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    Summary
    EudraCT Number:2018-002502-31
    Sponsor's Protocol Code Number:RP-L102-0118
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-08-02
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2018-002502-31
    A.3Full title of the trial
    A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A
    Ensayo clínico en Fase II para evaluar la eficacia de la infusión de células autólogas CD34+ transducidas con un vector lentiviral portador del gen FANCA (Medicamento Huérfano) en pacientes con anemia de Fanconi subtipo A.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A
    Ensayo clínico en Fase II para evaluar la eficacia de la infusión de células autólogas CD34+ transducidas con un vector lentiviral portador del gen FANCA (Medicamento Huérfano) en pacientes con anemia de Fanconi subtipo A.
    A.3.2Name or abbreviated title of the trial where available
    FANCOLEN-II
    FANCOLEN-II
    A.4.1Sponsor's protocol code numberRP-L102-0118
    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 SponsorRocket Pharmaceuticals, 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 supportRocket Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlpha Bioresearch, S.L.
    B.5.2Functional name of contact pointTeresa Bricio
    B.5.3 Address:
    B.5.3.1Street AddressPso. de la Castellana, 163 2ºIzq
    B.5.3.2Town/ cityMadrid
    B.5.3.3Post code28046
    B.5.3.4CountrySpain
    B.5.4Telephone number34917452520
    B.5.5Fax number34917450653
    B.5.6E-mailteresa.bricio@alphabioresearch.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 Yes
    D.2.5.1Orphan drug designation numberEU/3/10/822
    D.3 Description of the IMP
    D.3.1Product nameRP-L102
    D.3.4Pharmaceutical form Suspension for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCD34+CELLS
    D.3.9.3Other descriptive nameCD34+CELLS
    D.3.10 Strength
    D.3.10.1Concentration unit IU/kg international unit(s)/kilogram
    D.3.10.2Concentration typenot less then
    D.3.10.3Concentration number500000
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product Yes
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberMedicamento de Terapia Génica, código H0005064
    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 Yes
    D.3.11.13.1Other medicinal product typeCD34+ cells (preferentially fresh) from patients with Fanconi anemia subtype A (FA-A) will be transduced ex vivo with a lentiviral vector carrying the FANCA gene (orphan drug).
    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
    Fanconi anemia (subtype A)
    Anemia de Fanconi (subtipo A)
    E.1.1.1Medical condition in easily understood language
    Fanconi anemia (subtype A)
    Anemia de Fanconi (subtipo A)
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10055206
    E.1.2Term Fanconi's anemia
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The main objective of this trial is to assess the therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene in patients with Fanconi anemia subtype A (complementation group A, FA-A).
    El objetivo principal de este ensayo es evaluar la eficacia terapéutica de un procedimiento de terapia génica con u medicamento huérfano consistente en un vector lentiviral portador del gen FANCA en pacientes con anemia de Fanconi subtipo A (grupo de complementación, A, FA-A).
    E.2.2Secondary objectives of the trial
    Not applicable
    No aplica
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent
    2. Patients of the complementation group FA-A
    3. Minimum age: 1 year and a minimum weight of 8 kg.
    4. Maximum age: 12 years
    5. At least one of the following hematologic parameters below lower limits of normal of
    • Hemoglobin
    • Absolute neutrophils
    • Platelets
    6. At least 30 CD34+ cells/μL are determined in one BM aspiration within 3 months prior to CD34+ cell collection
    7. If the number of C34+ cells/ μL in BM is in the range of 10-29, PB parameters should meet two of the three following criteria:
    • Hemoglobin: ≥11g/dL
    • Neutrophils: ≥900 cells/μL
    • Platelets: ≥60,000 cells/μL
    8. Provide informed consent in accordance with current legislation
    9. Women of childbearing age must have a negative urine pregnancy test at the baseline visit, and accept the use of an effective contraception method during participation in the trial
    1. Anemia de Fanconi diagnosticada mediante un ensayo de fragilidad cromosómica en linfocitos en cultivo en presencia de DEB o un agente inductor de entrecruzamiento del DNA similar
    2. Pacientes del grupo de complementación FA-A.
    3. Edad mínima: 1 año y un peso mínimo de 8 kg.
    4. Edad máxima: 12 años.
    5. Al menos uno de los siguientes parámetros hematológicos por debajo de los limites normales
    • Hemoglobina
    • Neutrófilos totales
    • Plaquetas
    6. Al menos 30 células CD34+ /µL determinadas en un aspirado de MO en los tres meses anteriores a la colecta de las células CD34+
    7. Si el numero de células C34+ / µL en MO esta en el rango de 10-29, los paramentos de sangre periférica deben cumplir dos de tres de los siguientes criterios:
    • Hemoglobina: ≥11g/dL
    • Neutrófilos ≥900 células/µL
    • Plaquetas: ≥60,000 células/µL
    8. Dar consentimiento informado de acuerdo con la legislación vigente.
    9. Mujeres en edad fértil deben dar negativo en un test de embarazo en orina en la visita de inicio, y aceptar el uso de un método anticonceptivo efectivo durante su participación en el ensayo.
    E.4Principal exclusion criteria
    Patients with an HLA-identical sibling donor
    2. Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities predictive of these conditions in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the patient enters the clinical trial
    3. Patients with somatic mosaicism associated with stable or improved counts in all PB cell lineages
    4. Lansky performance index ≤ 60%
    5. Any concomitant disease or condition that, in the opinion of the Principal Investigator, deems the patient unfit to participate in the trial
    6. Pre-existing sensory or motor impairment > = grade 2 according to the criteria of the National Cancer Institute (NCI)
    7. Pregnant or breastfeeding women
    8. Hepatic dysfunction as defined by either:
    • Bilirubin > 1.5 x the upper limit of normal (ULN)
    • Alanine aminotransferase (ALT ) > 2.5 x ULN
    • Aspartate aminotransferase (ALT) > 2.5 x ULN.
    9. Renal dysfunction requiring either hemodialysis or peritoneal dialysis
    10. Pulmonary dysfunction as defined by either:
    • Need for supplemental oxygen during the prior 2 weeks (in absence of acute infection)
    • Oxygen saturation (by pulse oximetry) <90%.
    11. Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years
    1. Pacientes con un hermano donante HLA-idéntico
    2. Evidencia de síndrome mielodisplasico o leucemia, o anormalidades citogenéticas predictivas de estas condiciones en un aspirado de MO. Esta valoración debe hacerse mediante estudios validaos en los 3 meses anteriores ala entrada del paciente en ensayo.
    3. Pacientes con mosaicismo somático asociado con contajes estables o mejorados de diferentes linajes en sangre periférica
    4. Índice de Lansky ≤ 60%
    5. Cualquier enfermedad concomitante que, en opinión del Investigador Principal, haga al paciente no apto para participar en el estudio.
    6. Deterioro sensorial o motor preexistente > = grado 2 según los criterios del Instituto Nacional del Cáncer (NCI)
    7. Mujeres embarazadas o lactante
    8. Disfunción hepática definida por cualquiera de los siguientes criterios:
    • Bilirrubina > 1.5 x limite normal superior (ULN)
    • Alanina aminotransferasa (ALT ) > 2.5 x ULN
    • Aspartato aminotransferasa (ALT) > 2.5 x ULN.
    9. Disfunción renal que requiera hemodiálisis o diálisis peritoneal.
    10. Disfunción pulmonar definida por cualquiera de los siguientes criterios:
    • Necesidad de suplemento de oxigeno durante las 2 semanas anteriores (en ausencia de infección aguda)
    • Saturación de oxigeno (por oximetría de pulso) <90%.
    11. Evidencia de malignidad avanzada localizada o metastásica para cuya supervivencia esperada sea menor de 3 años
    E.5 End points
    E.5.1Primary end point(s)
    • Level of phenotypic correction of hematopoietic cells after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A
    • Level of engraftment of gene-corrected hematopoietic cells after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A
    • Clinical response (prevention or rescue of bone marrow failure (BMF)) after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A
    • Ongoing evaluation of the short- and long-term safety of the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A
    • Nivel de corrección fenotípica de células hematopoyéticas después de la infusión de células CD34 + autólogas transducidas con el vector lentiviral terapéutico en pacientes con subtipo A de anemia de Fanconi
    • Nivel de injerto de células hematopoyéticas corregidas genéticamente después de la infusión de células autólogas CD34 + transducidas con el vector lentiviral terapéutico en pacientes con subtipo A de anemia de Fanconi.
    • Respuesta clínica (prevención o rescate del fallo de la médula ósea (BMF)) después de la infusión de células autólogas CD34 + transducidas con el vector lentiviral terapéutico en pacientes con anemia de Fanconi subtipo A
    • Evaluación continua de la seguridad a corto y largo plazo de la infusión de células autólogas CD34 + transducidas con el vector lentiviral terapéutico en pacientes con subtipo A de anemia de Fanconi.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Up to 3 years after infusion
    Hasta 3 años después de la infusión
    E.5.2Secondary end point(s)
    Not applicable
    No aplica
    E.5.2.1Timepoint(s) of evaluation of this end point
    Not applicable
    No aplica
    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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    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 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 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
    This trial will end when all recruited patients complete the scheduled assessments.
    El ensayo finalizará cuando todos los pacientes incluidos hayan completado el calendario de visitas.
    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 months6
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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: 5
    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: 5
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    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 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 Yes
    F.3.3.7.1Details of other specific vulnerable populations
    Children under 12 years old, will sign an Informed Consent Form by legal representative
    El representante legal firmará el Consentimiento Informado para los niños con edad inferior a 12 año
    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 5
    F.4.2.2In the whole clinical trial 5
    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)
    Patients will be followed-up for 3 years. However, patients will be monitored outside the clinical trial for a total period of 15 years.
    Se realizará un seguimiento a los pacientes durante 3 años. Sin embargo, los pacientes serán seguidos, fuera del ensayo clínico, por un periodo total de 15 años.
    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 Decision2018-10-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-10-30
    P. End of Trial
    P.End of Trial StatusOngoing
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