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    Summary
    EudraCT Number:2011-001787-22
    Sponsor's Protocol Code Number:AC-007-IT
    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:2012-01-12
    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 number2011-001787-22
    A.3Full title of the trial
    An open-label, phase II study of Pomalidomide and Dexamethasone (PDex) for previously treated patients with AL amyloidosis
    Studio in aperto di fase II con pomalidomide e desametasone (PDex) per pazienti con amiloidosi AL precedentemente trattati.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Treatment with pomalidomide and dexamethasone for previously treated patients with AL amyloidosis.
    Terapia con pomalidomide e desametasone per il trattamento di pazienti affetti da amiloidosi AL gia' in precedenza trattati.
    A.3.2Name or abbreviated title of the trial where available
    PDex
    PDex
    A.4.1Sponsor's protocol code numberAC-007-IT
    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 SponsorOSPEDALE POLICLINICO S. MATTEO
    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 supportCelgene International Sarl
    B.4.2CountrySwitzerland
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationFondazione IRCCS Policlinico San Matteo
    B.5.2Functional name of contact pointCentro Amiloidosi
    B.5.3 Address:
    B.5.3.1Street AddressPiazzale Golgi 19
    B.5.3.2Town/ cityPavia
    B.5.3.3Post code27100
    B.5.3.4CountryItaly
    B.5.4Telephone number0382 502994
    B.5.5Fax number0382 502990
    B.5.6E-mailcentro.amiloidosi@smatteo.pv.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 namepomalidomide
    D.3.2Product code CC-4047
    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 INNPomalidomide
    D.3.9.1CAS number 19171-19-8
    D.3.9.2Current sponsor codeCC-4047
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number2
    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 Yes
    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.3.4Pharmaceutical form Oral drops, solution
    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 INNDEXAMETHASONE SODIUM SULFATE
    D.3.9.1CAS number 466-11-5
    D.3.9.4EV Substance CodeSUB01617MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number40
    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
    Previously treated AL amyloidosis
    Amiloidosi sistemica AL già trattata
    E.1.1.1Medical condition in easily understood language
    Previously treated AL amyloidosis
    Amiloidosi sistemica AL già trattata
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10002022
    E.1.2Term Amyloidosis
    E.1.2System Organ Class 10021428 - Immune system 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 evaluate the efficacy of pomalidomide + dexamethasone (PDex) in patients who did not achieve a complete response after initial treatment with both an alkylating agent and bortezomib.
    Determinare l’efficacia della terapia con Pomalidomide + Desametasone (PDex) in pazienti che non hanno ottenuto la remissione completa dopo trattamento con alchilanti e bortezomib.
    E.2.2Secondary objectives of the trial
    To assess the safety of PDex combination. To assess the survival of AL Amyloidosis patients treated with PDex.
    Determinare la sicurezza del trattamento con PDex. Determinare la sopravvivenza dei pazienti con amiloidosi AL trattati con PDex.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • At least 18 years. • Diagnosis of systemic AL amyloidosis. Symptomatic organ involvement. • Patients achieving less than complete response after initial treatment with an alkylating agent and bortezomib. Patients with AL amyloidosis who received 1 previous treatment, but who could not be treated with alkylators and/or bortezomib due to contraindications, will be eligible. • Measurable disease: difference between amyloidogenic (involved) and uninvolved free light chains (dFLC) >50 mg/L. • Hb ≥10 g/dL • ANC ≥1500/mcL. • Platelet count ≥100000/mcL. • eGFR ≥30 mL/min per 1.73 m2. • Performance status (ECOG) <3. • Total bilirubin <2.5 mg/dL. • Alkaline phosphatase <5 × url. • ALT <3 × url. Subjects must be informed about pregnancy program prevention
    • Età ≥ 18 anni • Diagnosi di amiloidosi sistemica AL • Interessamento d’organo (cuore, rene, fegato, sistema nervoso periferico, tessuti molli) • Pazienti che non hanno raggiunto la risposta completa dopo il trattamento iniziale con un agente alchilante e bortezomib. I pazienti con amiloidosi AL che hanno ricevuto un precedente trattamento, ma che non possono essere trattati con alchilanti e/o bortezomib a causa di controindicazioni, saranno eligibili. • Malattia misurabile: differenza tra la concentrazione delle catene leggere amiloidogeniche (interessate) e quelle non interessate (dFLC) &gt; 50 mg/L. • Hb ≥ 10 g/dL • ANC ≥ 1500/mcL • Piastrine ≥ 100000/mcL • eGFR ≥ 30 mL/min per 1.73m2 • Performance status (ECOG) &lt; 3 • Bilirubina totale &lt;2.5 mg/dL • Fosfatasi alcalina &lt; 5 x url • ALT &lt; 3 x url • Il soggetto deve essere consapevole delle precauzioni da prendere in gravidanza e i potenziali rischi di un’esposizione del feto.
    E.4Principal exclusion criteria
    • Amyloid-specific syndrome • New York Heart association (NYHA) class IV. • Known positivity for HIV or active hepatitis infection. • Pregnant or nursing women (men must agree to use an acceptable method for contraception for the duration of the study). • Uncontrolled infections. • Other active malignancies. • Patient has a prior history of thrombosis or venous thromboembolism or pulmonary embolism. • Known hypersensitivity to thalidomide or lenalidomide including development of erythema. • Previous or ongoing psychiatric illness (with the exclusion of reactive depression).
    • Sindrome amiloide-specifica • Classe New York Heart Association (NYHA) IV • Positività all’HIV o infezione attiva da epatite • Donne gravide o in allattamento (gli uomini devono acconsentire all’uso di un metodo accettabile di contraccezione per la durata dello studio) • Infezioni incontrollate • Altra patologia maligna attiva • Pazienti con una precedente storia di trombosi o trombo-embolia venosa o embolia polmonare • Ipersensibilità alla thalidomide o alla lenalidomide incluso lo sviluppo di eritema • Malattia mentale (con l’esclusione di depressione reattiva)
    E.5 End points
    E.5.1Primary end point(s)
    To assess the rate of hematologic response at 3 months
    Determinare la frequenza di risposta ematologica a 3 mesi
    E.5.1.1Timepoint(s) of evaluation of this end point
    3 months
    3 mesi
    E.5.2Secondary end point(s)
    Rate of adverse events. Rate of complete response at 3, 6, 9, and 12 months. Hematologic response rate at 6, 9, and 12 months. Organ response rate at 3, 6, 9, and 12 months. Progression-free survival. Overall survival.
    Determinare la frequenza di eventi avversi. Determinare la frequenza di risposta completa ai mesi 3, 6, 9, e 12. Determinare la frequenza di risposta ematologica ai mesi 6, 9, e 12. Determinare la frequenza di risposta d’organo ai mesi 3, 6, 9, e 12. Determinare la sopravvivenza libera da progressione. Determinare la sopravvivenza.
    E.5.2.1Timepoint(s) of evaluation of this end point
    See secondary endpoints
    Vedi end-point secondari
    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 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
    A subject will be considered as having completed the study if any of the following endpoints is met: • hematologic or organ progression • unacceptable toxicity, and the end of treatment visit has been performed.
    Ciascun paziente risulta aver completato lo studio quando ha effettuato la visita di fine trattamento a seduito di: - progressione (ematologica o d'organo) - tossicità inaccettabile
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years5
    E.8.9.1In the Member State concerned months0
    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: 19
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 9
    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 state28
    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)
    NA
    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 Decision2012-05-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2011-12-15
    P. End of Trial
    P.End of Trial StatusOngoing
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