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    Summary
    EudraCT Number:2014-004113-85
    Sponsor's Protocol Code Number:PA101-SM-02
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2014-12-22
    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 number2014-004113-85
    A.3Full title of the trial
    Randomized, Double-blind, Placebo-controlled, Crossover Design, Efficacy and Safety
    Study with PA101 in Patients with Indolent Systemic Mastocytosis
    Estudio de eficacia y seguridad con PA 101, aleatorizado, doble ciego,
    controlado con placebo de diseño cruzado en pacientes con mastocitosis
    sistémica indolent
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A controlled study in patients with Indolent Systemic Mastocytosis to determine the safety and efficacy of PA101 when compared with an existing drug and a placebo
    Estudio controlado en pacientes con mastocitosis sistemica indolente para
    determinar la eficacia y seguridad de PA comparado con otros fármacos
    existentes o placebo.
    A.4.1Sponsor's protocol code numberPA101-SM-02
    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 SponsorPatara Pharma, LLC
    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 supportPatara Pharma, LLC
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPremier Research
    B.5.2Functional name of contact pointProject Manager
    B.5.3 Address:
    B.5.3.1Street AddressEuropaplatz 5
    B.5.3.2Town/ cityDarmstadt
    B.5.3.3Post code64293
    B.5.3.4CountryGermany
    B.5.4Telephone number+34917080386
    B.5.5Fax number+496151 8280 110
    B.5.6E-mailaxel.fey@premier-research.com
    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 nameDisodium Cromoglycate
    D.3.2Product code PA101
    D.3.4Pharmaceutical form Inhalation solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPInhalation use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSODIUM CROMOGLICATE
    D.3.9.1CAS number 15826-37-6
    D.3.9.2Current sponsor codePA101
    D.3.9.4EV Substance CodeSUB12286MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typenot less then
    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 Yes
    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 Nalcrom
    D.2.1.1.2Name of the Marketing Authorisation holderSanofi
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameNalcrom
    D.3.4Pharmaceutical form Capsule
    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 INNSODIUM CROMOGLICATE
    D.3.9.1CAS number 15826-37-6
    D.3.9.4EV Substance CodeSUB12286MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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 placeboInhalation solution
    D.8.4Route of administration of the placeboInhalation use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Indolent Systemic Mastocytosis
    Mastocitosis sistémica Indolente
    E.1.1.1Medical condition in easily understood language
    Mastocytosis is a rare condition caused by an excess number of mast cells gathering in the body's tissues. In systemic mastocytosis, mast cells gather in tissues such as the skin, organs and bones.
    Mastocitosis es una enfermedad rara causada por exceso de mastocitos en tejidos del cuerpo. En la mastocitosis sistémica, los mastocitos acumulan en tejidos como la piel, los órganos y los huesos.
    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 17.1
    E.1.2Level PT
    E.1.2Classification code 10042949
    E.1.2Term Systemic mastocytosis
    E.1.2System Organ Class 10005329 - Blood and lymphatic 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 determine the efficacy profile of PA101 delivered via a high efficiency nebulizer (eFlow®, PARI) in comparison with placebo following 6 weeks of treatment in patients with indolent systemic mastocytosis (ISM) who are symptomatic despite using standard treatments
    Determinar el perfil de eficacia de PA101 administrado mediante un
    nebulizador de alta eficiencia (eFlow®, PARI) en comparación con
    placebo después de 6 semanas de tratamiento en pacientes con
    mastocitosis sistémica indolente (MSI) que presentan síntomas a pesar
    del uso de los tratamientos de referencia
    E.2.2Secondary objectives of the trial
    To compare the efficacy and safety profile of PA101 to marketed oral cromolyn sodium (open-label control)
    To assess the safety, tolerability, and pharmacokinetic (PK) profile of PA101
    Comparar el perfil de eficacia y seguridad de PA101 con el cromoglicato
    disódico oral comercializado (control en abierto).
    Evaluar el perfil de seguridad, tolerabilidad y farmacocinética (FC) de
    PA101
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Male or female patients 18-75 years of age, inclusive
    2. Diagnosed with indolent systemic mastocytosis (ISM) according to the WHO criteria and the consensus proposal (2001)
    3. Experiencing at least one qualifying symptom in at least two organ systems during the 3 months preceding the Screening Visit, despite the use of H1 and H2 antihistamines and other antimediator therapy
    4. Experiencing symptoms with a severity score of at least 4 for at least 7 out of 14 days during the Run-in Period with at least one qualifying symptom each from at least two organ systems, despite the use of H1 and H2 antihistamines and other anti-mediator therapy
    5. Willing and able to use an eDiary device daily for the duration of the study
    6. Completed at least 5 eDiary reports during each of two consecutive weeks of the Run-in Period
    7. Patients must digitally accept the licensing agreement in the eDiary software
    8. Willingness and ability to provide written informed consent
    1.Pacientes de ambos sexos de 18 a 75 años, inclusive.
    2.Pacientes con diagnóstico de mastocitosis sistémica indolente (MSI)
    según los criterios de la OMS y la propuesta de consenso (2001).
    3.Presentar al menos un síntoma que reúna los requisitos en un mínimo
    de dos categorías de órgano, aparato o sistema durante los tres meses
    anteriores a la visita de selección, a pesar del uso de antihistamínicos H1 y H2 y de otros tratamientos antimediadores.
    4.Presentar síntomas con una puntuación de intensidad de como mínimo 4, durante al menos 7 de 14 días durante el período de preinclusión y con un síntoma como mínimo que reúna los requisitos en al menos dos categorías de órgano, aparato o sistema, a pesar del uso de antihistamínicos H1 y H2 y de otros tratamientos antimediadores.
    5.Con disposición y capacidad para utilizar diariamente un diario
    electrónico durante todo el estudio.
    6.Completar al menos cinco informes del Diario-e durante las dos
    semanas consecutivas del período de preinclusión, antes de la
    aleatorización (informes semanales máximos posibles = 7 a la semana).
    7.Los pacientes deben aceptar digitalmente el acuerdo de licencia en el
    software del Diario-e.
    8.Con disposición y capacidad para otorgar el consentimiento informado por escrito
    E.4Principal exclusion criteria
    1. Advanced systemic mastocytosis (i.e., aggressive systemic mastocytosis [ASM], mast cell leukemia [MCL], or systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease [SM-AHNMD] )
    2. Current or recent history of clinically significant cardiovascular, hematological, renal, neurologic, hepatic, endocrine, psychiatric, malignant, or other illnesses that could put the patient at risk or compromise the quality of the study data as determined by the Investigator
    3. Use of oral cromolyn sodium within 6 weeks of the Screening Visit
    4. History of systemic corticosteroid, immunosuppressive, or anti-IgE monoclonal antibody therapy (e.g., omalizumab) within 6 months of the Screening Visit
    5. History of anaphylaxis requiring systemic treatment (i.e., corticosteroid or epinephrine) within 12 months of the Screening Visit
    6. An upper or lower respiratory tract infection within 4 weeks of the Screening Visit
    7. History of malignancy within the last 5 years, except basal cell carcinoma or cervix carcinoma in situ
    8. Major surgery within 6 months of the Screening Visit
    9. History of excessive use or abuse of alcohol (i.e., more than 3 units per day, or more than 21 units per week) within 12 months of the Screening Visit
    10. History of abusing legal drugs or use of illegal drugs or substances within 12 months of the Screening Visit
    11. Females who are pregnant or breastfeeding, or if of child-bearing potential unwilling to practice acceptable means of birth control or abstinence during the study
    12. Participation in any other investigational drug study within 4 weeks of the Screening Visit
    13. History of hypersensitivity or intolerance to aerosol medications or cromolyn sodium
    1.Mastocitosis sistémica avanzada (es decir, mastocitosis sistémica
    agresiva [MSA], leucemia mastocítica [LM] o mastocitosis sistémica con
    una enfermedad hematológica clonal no asociada a la línea celular de los mastocitos [SM-AHNMD, por sus siglas en inglés]).
    2.Presencia o antecedentes recientes de enfermedades clínicamente
    significativas cardiovasculares, hematológicas, renales, neurológicas,
    hepáticas, endocrinas, psiquiátricas, malignas o de otro tipo que podrían poner al paciente en riesgo o poner en peligro la calidad de los datos del estudio, según el criterio del investigador.
    3.Uso de cromoglicato disódico oral en las 6 semanas anteriores a la
    visita de selección.
    4.Antecedentes de tratamiento sistémico con corticosteroides,
    inmunosupresores o anticuerpos monoclonales anti-IgE (p. ej.,
    omalizumab) en los 6 meses anteriores a la visita de selección.
    5.Antecedentes de anafilaxia que requirió tratamiento sistémico (es
    decir, corticosteroides o epinefrina) en los 12 meses anteriores a la
    visita de selección.
    6.Infección de vías respiratorias altas o bajas en las 4 semanas
    anteriores a la visita de selección.
    7.Antecedentes de neoplasia maligna en los últimos 5 años, excepto
    carcinoma basocelular o carcinoma de cuello uterino in situ.
    8.Cirugía mayor en los 6 meses anteriores a la visita de selección.
    9.Antecedentes de consumo excesivo de alcohol o de alcoholismo (es
    decir, más de 3 unidades al día o más de 21 unidades a la semana) en los 12 meses anteriores a la visita de selección.
    10.Antecedentes de dependencia farmacológica o consumo de drogas en los 12 meses anteriores a la visita de selección.
    11.Mujeres que estén embarazadas o en período de lactancia o que, si
    son potencialmente fértiles, no quieren utilizar métodos anticonceptivos aceptables ni la abstinencia sexual durante el estudio.
    12.Participación en otro estudio con un fármaco en investigación en las 4 semanas anteriores a la visita de selección.
    13.Antecedentes de hipersensibilidad o intolerancia a medicamentos en
    aerosol o a cromoglicato disódico
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is change from baseline in total symptom score recorded in the daily eDiary using the MAS Plus questionnaire at Week 6.
    El criterio de evaluación primario de eficacia, es el cambio respecto al
    periodo basal en la puntuación total de síntomas utilizando el
    cuestionario MAS Plus a las 6 semanas
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 6
    Semana 6
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include the changes from baseline in each organ system domain and in each symptom score using the MAS Plus questionnaire and the change from baseline in quality of life as measured using the MIQ and SF-36 Questionnaire at Week 6. Another secondary endpoint is the PGIC score.
    Criterios de eficacia secundarios incluyen los cambios desde el inicio de
    cada dominio del sistema de órganos y en cada síntoma anotar utilizando
    el cuestionario MAS Plus y el cambio desde el inicio de la calidad de vida
    medida mediante el MIQ y SF-36 en la Semana 6. Otro objetivo
    secundario es la puntuación PGIC
    E.5.2.1Timepoint(s) of evaluation of this end point
    Week 6
    Semana 6
    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 No
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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 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 Yes
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    E.8.2.2Placebo Yes
    E.8.2.3Other Yes
    E.8.2.3.1Comparator description
    Nalcrom® 100 mg oral capsules
    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.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA6
    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
    Ultimo Paciente ultima visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months12
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial months12
    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: 30
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 6
    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 state6
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 36
    F.4.2.2In the whole clinical trial 36
    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
    Ninguno
    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 Decision2015-02-18
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-01-30
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2016-06-22
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