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    Summary
    EudraCT Number:2016-001955-29
    Sponsor's Protocol Code Number:024PKAN15004
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2017-03-30
    Trial results View 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 ConcernedSpain - AEMPS
    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
    Eficacia, seguridad y tolerabilidad de Fosmetpantotenato (RE-024), un tratamiento sustitutivo con Fosfopantotenato, en pacientes con neurodegeneración asociada a la cinasa del Pantotenato (PKAN): Estudio aleatorizado, en doble ciego y controlado con placebo, con una extensión abierta.
    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)
    Eficacia, seguridad y tolerabilidad de Fosmetpantotenato en pacientes con neurodegeneración asociada a la cinasa del Pantotenato
    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 number+34913913800
    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 nameFosmetpantotenate
    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 INNFosmetpantotenate
    D.3.9.1CAS number 1858268-66-2
    D.3.9.2Current sponsor codeRE-024
    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.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 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.
    Neurodegeneración asociada a la cinasa del pantotenato (PKAN), un desorden genetico recesivo autosomal, la forma más común de Neurogeneración con acumulación de hierro en el cerebro (NBIA). Es una enfermedad neurogenerativa, progresiva y a menudo mortal.
    E.1.1.1Medical condition in easily understood language
    Neurodegenerative disease.
    Enfermedad neurodegenerative.
    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 19.1
    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.
    El objetivo de eficacia de este estudio es evaluar la eficacia del fosmetpantotenato a lo largo de 24 semanas en pacientes con PKAN.
    El objetivo de seguridad de este studio es evaluar la seguridad y tolerabilidad del fosmetpantotenato en pacientes 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.
    Determinar la farmacocinética tras dosis repetidas de fosmetpantotenato en pacientes con PKAN.
    Explorar posibles biomarcadores de la enfermedad y su posible respuesta al tratamiento en pacientes con PKAN.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - The patient has a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 (PANK2) gene.
    - The patient has a score of >= 6 on the Pantothenate Kinase-associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale.
    - Estar diagnosticado de PKAN, indicado por mutaciones confirmadas del gen de la cinasa del pantotenato 2 (PANK2).
    - Tener una puntuación de >= 6 en la escala de Actividades cotidianas en la Neurodegeneración asociada a la cinasa del pantotenato (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 for the double-blind period of the study.
    - The patient has taken deferiprone within 30 days prior to screening.
    - El paciente ha precisado soporte ventilatorio invasivo regular o intermitente para mantener las constantes vitales en las 24 semanas previas a la aleatorización.
    - El paciente ha tenido implantado un dispositivo de estimulación cerebral profunda (DBS) en los 6 meses anteriores a la selección.
    - El paciente no puede o no está dispuesto a permanecer, durante el periodo en doble ciego del estudio, con sus dosis de medicamentos y tratamientos de mantenimiento de PKAN concomitantes permitidos anteriores al estudio.
    - El paciente ha tomado deferiprona en los 30 días anteriores a la selección.
    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
    Criterio principal de valoración
    - Variación de la puntuación de PKAN-ADL

    Criterio de valoración de la seguridad
    - Seguridad y tolerabilidad
    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.
    En cada paciente, para el análisis se utilizará la variación de la puntuación del PKAN-ADL en las semanas 3, 6, 12, 18 y 24 del periodo en doble ciego respecto a la puntuación basal.
    E.5.2Secondary end point(s)
    Secondary Efficacy Endpoint
    - Change in the score from Part III of the UPDRS
    El criterio secundario de valoración
    - Variación de la puntuación de la Parte III de la UPDRS
    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.
    Medidas registradas continuadas en cada visita del estudio desde la basal hasta el fin del periodo de tratamiento en doble ciego de 24 semanas.
    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
    Tolerability
    Tolerabilidad
    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 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 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 Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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 No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    Czech Republic
    France
    Germany
    Italy
    Norway
    Spain
    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
    LVLS
    Ultima visita del ultimo sujeto
    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 months8
    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 months8
    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.1.1Number of subjects for this age range: 41
    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.1.1.5.1Number of subjects for this age range: 20
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number 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 state16
    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.
    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 Decision2017-10-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-29
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2019-08-28
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