studio di rollover di fase III, multicentrico, in aperto, per valutare ulteriormente la sicurezza el’efficacia di palovarotene capsule in partecipanti di entrambi i sessi e di età >= 14 anni con fibrodisplasia ossificante progressiva (FOP) che hanno completato lo studio PVO-1A-301 o lo studio PVO-1A-202/PVO-1A-204 e che potrebbero trarre beneficio dal trattamento con palovarotene.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Rollover Study to Further Evaluate the Safety and Efficacy of Palovarotene Capsules in Male and Female Participants Aged >=14 Years with Fibrodysplasia Ossificans Progressiva (FOP) Who Have Completed the Relevant Parent Studies.
    Studio di rollover per valutare ulteriormente la sicurezza e l’efficacia di palovarotene capsule nei partecipanti di sesso maschile e femminile di età >=14 anni con fibrodisplasia ossificante progressiva (FOP) che hanno completato i relativi studi originali.
    A.3.2Name or abbreviated title of the trial where available
    PIVOINE
    PIVOINE
    A.4.1Sponsor's protocol code numberCLIN-60120-452
    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 SponsorIPSEN PHARMA SAS
    B.1.3.4CountryFrance
    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 supportIpsen Pharma SAS
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIPSEN Pharma SAS
    B.5.2Functional name of contact pointMedical Affairs Clinical Operations
    B.5.3 Address:
    B.5.3.1Street Address65, quai Georges Gorse
    B.5.3.2Town/ cityBoulogne-Billancourt
    B.5.3.3Post code92100
    B.5.3.4CountryFrance
    B.5.4Telephone number33761566463
    B.5.6E-mailBayane.Tannir@ipsen.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/3/14/1368
    D.3 Description of the IMP
    D.3.1Product namePalovarotene
    D.3.2Product code [IPN60120]
    D.3.4Pharmaceutical form Capsule, hard
    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 INNPalovarotene
    D.3.9.1CAS number 410528-02-8
    D.3.9.2Current sponsor codeIPN60120
    D.3.9.4EV Substance CodeSUB75998
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number1 to 10
    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 Yes
    D.2.1.1.1Trade name IDROCORTISONE ACETATO DYNACREN - 1 % CREMA 1 TUBO DA 30 G
    D.2.1.1.2Name of the Marketing Authorisation holderDYNACREN Laboratorio Farmaceutico del Dr. A. Francioni e di M. Gerosa s.r.l.
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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 nameIDROCORTISONE ACETATO DYNACREN 1% CREMA
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Cream
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPCutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNACIDO FUSIDICO/IDROCORTISONE ACETATO
    D.3.9.1CAS number 50-03-3
    D.3.9.2Current sponsor code-
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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
    Fibrodysplasia Ossificans Progressiva (FOP)
    Fibrodiplasia Ossificante Progressiva (FOP)
    E.1.1.1Medical condition in easily understood language
    Fibrodysplasia Ossificans Progressiva
    Fibrodisplasia Ossificante Progressiva
    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 10068715
    E.1.2Term Fibrodysplasia ossificans progressiva
    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
    To further evaluate the safety of palovarotene in adult and paediatric participants with FOP.
    Valutare ulteriormente la sicurezza del palovarotene nei partecipanti adulti e pediatrici affetti da FOP.
    E.2.2Secondary objectives of the trial
    • To describe range of motion, as assessed by the Cumulative Analogue Joint Involvement Scale (CAJIS) for FOP, at the Inclusion Visit and over time under palovarotene treatment.
    • To describe the use of assistive devices and adaptations for daily living by FOP participants, at the Inclusion Visit and over time under palovarotene treatment.
    • To describe physical function, using the adult form of the FOP Physical Function Questionnaire, at the Inclusion Visit and over time under palovarotene treatment.
    • To describe the FOP healthcare utilization in patients with FOP.
    • To describe the parameters of lung function at the Inclusion Visit and over time under palovarotene treatment.
    • To describe physical and mental health, using the adult form of the Patient Reported Outcomes Measurement Information System Global Health Scale, at the Inclusion Visit and over time under palovarotene treatment.

    Please refer to protocol for other secondary objectives.
    •Descrivere il raggio di movimento, come valutato dalla Scala analogica cumulativa del coinvolgimento articolare (CAJIS) per la FOP, alla visita di inclusione e durante il periodo di trattamento con il palovarotene.
    •Descrivere l'uso di dispositivi di assistenza e adattamenti per la vita quotidiana da parte dei partecipanti con FOP, alla visita di inclusione e durante il periodo di trattamento con il palovarotene.
    •Descrivere la funzione fisica, utilizzando il formulario per adulti del Questionario sulla funzione fisica per la FOP, alla visita di inclusione e durante il periodo di trattamento con il palovarotene.
    •Descrivere l’utilizzo delle cure sanitarie per la FOP in pazienti affetti da FOP.
    •Descrivere i parametri della funzione polmonare alla visita di inclusione e durante il periodo di trattamento con il palovarotene.

    Si prega di far riferimento al protocollo per altri obiettivi secondari.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Participants are eligible to be included in the study only if all of the following criteria apply:
    1. Participant has completed the EOS or End of Treatment Visit of Study PVO-1A-301 or PVO-1A-202 (PVO-1A-202 Parts C and D correspond to Study PVO-1A-204 in France) and did not previously withdraw consent from any of the parent studies to be eligible for Study CLIN-60120-452.
    2. In the investigator’s judgment, the participant may benefit from continued participation in a palovarotene study.

    Age
    3. Participant must be >=14 years of age (aligned with the age of treated participants in the ongoing parent studies PVO-1A-301 and PVO-1A-202/PVO-1A-204) and qualify as 100% skeletally mature (if <18 years, based on assessments carried out at parent EOS Visit; if =18 years, automatically considered 100% skeletally mature) or have reached final adult height based on investigator’s assessment*, at the time the Study CLIN60120-452 informed consent is signed.
    *The above criteria can be reached at any point after parent study end for the participant to be eligible for Study CLIN-60120-452, as long as it is prior to palovarotene becoming commercially available in the country where the study is being conducted.

    Sex
    4. Male, or female who is not pregnant or breastfeeding, and has met all of the following conditions:
    • Females of child-bearing potential (FOCBP) (defined in Section 10.2.1 of the protocol) must have a negative blood or urine pregnancy test (with sensitivity of at least 50 mIU/mL) prior to administration of palovarotene;
    • FOCBP must agree to remain abstinent from heterosexual sex during treatment and for one month after treatment or, if sexually active, to use two effective methods of birth control (described in Section 10.2.2 of the protocol) during and for one month after treatment;
    Additionally, sexually active FOCBP must already be using two effective methods of birth control one month before treatment is to start;
    • Specific risk of the use of retinoids during pregnancy, and the agreement to remain abstinent or use two effective methods of birth control will be clearly defined in the informed consent and the participant or legally authorised representatives (e.g. parents, caregivers or legal guardians) must specifically sign this section.
    Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.

    Informed Consent and Assent
    5. Capable of giving signed informed consent or providing assent as described in Section 10.1.3 of the protocol.
    I partecipanti possono essere inclusi nello studio soltanto se soddisfano tutti i seguenti criteri:
    1. Il/la partecipante ha completato l'EOS o la Visita di fine trattamento dello studio PVO-1A-301 o PVO-1A-202 (le parti C e D del PVO-1A-202 corrispondono allo studio PVO-1A-204 in Francia) e non ha precedentemente ritirato il modulo di consenso di uno qualsiasi degli studi principali per essere idoneo/a allo studio CLIN-60120-452.
    2. A giudizio dello sperimentatore, il/la partecipante può trarre beneficio partecipando alla continuazione di uno studio sul palovarotene.
    Età
    3. Il/la partecipante deve avere un'età >= a 14 anni (in linea con l'età dei partecipanti trattati durante gli studi principali in corso PVO-1A-301 e PVO-1A- 202/PVO-1A-204) e deve essere qualificato/a come scheletricamente maturo/a al 100% (se <18 anni, sulla base delle valutazioni effettuate alla visita dell’EOS principale; se =18 anni, è considerato/a in automatico scheletricamente maturo/a al 100%) o ha raggiunto l'altezza adulta finale in base alla valutazione dello sperimentatore*, al momento della firma del modulo di consenso per lo studio CLIN60120-452.
    *I criteri di cui sopra possono essere raggiunti in qualsiasi momento dopo la fine dello studio principale affinché il partecipante sia idoneo allo studio CLIN-60120-452, purché avvenga anteriormente alla messa in commercio di palovarotene nel paese in cui lo studio è condotto.
    Sesso
    4. Di sesso maschile, o femminile non in stato di gravidanza o allattamento e che ha soddisfatto tutte le seguenti condizioni:
    • Le donne potenzialmente fertili (come definito nella sezione 10.2.1 del protocollo) devono avere un test di gravidanza su sangue o urine negativo (con sensibilità di almeno 50 mUI/mL) precedente alla somministrazione di palovarotene;
    • Le donne potenzialmente fertili devono acconsentire all’astinenza dall’attività eterosessuale durante il trattamento e per un mese dopo il trattamento o, se sessualmente attive, di utilizzare due metodi contraccettivi efficaci (descritti nella sezione 10.2.2 del protocollo) durante e per un mese dopo il trattamento;
    • Inoltre, le donne potenzialmente fertili sessualmente attive devono utilizzare due metodi contraccettivi efficaci già un mese prima dell'inizio del trattamento;
    • Il rischio specifico dell'uso dei retinoidi durante la gravidanza e il consenso all’astinenza sessuale o utilizzo di due metodi contraccettivi efficaci saranno chiaramente definiti nel consenso informato e le partecipanti o i rappresentanti legalmente autorizzati (ad es. genitori, caregiver o tutori legali) devono specificamente firmare questa sezione. L'uso di contraccettivi da parte delle donne deve essere coerente con le normative locali relative ai metodi di contraccezione per coloro che partecipano a studi clinici.
    Assenso e consenso informato
    5. Essere in grado di fornire il consenso informato firmato o fornire l'assenso come descritto nella sezione 10.1.3 del protocollo.
    E.4Principal exclusion criteria
    Participants will not be included in the study if any of the following criteria apply:

    Medical conditions
    1. History of allergy or hypersensitivity to retinoids, gelatin, lactose (note that lactose intolerance is not exclusionary) or palovarotene, or unresponsiveness to prior treatment with palovarotene.
    2. Uncontrolled cardiovascular, hepatic, pulmonary, gastrointestinal, endocrine, metabolic, ophthalmologic, immunologic, psychiatric, or other significant disease.
    3. Intercurrent known or suspected non-healed fracture at any location;
    4. Any other medical condition/clinically significant abnormalities that would expose the participant to undue risk or interfere with study assessments.
    5. Amylase or lipase >2× above the upper limit of normal (ULN) or with a history of chronic pancreatitis.
    6. Elevated aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >2.5× ULN.
    7. Fasting triglycerides >400 mg/dL with or without therapy.
    8. Suicidal ideation (type 4 or 5) or any suicidal behaviour at Inclusion Visit as defined by the Columbia-Suicide Severity Rating Scale (C-SSRS).

    Prior/concomitant therapy
    9. Current use of vitamin A or beta carotene, multivitamins containing vitamin A or beta carotene, or herbal preparations, fish oil, and unable or unwilling to discontinue use of these products during palovarotene treatment.
    10. Exposure to synthetic oral retinoids other than palovarotene within 4 weeks of the Inclusion Visit.
    11. Concurrent treatment with tetracycline or any tetracycline derivatives due to the potential increased risk of pseudotumor cerebri.
    12. Use of concomitant medications that are strong inhibitors or inducers of cytochrome P450 (CYP450) 3A4 activity (see Section 6.8 and 10.5 of protocol); or kinase inhibitors such as imatinib.

    Other exclusion criteria
    13. Palovarotene is commercially available in the country where the study is being conducted.
    14. Any reason that, in the opinion of the investigator, would lead to the inability of the participant and/or family to comply with the protocol.
    Non saranno inclusi nello studio i partecipanti a cui si applica uno qualsiasi dei seguenti criteri:
    Condizioni cliniche
    1. Anamnesi di allergia o ipersensibilità a retinoidi, gelatina, lattosio (si noti che l'intolleranza al lattosio non è un criterio di esclusione) o palovarotene, o mancata risposta al precedente trattamento con palovarotene.
    2. Malattie non controllate cardiovascolari, epatiche, polmonari, gastrointestinali, endocrine, metaboliche, oftalmologiche, immunologiche, psichiatriche o altre malattie significative.
    3. Frattura intercorrente nota o sospetta non cicatrizzata in qualsiasi parte del corpo;
    4. Qualsiasi altra condizione medica/anomalia clinicamente significativa che esponga il/la partecipante a rischi indebiti o che interferisca con le valutazioni dello studio.
    5. Amilasi o lipasi >2 volte al di sopra del limite superiore alla norma (ULN) o con una storia di pancreatite cronica.
    6. Aspartato aminotransferasi (AST) elevata o alanina aminotransferasi (ALT) 2,5 volte superiore all’ULN.
    7. Trigliceridi a digiuno >400 mg/dL con o senza terapia.
    8. Pensieri suicidari (tipo 4 o 5) o qualsiasi comportamento suicidario rilevato alla visita di inclusione come definito dalla Scala della Columba University per la valutazione della gravità del suicidio (Columbia-Suicide Severity Rating Scale - C-SSRS).

    Terapia precedente/concomitante
    9. Uso attuale di vitamina A o beta carotene, multivitaminici contenenti vitamina A o beta carotene, o preparati a base di erbe, olio di pesce la cui interruzione non è possibile o non è voluta dal/dalla paziente durante il trattamento con palovarotene.
    10. Esposizione a retinoidi sintetici di uso orale diversi dal palovarotene nelle 4 settimane precedenti alla visita di inclusione.
    11. Trattamento concomitante con tetraciclina o qualsiasi derivato della tetraciclina a causa del potenziale aumento del rischio di pseudotumor cerebri.
    12. Uso concomitante di farmaci che sono potenti inibitori o induttori dell'attività del citocromo P450 (CYP450) 3A4 (consultare i paragrafi 6.8 e 10.5 del protocollo); o inibitori della chinasi, come imatinib
    Ulteriori criteri di esclusione
    13. Il palovarotene si trova disponibile in commercio nel paese in cui lo studio è condotto.
    14. Qualsiasi motivo che, a giudizio dello sperimentatore, potrebbe condurre all'incapacità del/della partecipante e/o della famiglia di rispettare il protocollo.
    E.5 End points
    E.5.1Primary end point(s)
    • Incidence and description of all treatment emergent adverse events (TEAEs) whether or not they are considered as related to the study intervention;
    • Incidence and description of all serious and nonserious treatment-related TEAEs;
    • Incidence and description of all serious TEAEs, whether or not they are considered as related to the study intervention;
    • Incidence and description of all nonserious TEAEs whether or not they are considered as related to the study intervention.
    • Incidenza e descrizione di tutti gli eventi avversi originati dal trattamento (TEAE), indipendentemente dal fatto che siano considerati correlati all'intervento dello studio;
    • Incidenza e descrizione di tutti i TEAE gravi e non gravi correlati al trattamento;
    • Incidenza e descrizione di tutti i TEAE gravi, indipendentemente dal fatto che siano considerati correlati all'intervento dello studio;
    • Incidenza e descrizione di tutti i TEAE non gravi, indipendentemente dal fatto che siano considerati correlati all'intervento dello studio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    At Inclusion Visit (Day 1) and then every 6 months at Follow-up Visits until End of Study or Early Withdrawal.
    Alla visita di inclusione (giorno 1) e successivamente a ogni 6 mesi alle visite di follow-up fino alla fine dello studio o al ritiro anticipato.
    E.5.2Secondary end point(s)
    • Raw values and change from the Inclusion Visit in CAJIS total score at each Follow-up Visit;
    • Raw values and shift from the Inclusion Visit in the use of assistive devices and adaptations for daily living at each Follow-up Visit;
    • Raw values and change from the Inclusion Visit in % of worst score for total score, upper extremities subscore and mobility subscore using the adult form of the FOP-PFQ for all participants, at each Follow-up Visit;
    • Type and frequency of healthcare utilization
    • Raw values and change from the Inclusion Visit in observed and % predicted FVC at each Follow-up Visit;
    • Raw values and change from the Inclusion Visit in observed and % predicted FEV1 at each Follow-up Visit;
    • Raw values and change from the Inclusion Visit in absolute and % predicted FEV1/FVC ratio at each Follow-up Visit;
    • Raw values and change from the Inclusion Visit in observed and % predicted DLCO at each Follow-up Visit;
    • Raw values and change from the Inclusion Visit in physical and mental function (mean global physical and mental health score converted into T-scores), using the adult form of the PROMIS Global Health Scale for all participants, at each Follow-up Visit;
    • Raw values and change from the Inclusion Visit in number of investigator-reported flareups, flare-up outcomes (new bone growth, restricted movement) and flare-up duration at each Follow-up Visit by body location and overall;
    • Percentage of participants with new bone growth overall and by flare-up status at each Follow-up Visit;
    ; • Valori grezzi e variazione rispetto alla visita di inclusione nel punteggio totale del CAJIS a ogni visita di follow-up;
    • Valori grezzi e spostamento rispetto alla Visita di inclusione quanto all’uso di dispositivi di assistenza e adattamenti per la vita quotidiana a ogni Visita di follow-up;
    • Valori grezzi e variazione rispetto alla Visita di inclusione in % del punteggio peggiore per il punteggio totale, il sottopunteggio degli arti superiori e il sottopunteggio della mobilità utilizzando il questionario per adulti FOP-PFQ per tutti i partecipanti, a ogni Visita di follow-up;
    • Tipo e frequenza di utilizzo dell'assistenza sanitaria
    • Valori grezzi e variazione rispetto alla Visita di inclusione della FVC osservata e percentualmente prevista a ogni Visita di follow-up;
    • Valori grezzi e variazione rispetto alla Visita di inclusione della FEV1 osservata e percentualmente prevista a ogni Visita di follow-up;
    • Valori grezzi e variazione rispetto alla Visita di inclusione della FEV1/FVC assoluta e percentualmente prevista a ogni Visita di follow-up;
    • Valori grezzi e variazione rispetto alla Visita di inclusione della DLCO osservata e percentualmente prevista a ogni Visita di follow-up;
    • Valori grezzi e variazione rispetto alla Visita di inclusione nella funzione fisica e mentale (punteggio medio globale di salute fisica e mentale convertito in punteggi T), utilizzando il modulo per adulti della Scala di valutazione della salute globale PROMIS per tutti i partecipanti, a ogni Visita di follow-up;
    • Valori grezzi e variazione rispetto alla Visita di inclusione nel numero di riacutizzazioni segnalate dallo sperimentatore, esiti di riacutizzazioni (nuova crescita ossea, movimento limitato) e durata della riacutizzazione a ciascuna Visita di follow-up tramite la posizione corporea e lo stato complessivo;
    • Percentuale di partecipanti con nuova crescita ossea complessiva e per stato di riacutizzazione ad ogni Visita di follow-up;
    E.5.2.1Timepoint(s) of evaluation of this end point
    At Inclusion Visit (Day 1) and then every 6 months at Follow-up Visits until End of Study or Early Withdrawal.; Alla visita di inclusione (giorno 1) e successivamente a ogni 6 mesi alle visite di follow-up fino alla fine dello studio o al ritiro anticipato.
    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 No
    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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA4
    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
    Australia
    Argentina
    Brazil
    Canada
    France
    Italy
    Spain
    Sweden
    United Kingdom
    United States
    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 Patient Last Visit (LPLV)
    Ultima Visita dell'ultimo Paziente (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) 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: 33
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 53
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 1
    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 state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 18
    F.4.2.2In the whole clinical trial 87
    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)
    Participants may continue to receive palovarotene once it becomes commercially available or when another access programme becomes available, whichever is sooner.
    I partecipanti possono continuare a ricevere palovarotene una volta che sarà disponibile in commercio o quando sarà disponibile un altro programma di accesso, a seconda di quale evento si verifichi per primo.
    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 Decision2022-02-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-02-21
    P. End of Trial
    P.End of Trial StatusOngoing
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