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    Summary
    EudraCT Number:2011-002516-85
    Sponsor's Protocol Code Number:01-2011
    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:2011-12-28
    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 number2011-002516-85
    A.3Full title of the trial
    Open-label study to evaluate the efficacy and tolerability of orally administered rosiglitazone in patients with progressive bone Fibrodysplasia (FOP)
    Studio in aperto per valutare l'efficacia e la tollerabilita' di rosiglitazone somministrato per via orale in pazienti affetti da fibrodisplasia ossea progressiva (FOP)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Open-label study to evaluate the efficacy and tolerability of orally administered rosiglitazone in patients with progressive bone Fibrodysplasia (FOP)
    Studio in aperto per valutare l'efficacia e la tollerabilita' di rosiglitazone somministrato per via orale in pazienti affetti da fibrodisplasia ossea progressiva (FOP)
    A.4.1Sponsor's protocol code number01-2011
    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 SponsorA.O. UNIVERSITARIA INTEGRATA DI VERONA
    B.1.3.4CountryItaly
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportGLAXOSMITHKLINE
    B.4.2CountryItaly
    B.4.1Name of organisation providing supportUniversita' di Verona (fondi per i dosaggi non previsti dalla pratica clinica)
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAzienda Ospedaliera Universitaria Integrata Verona
    B.5.2Functional name of contact pointSSO di Reumatologia OCM
    B.5.3 Address:
    B.5.3.1Street AddressPiazzale Stefani 1
    B.5.3.2Town/ cityVerona
    B.5.3.3Post code37100
    B.5.3.4CountryItaly
    B.5.4Telephone number045-6338607
    B.5.5Fax number045-6338686
    B.5.6E-maildavide.gatti@univr.it
    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 nameRosiglitazone
    D.3.4Pharmaceutical form Coated 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 INNROSIGLITAZONE MALEATE
    D.3.9.1CAS number 155141-29-0
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number4
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy 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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameRosiglitazone
    D.3.4Pharmaceutical form Coated 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 INNROSIGLITAZONE MALEATE
    D.3.9.1CAS number 155141-29-0
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number8
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy 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 Progressive (FOP) is a rare genetically based disability, characterized by progressive heterotopic ossification, usually associated with congenital malformation of the toes.
    La Fibrodisplasia Ossificante Progressiva (FOP) è una malattia rara su base genetica invalidante, caratterizzata da ossificazione eterotopica progressiva, tipicamente associata a malformazione congenita degli alluci.
    E.1.1.1Medical condition in easily understood language
    Fibrodysplasia Ossificans Progressive (FOP) is a rare genetically based disability characterized by progressive heterotopic ossification.
    La Fibrodisplasia Ossificante Progressiva (FOP) è una malattia rara su base genetica invalidante caratterizzata da ossificazione eterotopica 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 14.1
    E.1.2Level HLGT
    E.1.2Classification code 10028393
    E.1.2Term Musculoskeletal and connective tissue disorders NEC
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue 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 verify the safety and efficacy of therapy with Rosiglitazone in patients with FOP evaluating the range variation of the joint mobility of wrists, elbows, shoulders, hips, knees and ankles using both a scale from 1(free joint) to 4 (locked joint) and a measure with a protractor; To evaluate a path-test of 10 metres
    Verificare efficacia e tollerabilità della terapia con Rosiglitazone in pazienti affetti da FOP attraverso la valutazione della variazione del range di mobilità articolare di polsi, gomiti, spalle, anche, ginocchia e caviglie sia mediante una scala da 1 (articolazione completamente libera) a 4 (articolazione completamente bloccata) che mediante misura con goniometro; test al cammino su 10 metri
    E.2.2Secondary objectives of the trial
    To evaluate changes in quality of life through the SF36 questionnaire; to evaluate changes of the bone turnover biomarkers concentration during the therapy; to evaluate changes of occiput-wall, finger-floor and bimalleolar distances; to monitor the ectopic ossifications (subgroup) size using an echographic evaluation.
    Valutare le modifiche della qualità di vita valutata con SF36; variazione dei markers di turnover osseo durante terapia; variazione delle distanze occipite-muro, dito-pavimento e bi malleolare; Valutazione ecografica per monitorare la dimensione delle ossificazioni ectopiche (sottogruppo)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    ·patients of both sexes, older than 18 ·patients affected by FOP genetically confirmed ·patients with a joint mobility limitation > grade 2 (on a scale of 4 points) on at least one joint ·written informed consent signed by the patient or by a legally acceptable representative
    ·Pazienti di ambo i sessi, di età superiore a 18 anni; ·Pazienti con Fibrodisplasia Ossea Progressiva confermata geneticamente ·Limitazione della mobilità articolare &gt; grado 2 su una scala a 4 punti su almeno 1 articolazione; ·Firma del consenso informato da parte del paziente o di un suo rappresentante legalmente riconosciuto
    E.4Principal exclusion criteria
    ·Patients affected by Bone Fibrodysplasia other than FOP ·Patients affected by heart failure, ischemic heart disease or with impaired liver function (ALT> 2 times the upper limit of normal) ·Patients under 18 ·Known hypersensitivity to the drug or the excipients 3.Pregnant or lactating women 4.alcoholism 5.drug dependence
    ·Pazienti affetti da fibrodisplasia ossea diversa dalla FOP ·Pazienti affetti da insufficienza cardiaca, cardiopatia ischemica o con compromissione della funzionalità epatica (transaminasi &gt; 2 volte range di normalità).·Pazienti con età inferiore a 18 anni ·Ipersensibilità nota al farmaco o agli eccipienti. ·Donne in gravidanza o in allattamento; ·Alcoolismo; ·Farmaco-dipendenza
    E.5 End points
    E.5.1Primary end point(s)
    1)To evaluate the range of motion of wrists, elbows, shoulders, hips, knees, ankles before beginning treatment and after the end of treatment (follow-up) using a scale from 1 (free joint) to 4 (locked joint); 2)To evaluate the range of motion of wrists, elbows, shoulders, hips, knees, ankles before beginning treatment and after the end of treatment (follow-up) using a protractor (measured in degrees); 3)To record the time the patient spent (seconds) to perform the 10 metres-path test at the end of treatment (last visit).
    1)range di mobilità di a) polsi b) gomiti c) spalle d) anche e) ginocchia f) caviglie rilevato alla visita basale e alle successive visite di follow-up misurato tramite scala ordinale con punteggi da 1 (articolazione completamente libera) a 4 (articolazione completamente bloccata); 2)range di mobilità di a) polsi b) gomiti c) spalle d) anche e) ginocchia f) caviglie rilevato alla visita basale e alle successive visite di follow-up misurata in gradi (tramite l’utilizzo di un goniometro); 3)tempo impiegato (in secondi) per compiere il test del cammino dei 10 metri rilevato alla visita finale.
    E.5.1.1Timepoint(s) of evaluation of this end point
    nd
    nd
    E.5.2Secondary end point(s)
    1)to evaluate the quality of life using the SF-36 questionnaire before beginning treatment and at the end of treatment (follow-up); 2)To assess the bone turnover biomarkers (ALP(U/I), bone ALP (U/I), sCTX (mcg/l), PHT (pg/ml), DKK1 (pmol/l), osteocalcin (nmol/l), sclerostin (pmol/l), P1NP (ng/ml), Vit D (nmol/l)) concentration before beginning treatment and at the end of treatment (follow-up); 3)To record the occiput-wall distance (cm) before beginning treatment and at the end of treatment (follow-up); 4)To record the finger-floor distance (cm) before beginning treatment and at the end of treatment (follow-up); 5)To record the bimalleolar distance (cm) before beginning treatment and at the end of treatment (follow-up); 6)To evaluate the ectopic ossifications size (diameter in mm) using an echographic evaluation before beginning treatment and at the end of treatment (follow-up) (only in patients whose disease has not affected the traditional echographic windows)
    1)Punteggio della qualità della vita (misurato tramite questionario SF-36) rilevato alla visita basale e alle successive visite di follow-up; 2)Concentrazione dei markers di turnover osseo (ALP (U/I), bone ALP (U/I), sCTX (mcg/l), osteocalcina (nmol/l), PTH (pg/ml), DKK1 (pmol/l), Sclerostina (pmol/l), P1NP (ng/ml), Vit D (nmol/l)) rilevata alla visita basale e alle successive visite di follow-up; 3)Distanza (in cm) occipite-muro alla visita basale e alle successive visite di follow-up; 4)Distanza (in cm) dito-pavimento alla visita basale e alle successive visite di follow-up; 5)Distanza bi malleolare (in cm) alla visita basale e alle successive visite di follow-up; 6)Dimensione delle ossificazioni ectopiche (diametri in mm) tramite ecografia alla visita basale e alle successive visite di follow-up (solo nel sottogruppo di pazienti in cui la malattia non abbia compromesso le finestre ecografiche tradizionali)
    E.5.2.1Timepoint(s) of evaluation of this end point
    nd
    nd
    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 No
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months36
    E.8.9.1In the Member State concerned days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 0
    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: 18
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state18
    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)
    ND
    ND
    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 Decision2011-10-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-10-12
    P. End of Trial
    P.End of Trial StatusOngoing
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