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    Summary
    EudraCT Number:2021-003149-39
    Sponsor's Protocol Code Number:C4181008
    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:2021-09-17
    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 number2021-003149-39
    A.3Full title of the trial
    A PHASE 2 OPEN LABEL EXTENSION STUDY TO ASSESS THE LONG-TERM SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIA
    ESTUDIO DE EXTENSIÓN ABIERTO EN FASE II PARA EVALUAR LA SEGURIDAD, TOLERABILIDAD, FARMACOCINÉTICA Y EFICACIA A LARGO PLAZO DE RECIFERCEPT EN NIÑOS CON ACONDROPLASIA
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2 study of long-term safety, tolerability, PK and efficacy of recifercept in achondroplasia
    Estudio en fase II de la seguridad, tolerabilidad, FC y eficacia a largo plazo de recifercept en la acondroplasia
    A.4.1Sponsor's protocol code numberC4181008
    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 SponsorPfizer 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 supportPfizer Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationPfizer Inc.
    B.5.2Functional name of contact pointClinical Trials.gov Call Centre
    B.5.3 Address:
    B.5.3.1Street Address235 East 42nd Street
    B.5.3.2Town/ cityNew York
    B.5.3.3Post codeNY 10017
    B.5.3.4CountryUnited States
    B.5.4Telephone number+1800718-1021
    B.5.6E-mailClinicalTrials.gov_Inquiries@pfizer.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/3/17/1843
    D.3 Description of the IMP
    D.3.1Product nameRecifercept (proposed INN)
    D.3.2Product code PF-07256472
    D.3.4Pharmaceutical form Lyophilisate for solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRecifercept (proposed INN)
    D.3.9.2Current sponsor codePF-07256472
    D.3.9.3Other descriptive nameTA-46
    D.3.9.4EV Substance CodeSUB190544
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    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) Yes
    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 Yes
    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
    Achondroplasia
    Acondroplasia
    E.1.1.1Medical condition in easily understood language
    Short stature
    Baja estatura
    E.1.1.2Therapeutic area Body processes [G] - Bones and nerves physological processes [G11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10000452
    E.1.2Term Achondroplasia
    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
    - Evaluate the long-term safety and tolerability of recifercept doses and dosing regimes in participants aged ≥15m to <12 years with achondroplasia.

    - To assess long-term efficacy of recifercept to increase height growth in children with achondroplasia.
    - Evaluar la seguridad y la tolerabilidad a largo plazo de dosis y pautas posológicas de recifercept en participantes de ≥15 meses a <12 años con acondroplasia.

    -Evaluar la eficacia a largo plazo de recifercept para aumentar el crecimiento de la estatura en niños con acondroplasia.
    E.2.2Secondary objectives of the trial
    • To evaluate the PK of recifercept in children aged ≥15m to <12 years old with achondroplasia.

    • To assess efficacy of recifercept to improve achondroplasia-related complications.

    • Assess change in individual safety parameters.
    - Evaluar la FC de recifercept en niños de ≥15 meses a <12 años con acondroplasia.

    - Evaluar la eficacia de recifercept para mejorar complicaciones relacionadas con la acondroplasia.

    - Evaluar la variación de los parámetros de seguridad individuales.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Age and Sex:
    1. Male and female participants between the ages of ≥15 months to ≤12 years inclusive, at Visit 1 (Screen 1).
    • Refer to Appendix 4 for reproductive criteria for male (Section 10.4.1) and female (Section 10.4.2) participants.

    Type of Participant and Disease Characteristics:
    2. Participants who are willing and able to comply with all scheduled visits, treatment plan, laboratory tests lifestyle considerations and other study procedures.
    3. Completed the C4181005 Phase 2 study.
    4. Able to stand independently for height measurements (if ≥2 years of age at enrollment).

    Informed Consent:
    5. Capable of giving signed informed consent/assent as described in Appendix 1, which includes compliance with the requirements and restrictions listed in the ICD and in this protocol.
    6. Following receipt of oral and written information about the trial, the child (depending on local IRB/independent EC requirements) must provide assent, and 1 or both (according to local regulations) parent(s) or legal guardians of the child must provide signed informed consent before any trial-related activity is carried out.
    Edad y sexo:
    1. Participantes de sexo masculino y femenino entre >15 meses y <12 años, inclusive, en la visita 1 (selección 1).
    - Consulte el Apéndice 4 del protocolo para ver los criterios reproductivos para los participantes de sexo masculino (Sección 10.4.1 del protocolo) y de sexo femenino (Sección 10.4.2 del protocolo).

    Tipo de participante y características de la enfermedad:
    2. Los participantes deben estar dispuestos y ser capaces de cumplir con todas las visitas programadas, el plan de tratamiento, las pruebas analíticas, las consideraciones sobre el estilo de vida y otros procedimientos del estudio.
    3. Completaron el estudio de fase II C4181005.
    4. Capacidad para permanecer de pie de forma independiente para las mediciones de la estatura (si ≥2 años de edad en el momento de la inscripción).

    Consentimiento informado:
    5. Capacidad de otorgar el consentimiento/asentimiento informado firmado, tal y como se describe en el Apéndice 1 del protocolo, lo que incluye el cumplimiento de los requisitos y las restricciones indicadas en el DCI y en este protocolo.
    6. Tras recibir la información verbalmente y por escrito sobre el ensayo, el niño (en función de los requisitos del comité institucional de evaluación [CIE]) debe otorgar el asentimiento, y 1 o ambos (de acuerdo con la normativa local) de los progenitores o tutores legales del niño deben otorgar el consentimiento informado firmado antes de llevar a cabo cualquier actividad relacionada con el ensayo.
    E.4Principal exclusion criteria
    Medical Conditions:
    1. Presence of co-morbid conditions or circumstances that, in the opinion of the investigator, would affect interpretation of growth data or ability to complete the trial procedures.
    2. Other medical or psychiatric condition including recent (within the past year) or active suicidal ideation/behavior or laboratory abnormality that may increase the risk of study participation or, in the investigator’s judgment, make the participant inappropriate for the study.
    3. Presence of severe obesity (BMI >95th percentile on Hoover-Fong BMI charts) [Hoover-Fong et al, 2008].
    4. Known closure of long bone growth plates (cessation of height growth).
    5. Body weight ≥ 45 kg.
    6. History of hypersensitivity to study intervention or any excipients.

    Prior/Concomitant Therapy:
    7. Current use of any prohibited concomitant medication(s) or those unwilling/unable to use a permitted concomitant medication(s). Refer to Section 6.8 Concomitant Therapy.
    8. History of any prior treatment with human growth hormone or related products (including IGF-1).
    9. History of receipt of any treatment that are known to potentially affect growth (including oral steroids >5 days in the last 6 months, high dose inhaled corticosteroids (>800 µg/day beclomethasone equivalent) and medication for attention deficit hyperactivity disorder).
    10. History of limb lengthening surgery (defined as distraction osteogenesis/Ilizarov/callostasis technique following submetaphyseal osteotomy to extend bone length).
    11. Any limb lengthening/corrective orthopaedic surgery planned at any point during the trial period.
    12. Less than 6 months since fracture or surgical procedure of any bone determined from the screening visit date.
    13. Presence of any internal guided growth plates/devices.
    14. History of removal of internal guided growth plates/devices within less than 6 months.

    Prior/Concurrent Clinical Study Experience:
    15. History of receipt of any other (except recifercept) IP for achondroplasia or that may affect growth/interpretation of growth parameters.
    16. Previous administration with an investigational drug (not for achondroplasia/growth affecting) within 30 days (or as determined by the local requirement) or 5 half lives preceding the first dose of study intervention used in this study (whichever is longer).

    Other Exclusions:
    17. Investigator site staff or Pfizer employees directly involved in the conduct of the study, site staff otherwise supervised by the investigator, and their respective family members.
    Enfermedades:
    1. Presencia de afecciones o circunstancias concomitantes que, en opinión del investigador, puedan afectar a la interpretación de los datos de crecimiento o la capacidad de completar los procedimientos del ensayo.
    2. Otras enfermedades o alteraciones psiquiátricas, incluidos los comportamientos o ideas suicidas recientes (en el último año), o anomalías de laboratorio que puedan aumentar el riesgo de la participación en el estudio o, a juicio del investigador, hagan que el participante no sea apto para el estudio.
    3. Presencia de la obesidad importante (IMC > percentil 95 según las tablas de IMC de Hoover-Fong) [Hoover-Fong et al, 2008].
    4. Antecedentes conocidos de cierre de las placas de crecimiento de los huesos largos (cese del crecimiento de la estatura).
    5. Peso corporal >45 kg.
    6. Antecedentes de hipersensibilidad al tratamiento del estudio o a cualquier excipiente.

    Tratamiento previo/concomitante:
    7. Uso actual de cualquier medicamento concomitante prohibido o aquellos que no estén dispuestos/no puedan usar medicamentos concomitantes permitidos. Consulte la Sección 6.8 del protocolo (Tratamiento concomitante).
    8. Antecedentes de cualquier tratamiento anterior con hormona de crecimiento humana o productos relacionados (incluido el IGF-1).
    9. Antecedentes de recepción de cualquier tratamiento conocido por afectar al crecimiento (incluyendo corticoesteroides por vía oral >5 días en los últimos 6 meses, dosis altas de corticoesteroides inhalados [>800 μg/día de beclometasona o equivalente] y medicación para el trastorno por déficit de atención con hiperactividad).
    10. Antecedentes de cirugía de alargamiento de extremidades (definida como osteogénesis por distracción/técnica de Ilizarov/callostasis después de una osteotomía submetafisaria para extender la longitud del hueso).
    11. Cualquier cirugía de alargamiento de extremidades/ortopédica correctiva prevista en cualquier momento durante el período del ensayo.
    12. Haber transcurrido un período inferior a 6 meses desde una fractura o procedimiento quirúrgico de cualquier hueso determinado a partir de la fecha de la visita de selección.
    13. Presencia de cualquier placa/dispositivo de crecimiento guiado interno.
    14. Antecedentes de retirada de cualquier placa/dispositivo de crecimiento guiado interno, en un plazo inferior a 6 meses.

    Experiencia en estudios clínicos previos o simultáneos:
    15. Antecedentes de recepción de cualquier otro PEI (excepto recifercept) para la acondroplasia o que pueda afectar al crecimiento/interpretación de los parámetros de crecimiento.
    16. Administración previa de un fármaco en investigación (que no sea para la acondroplasia/que no afecte al crecimiento) en los 30 días (o según lo determine el requisito local) o 5 semividas previos a la primera dosis del tratamiento del estudio utilizado en este estudio (lo que dure más).

    Otras exclusiones:
    17. Empleados del centro de investigación o de Pfizer directamente implicados en la realización del estudio, empleados del centro supervisados de alguna otra manera por el investigador, y sus respectivos familiares.
    E.5 End points
    E.5.1Primary end point(s)
    • The primary efficacy estimand is intended to provide a population level estimate of the effect of the IP on a continuous endpoint.
    • Population-level summary: ratio between participants in the trial and a reference population [Merker et al, 2018] in growth of height at 24 month; ratio between treated and reference population is observed change-from-baseline of treated participants standardized by reference participant given age and gender.
    - El estimando de la eficacia principal está diseñado para ofrecer un cálculo a nivel poblacional del efecto del PEI en un criterio de valoración continuo.
    - Resumen a nivel de población: cociente entre los participantes en el ensayo y una población de referencia [Merker et al, 2018] en el crecimiento de la estatura a los 24 meses; el cociente entre la población tratada y la de referencia es la variación con respecto al valor inicial observada en los participantes tratados estandarizada según el participante de referencia de una edad y sexo determinados.
    E.5.1.1Timepoint(s) of evaluation of this end point
    D1, 91, 181, 271, 361, 451, 541, 631, 721
    D1, 91, 181, 271, 361, 451, 541, 631, 721
    E.5.2Secondary end point(s)
    N/A
    N/A
    E.5.2.1Timepoint(s) of evaluation of this end point
    N/A
    N/A
    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 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 Yes
    E.8.1.7.1Other trial design description
    Estudio de extensión abierto
    Open Label Extension study
    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 trial3
    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 EEA5
    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
    Australia
    Belgium
    Denmark
    Italy
    Japan
    Portugal
    Spain
    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
    Último paciente última visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days27
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days27
    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: 63
    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) Yes
    F.1.1.4.1Number of subjects for this age range: 9
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 54
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    63 children with achondroplasia aged ≥ 15 months to ≤12 years inclusive.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 40
    F.4.2.2In the whole clinical trial 63
    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)
    Once a final therapeutic dose has been identified, the Pfizer IRC may decide to extend the study treatment duration and data collection (including final adult height) period beyond 24 months. Any significant changes to the protocol will be made via substantial protocol amendment.

    No intervention will be provided to study participants at the end of their study participation.
    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 Decision2021-12-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-12-13
    P. End of Trial
    P.End of Trial StatusOngoing
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