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    Summary
    EudraCT Number:2022-002611-29
    Sponsor's Protocol Code Number:82050
    National Competent Authority:Netherlands - Competent Authority
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2023-03-26
    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 ConcernedNetherlands - Competent Authority
    A.2EudraCT number2022-002611-29
    A.3Full title of the trial
    DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD): a randomized double-blind placebo-controlled trial
    Denosumab voor de behandeling van Fibreuze Dysplasie/McCune-Albright syndroom in volwassenen (de DeFiD studie): een gerandomiseerde dubbel geblindeerde placebo studie
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    DEnosumab for the treatment of FIbrous Dysplasia/McCune-Albright Syndrome in adults (DeFiD)
    Denosumab voor de behandeling van Fibreuze Dysplasie/McCune-Albright syndroom in volwassenen (de DeFiD studie)
    A.3.2Name or abbreviated title of the trial where available
    DeFiD
    A.4.1Sponsor's protocol code number82050
    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 SponsorLeiden University Medical Center
    B.1.3.4CountryNetherlands
    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 supportLeiden University Medical Center
    B.4.2CountryNetherlands
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationLeiden University Medical Center
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street AddressAlbinusdreef 2
    B.5.3.2Town/ cityLeiden
    B.5.3.3Post code2333ZA
    B.5.3.4CountryNetherlands
    B.5.6E-mailbot@lumc.nl
    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 Yes
    D.2.1.1.1Trade name Denosumab (Xgeva)
    D.2.1.1.2Name of the Marketing Authorisation holderAmgen Europe B.V.
    D.2.1.2Country which granted the Marketing AuthorisationNetherlands
    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.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    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 RoleComparator
    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 Natrium Chloride 0.9%
    D.2.1.1.2Name of the Marketing Authorisation holderB.BRAUN MELSUNGEN AG
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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.3.4Pharmaceutical form Injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboInjection
    D.8.4Route of administration of the placeboSubcutaneous use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboInjection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Fibrous Dysplasia/ McCune Albright Syndrome
    Fibreuze Dysplasie/McCune Albright Syndroom
    E.1.1.1Medical condition in easily understood language
    Fibrous Dysplasia/McCune-Albright syndrome (FD/MAS) is a rare disease, consisting of the replacement of normal bone tissue with fibrous tissue.
    Fibreuze dysplasie is een zeldzame genetische aandoening ve, waarbij
    gezond bot wordt vervangen door fibreus weefsel.
    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 Denosumab on pain, assessed by the difference in maximum pain scores after 6 months (2 injections).
    Het effect van Dmab op pijn, bepaald met het verschil in maximale pijnscore na 6 maanden (2 injecties)
    E.2.2Secondary objectives of the trial
    • Evaluation of Dmab effect on Quality of life, physical activity
    • Evaluation of possible neuropathic component of the reported pain
    • Evaluation of analgesics use
    • Evaluation of changes in mobility
    • Evaluation of Dmab effect on FD lesion size and activity
    • Evaluation of Dmab effect on bone density
    • Het effect van Denosumab op de kwaliteit van leven en op de beoordeling van fysieke activiteit te evalueren
    • De prevalentie van een mogelijke neuropathische component van de gerapporteerde pijn te evalueren
    • Onderzoeken van analgetica gebruik
    • Het effect van Denosumab op de beoordeling van fysieke activiteit te onderzoeken
    • Het effect van Denosumab op de activiteit en de grootte van de FD laesies
    • Het effect van Denosumab op botdichtheid
    •
    •
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Being symptomatic with an established diagnosis of FD/MAS and closed growth plates (>18 years)
    - Pain in the region of an FD localization, not responding to adequate pain treatment and
    without mechanical component e.g. impending fracture
    - Pain score from FD lesion for maximum or average pain on VAS ≥ 4
    - Increased lesional activity defined as increased bone turnover markers (ALP, P1NP or CTX) or increased activity on Na[18F]-PET/CT or bone scintigraphy in at least one lesion
    - Normal levels of calcium, parathyroid hormone and vitamin D (supplementation is allowed)
    - Treated hypophosphatemia (defined as >0.7 at two separate measures)
    - good dental health (last check within the last 12 months)
    - Patienten met FD diagnose met symptomen en gesloten groeischijven (>18 jaren)
    - Pijn op de locatie van de FD laesies, niet verbeterd op adequate pijnmedicatie en zonder mechanische component bijv. (dreigende) fractuur
    - Maximale of gemiddelde pijn score op VAS ≥ 4
    - Verhoogde lesionale botactiviteit, gedefinieerd als verhoogde bot turnover markers (ALP, P1NP or CTX) of verhoogde activiteit op Na[18F]-PET/CT of bot scintigrafie in minimaal een laesie
    - Normale waardes van calcium, PTH en vitamine D (suppletie is toegestaan)
    - Behandelde hypofosfatemie (gedefinieerd als <0.7 tijdens twee metingen)
    - Goede mondhygiene (laaste tandarts bezoek maximaal 12 maanden geleden)
    E.4Principal exclusion criteria
    - Active pregnancy wish, pregnancy or nursing
    - Pain not related to FD
    - Uncontrolled endocrine disease
    - Untreated vitamin D deficiency, hypocalcemia or hypophosphatemia
    - Previous use of bisphosphonates or Dmab < 6 months before inclusion (‘6 months wash out’)
    - Previously reported side effects on Dmab
    - Inability to fulfil study requirements
    - Poor untreated dental health without intention to get treatment
    - Treatment with other bone influencing drugs, such as high doses corticosteroids
    - Actieve zwangerschap wens, zwangershap of borstvoeding
    - Pijn niet gerelateerd aan FD
    - Ongecontroleerd endocriene aandoening
    - Onbehandeld vitamine D deficientie, hypocalcemie of hypofosfatemie
    - Behandeling met bisfosfonaten of Denosumab < 6 maanden voor inclusie
    - Voorheen bijwerkingen bij Denosumab behandeling
    - Onvermogen om aan studievereisten te voldoen
    - Slechte, onbehandelde dentale hygiene zonder wens voor behandeling
    - Behandeling met andere medicijnen die het skelet kunnen beinvloeden zoals hogere doseringen van corticosteroiden
    E.5 End points
    E.5.1Primary end point(s)
    The effect of Dmab on pain, assessed by the difference in maximum pain score after 6 months (2 injections) by Brief Pain Inventory
    Het effect van Dmab op pijn, bepaald met het verschil in maximale pijnscore na 6 maanden (2 injecties) door Brief Pain Inventory (BPI)
    E.5.1.1Timepoint(s) of evaluation of this end point
    after 6 months
    na 6 maanden
    E.5.2Secondary end point(s)
    • to evaluate the effect of Dmab on average pain scores after 3, 6 months of treatment and in case of open label treatment after 9 and 12 months
    • to evaluate the number of patients with 50% reduction of maximal pain (BPI) after 3, 6 months of treatment and in case of open label treatment after 9 and 12 months
    • to evaluate the effect of Dmab on quality of life, assessed with questionaries (SF-36) at baseline, 3 months and after 6 months and in case of open label treatment after 9 and 12 months
    • to evaluate the effect of Dmab on average weekly pain assessed through a pain diary with VAS score
    • to investigate the effect of Dmab on Physical activity assessment (Health Assessment Questionnaire – Disability Index and screenshot of pedometer of activity during the last week on smartphone) measured at baseline, 3 months and 6 months, and in case of open label treatment after 9 and 12 months
    • to evaluate the prevalence of possible neuropathic component of the reported pain through Pain Detect questionnaire
    • to investigate the number of analgesics use and dosage used at baseline, 3 months and 6 months and in case of open label treatment after 9 and 12 months
    • to assess the effect of Dmab on disease activity through laboratory measurements of bone markers at baseline, 3 months and 6 months, and in case of open label treatment after 9 and 12 months
    • to assess the effect of Dmab on lesions activity and lesions size through bone scans at baseline and after 6 months, and in the case of open label treatment after 12 months
    • to assess disease quantification by nuclear imaging before and after treatment (Skeletal Burden Score (SBS)
    • to assess bone density and the presence of vertebral fractures (Dual-energy X-ray absorptiometry (DXA) + Vertebral Fractures Assessment (VFA) at baseline and after 12 months
    • to assess potential side effects in the form of Atypical femoral fractures by performing and extended DXA after 12 months
    • Het effect van Dmab op gemiddelde pijnscores na 3 en 6 maanden van behandeling te evalueren (middels BPI) en in geval van open-labelbehandeling na 9 en 12 maanden
    • Het aantal patiënten te evalueren met 50% vermindering van maximale pijn (BPI) na 3 maanden en na 6 maanden en in geval van open-labelbehandeling na 9 en 12 maanden
    • Het effect van Dmab op de kwaliteit van leven te evalueren, beoordeeld met vragenlijsten (SF-36) bij baseline, 3 maanden en na 6 maanden en in geval van open-labelbehandeling na 9 en 12 maanden
    • Het effect van Dmab op de gemiddelde wekelijkse pijn te evalueren, beoordeeld door middel van een pijndagboek met VAS-score
    • Het effect van Dmab op de beoordeling van fysieke activiteit te onderzoeken (Health Assessment Questionnaire – Disability Index en screenshot van stappenteller van activiteit gedurende de laatste week op smartphone), gemeten bij baseline, 3 maanden en 6 maanden, en in geval van open-labelbehandeling na 9 en 12 maanden
    • De prevalentie van een mogelijke neuropathische component van de gerapporteerde pijn te evalueren door middel van de Pain Detect-vragenlijst bij baseline, na 3 en 6 maanden, en in geval van open-labelbehandeling na 9 en 12 maanden
    • Onderzoeken van hoeveelheid en dosering analgetica gebruik bij baseline, 3 maanden en 6 maanden en in geval van open-labelbehandeling na 9 en 12 maanden
    • Het effect van Dmab op ziekteactiviteit te beoordelen door middel van laboratorium bepalingen van botmarkers bij baseline, 3 maanden en 6 maanden, en in geval van open-labelbehandeling na 9 en 12 maanden
    • Het effect van Dmab op de activiteit en de grootte van de laesies te beoordelen door middel van botscans bij baseline en na 6 maanden, en in het geval van open-label behandeling na 12 maanden
    • Het beoordelen van ziektekwantificering door middel van nucleaire beeldvorming voor en na de behandeling (Skeletal Burden Score (SBS)
    • De botdichtheid en de aanwezigheid van wervelfracturen te beoordelen (Dual-energy X-ray absorptiometry (DXA) + Vertebral Fractures Assessment (VFA) bij aanvang en na 12 maanden
    • Mogelijke bijwerkingen in de vorm van atypische femurfracturen te beoordelen door het uitvoeren van een verlengde DXA na 12 maanden
    E.5.2.1Timepoint(s) of evaluation of this end point
    at baseline and after 6 months, and in the case of open label treatment after 12 months
    bij baseline en na 6 maanden, en in het geval van open-label behandeling na 12 maanden
    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 No
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 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 concerned2
    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 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 years2
    E.8.9.1In the Member State concerned months4
    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.1.1.1In 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: 82
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state82
    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 patients will receive one-time intravenous zoledronate after exit from the study, a standard treatment after stopping Denosumab
    Alle patiënten zullen eenmalig intraveneus zoledronaat krijgen na het beëindigen van de studie, een standaardbehandeling na het stoppen met Denosumab
    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 Decision2023-06-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2023-03-24
    P. End of Trial
    P.End of Trial StatusOngoing
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