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    The EU Clinical Trials Register currently displays   43977   clinical trials with a EudraCT protocol, of which   7312   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2016-001955-29
    Sponsor's Protocol Code Number:024PKAN15004
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2020-11-05
    Trial results View results
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    A. Protocol Information
    A.1Member State ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-001955-29
    A.3Full title of the trial
    Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension
    Efficacia, sicurezza e tollerabilit¿ di fosmetpantotenato (RE-024), una terapia sostitutiva di fosfopantotenato, nei pazienti con neurodegenerazione associata alla pantotenato-chinasi (PKAN): studio randomizzato, in doppio cieco, controllato verso placebo con estensione in aperto.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)
    Efficacia, sicurezza e tollerabilit¿ di fosmetpantotenato in pazienti con neurodegenerazione associata alla pantotenato-chinasi (PKAN)
    A.3.2Name or abbreviated title of the trial where available
    A.4.1Sponsor's protocol code number024PKAN15004
    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 SponsorRETROPHIN, 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 supportRetrophin, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRetrophin Inc.
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street Address3721 Valley Centre Drive, Suite 200
    B.5.3.2Town/ citySan Diego
    B.5.3.3Post codeCA 92130
    B.5.3.4CountryUnited States
    B.5.4Telephone number0018776595518
    B.5.5Fax number000000
    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/3/16/1612
    D.3 Description of the IMP
    D.3.1Product nameFosmetpantotenato
    D.3.2Product code [RE-024]
    D.3.4Pharmaceutical form Powder for oral suspension
    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 INNFosmetpantotenato
    D.3.9.1CAS number 1858268-66-2
    D.3.9.2Current sponsor codeRE-024
    D.3.9.4EV Substance CodeSUB186881
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number75
    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. cell therapy medicinal product Information not present in EudraCT
    D. therapy medical product Information not present in EudraCT
    D. Engineered Product Information not present in EudraCT
    D. ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D. 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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboPowder for oral suspension
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
    Neurodegenerazione associata alla pantotenato-chinasi (PKAN), un disordine genetico autosomico recessivo, la forma pi¿ comune di neurodegenerazione con accumulo di ferro nel cervello (NBIA). E' una malattia neurodegenerativa progressiva, spesso fatale.
    E.1.1.1Medical condition in easily understood language
    Neurodegenerative disease.
    Malattia neurodegenerativa.
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level PT
    E.1.2Classification code 10053643
    E.1.2Term Neurodegenerative disorder
    E.1.2System Organ Class 10029205 - Nervous 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
    The efficacy objective of this study is to evaluate the efficacy of fosmetpantotenate over 24 weeks in patients with PKAN.
    The safety objective of the study is to assess the safety and tolerability of fosmetpantotenate in patients with PKAN.
    L'obiettivo di efficacia dello studio ¿ valutare l¿efficacia di fosmetpantotenato nell¿arco di 24 settimane nei pazienti con PKAN.
    L'obiettivo di sicurezza dello studio ¿ valutare la sicurezza e la tollerabilit¿ di fosmetpantotenato nei pazienti con PKAN.
    E.2.2Secondary objectives of the trial
    To determine the PK following multiple doses of fosmetpantotenate in patients with PKAN.
    To explore potential biomarkers of disease, along with their potential response to treatment in patients with PKAN.
    Stabilire la farmacocinetica (PK) in seguito a dosi multiple di fosmetpantotenato nei pazienti con PKAN.
    Esplorare potenziali biomarcatori della malattia, insieme alla potenziale risposta al trattamento nei
    pazienti con PKAN.
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives

    Other types of substudies
    Specify title, date and version of each substudy with relative objectives: Additional Exploratory Assessment for Research

    A blood sample will be collected at 2-3 different investigational sites from up to a total of 8 eligible patients (half adult, half pediatric) with different confirmed mutations in PANK2 to generate peripheral blood mononuclear cells (PBMCs).

    To be eligible to participate, patients must have negative serologic tests for all tests outlined in Section 15.2.2 in the Clinical Trial Protocol.
    Patients will be required to sign a separate consent to participate in this exploratory sub-study. If a patient discontinues participation in the substudy, their participation in the main study will not be affected.

    Altre tipologie di sottostudi
    specificare il titolo, la data e la versione di ogni sottostudio con i relativi obiettivi: Valutazione esplorativa aggiuntiva per ricerca

    Sarà raccolto un campione di sangue in 2-3 centri sperimentali differenti, per un totale di 8 pazienti eleggibili (metà adulti, metà pediatrici) con mutazioni confermate diverse in PANK2, per generare cellule mononucleate da sangue periferico (PBMC).

    Per essere idonei a partecipare, i pazienti devono avere le analisi sierologiche negative per tutte le analisi indicate in Sezione 15.2.2 del Protocollo Clinico.
    I pazienti dovranno firmare un consenso separato per la partecipazione a questo sotto-studio esplorativo. Se un paziente interrompe la partecipazione al sotto-studio, non ci saranno ripercussioni sulla partecipazione allo studio principale.
    E.3Principal inclusion criteria
    - The patient has a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 (PANK2) gene (if available, the specific mutation will be recorded).
    - The patient has a score of = 6 on the Pantothenate Kinase-associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale.
    - Il paziente presenta una diagnosi di PKAN, come indicato dalle mutazioni confermate nel gene della
    pantotenato-chinasi 2 (PANK2) (se disponibile, la specifica mutazione sarà registrata).
    - Il paziente presenta un punteggio =6 nella scala delle attività quotidiane svolte con neurodegenerazione associata alla pantotenato-chinasi (Pantothenate Kinase-associated Neurodegeneration Activities of Daily Living, PKAN-ADL).
    E.4Principal exclusion criteria
    - The patient has required regular or intermittent invasive ventilatory support to maintain vital signs within 24 weeks prior to randomization.
    - The patient has had a deep brain stimulation (DBS) device implanted within 6 months prior to screening.
    - The patient is unable or unwilling to remain on their pre-study dose(s) of allowed concomitant PKAN maintenance medications and therapies (including DBS settings) for the double-blind period of the study.
    - The patient has taken deferiprone within 30 days prior to screening.
    - Il paziente ha richiesto supporto ventilatorio invasivo regolare o intermittente per mantenere i segni vitali nelle ultime 24 settimane prima della randomizzazione.
    - Il paziente si è sottoposto all’impianto di un dispositivo per la stimolazione cerebrale profonda (deep brain stimulation, DBS) nei 6 mesi precedenti lo screening.
    - Il paziente non è in grado o non è disposto a continuare ad utilizzare la/e dose/i dei farmaci e le terapie di mantenimento concomitanti consentiti per la PKAN (incluso l'impianto del DBS) che utilizzava prima dello studio per la parte in doppio cieco dello studio.
    - Il paziente ha assunto deferiprone nei 30 giorni precedenti lo screening.
    E.5 End points
    E.5.1Primary end point(s)
    Primary Efficacy Endpoint
    - Change in the score from the PKAN-ADL
    Safety Endpoint
    - Safety and tolerability
    Endpoint di efficacia primario
    - Cambiamento nel punteggio PKAN-ADL
    Endpoint di sicurezza
    - Sicurezza e tollerabilità
    E.5.1.1Timepoint(s) of evaluation of this end point
    For each patient, the change from Baseline in PKAN-ADL scores at Weeks 3, 6, 12, 18, and 24 of the double-blind period will be used for analysis.
    Per ogni paziente, il cambiamento del punteggio PKAN-ADL dal basale alle settimane 3, 6, 12, 18 e 24 del periodo di doppio cieco che sarà utilizzato per l'analisi.
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoint
    - Change in the score from Part III of the UPDRS
    Endpoint secondario di efficacia
    - Cambiamento nel punteggio della Parte III UPDRS (Unified Parkinson¿s Disease Rating Scale)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Continuous measures recorded at each study visit from Baseline to the end of the 24-week double-blind period.
    Continue misure registrate ad ogni visita di studio dal basale alla fine del periodo di 24 settimane in doppio cieco.
    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 No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    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. 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 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 Yes
    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 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 EEA11
    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
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    E.8.9.1In the Member State concerned months5
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years6
    E.8.9.2In all countries concerned by the trial months5
    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. of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F. of subjects for this age range: 21
    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 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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 82
    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)
    None. The investigator is responsible for ensuring consideration has been given to the post-study care of the patient's medical needs.
    Nessuno. Lo sperimentatore ¿ responsabile di garantire che sono state valutate le necessit¿ mediche del paziente nel periodo post-studio.
    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 Decision2017-11-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-03
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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