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    Summary
    EudraCT Number:2021-003149-39
    Sponsor's Protocol Code Number:C4181008
    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:2021-12-14
    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 ConcernedItaly - Italian Medicines Agency
    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
    STUDIO DI ESTENSIONE IN APERTO DI FASE 2 VOLTO A VALUTARE LA SICUREZZA, LA TOLLERABILITÀ, LA FARMACOCINETICA E L’EFFICACIA A LUNGO TERMINE DI RECIFERCEPT NEI BAMBINI 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
    Studio di fase 2 su sicurezza, tollerabilità, farmacocinetica ed efficacia a lungo termine di recifercept nell’acondroplasia
    A.3.2Name or abbreviated title of the trial where available
    -
    -
    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+18007181021
    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
    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
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee 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 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
    bassa statura
    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 No
    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.
    - Valutare la sicurezza e la tollerabilità a lungo termine delle dosi e dei regimi posologici di recifercept nei partecipanti di età compresa tra =15 mesi e <12 anni affetti da acondroplasia.
    - Valutare l’efficacia a lungo termine di recifercept nel far aumentare l’altezza dei bambini 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.
    - Valutare la PK di recifercept in bambini di età compresa tra =15 mesi e <12 anni affetti da acondroplasia.
    - Valutare l’efficacia di recifercept per migliorare le complicazioni correlate all’acondroplasia.
    -Valutare la variazione dei parametri di sicurezza personali.
    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.
    Età e sesso:
    1. Partecipanti di sesso maschile e femminile di età compresa tra >15 mesi e <12 anni inclusi, alla Visita 1 (Screening 1).
    Fare riferimento all’Appendice 4 per i criteri riproduttivi dei partecipanti di sesso maschile (Sezione 10.4.1 del protocollo) e femminile (Sezione 10.4.2 del protocollo).
    Caratteristiche dei partecipanti e della malattia:
    2. Partecipanti che abbiano la volontà e la capacità di rispettare il programma delle visite, il piano di trattamento, i test di laboratorio, le considerazioni relative allo stile di vita e altre procedure dello studio.
    3. Completamento dello studio C4181005 di fase 2.
    4. Capacità di stare in piedi autonomamente per misurare l’altezza (se età =2 anni all’arruolamento).
    Consenso informato:
    5. Capacità di fornire il consenso/assenso informato firmato come descritto nell’Appendice 1 del protocollo, che include la conformità ai requisiti e alle restrizioni elencati nel DCI e nel presente protocollo.
    6. Assenso del bambino (in base ai requisiti del Consiglio di revisione istituzionale [IRB]/Comitato etico indipendente [CEI] locale) e firma del consenso informato da parte di 1 o entrambi i genitori o tutori legali (in base alle normative locali) forniti in seguito alla ricezione di informazioni orali e scritte sulla sperimentazione e prima di avviare qualsiasi attività correlata
    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.
    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
    Condizioni mediche:
    1. Presenza di condizioni di comorbilità o circostanze che, secondo il parere dello sperimentatore, potrebbero influenzare l’interpretazione dei dati sulla crescita o la capacità di completare le procedure della sperimentazione.
    2. Altra condizione medica o psichiatrica, compresa l’ideazione o la condotta suicidaria recente (nell’anno precedente) o attiva, oppure un’anomalia di laboratorio che potrebbe aumentare il rischio associato alla partecipazione allo studio o che, a giudizio dello sperimentatore, renderebbe il soggetto inadatto allo studio.
    3. Presenza di obesità grave (IMC >95o percentile nei grafici per IMC di Hoover-Fong) [Hoover-Fong et al, 2008].
    4. Nota chiusura delle cartilagini di accrescimento delle ossa lunghe (cessazione della crescita in altezza).
    5. Peso corporeo >45 kg.
    6. Anamnesi di ipersensibilità al trattamento dello studio o a qualsiasi eccipiente
    Terapie precedenti/concomitanti:
    7. Uso attuale di qualsiasi farmaco concomitante proibito o mancata volontà/capacità di assumere un farmaco concomitante consentito. Fare riferimento alla Sezione 6.8 Terapia concomitante del protocollo.
    8. Anamnesi di qualsiasi trattamento precedente con ormone della crescita umano o prodotti correlati (compreso l’IGF-1).
    9. Anamnesi di assunzione di qualsiasi trattamento noto per possibili effetti sulla crescita (ad es. steroidi per via orale >5 giorni nei 6 mesi precedenti, corticosteroidi per via inalatoria a dosi elevate [>800 µg/die beclametasone equivalente] e farmaci per il disturbo da deficit di attenzione e iperattività).
    10. Anamnesi di intervento chirurgico di allungamento degli arti (quale osteogenesi per distrazione/tecnica di Ilizarov/callotasi a seguito di osteotomia submetafisaria per l’estensione della lunghezza ossea).
    11. Previsione di qualsiasi intervento chirurgico ortopedico correttivo o per l’allungamento degli arti in qualsiasi momento nel periodo della sperimentazione.
    12. Frattura o procedura chirurgica di qualsiasi osso verificatasi nei 6 mesi precedenti alla data della visita di screening.
    13. Presenza di piastre/dispositivi interni per la crescita guidata.
    14. Anamnesi di rimozione di piastre/dispositivi interni per la crescita guidata nei 6 mesi precedenti.
    Esperienza pregressa/concomitante con studi clinici:
    15. Anamnesi di assunzione di qualsiasi altro IP per l’acondroplasia (tranne recifercept) o in grado di influire sulla crescita/sull’interpretazione dei parametri di crescita.
    16. Precedente somministrazione di un farmaco sperimentale (non per acondroplasia/in grado di influire sulla crescita) entro 30 giorni (o secondo quanto stabilito dai requisiti locali) o 5 emivite prima della prima dose del trattamento dello studio utilizzato nel presente studio (a seconda di quale sia il periodo più lungo).
    Altre esclusioni:
    17. Il personale del centro dello sperimentatore e i dipendenti di Pfizer direttamente coinvolti nella conduzione dello studio, il personale del centro altrimenti supervisionato dallo sperimentatore e i rispettivi familiari.
    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]
    - Sicurezza e tollerabilità a lungo termine di recifercept valutate in base alla frequenza e alla gravità di eventi avversi (AE)/eventi avversi seri (SAE).
    -Aumento dell’altezza al di sopra dei livelli attesi nella popolazione di riferimento [Merker et al, 2018].
    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.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 Yes
    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
    Studio di estensione in aperto
    Studio di estensione in aperto
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Yes
    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 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 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 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
    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
    LVLS
    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 days0
    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 days13
    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) 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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    63 children with achondroplasia aged = 15 months to =12 years inclusive.
    63 bambini con acondroplasia di età compresa tra = 15 mesi e =12 anni inclusi
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state8
    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.
    Una volta identificata una dose terapeutica finale, l'IRC di Pfizer può decidere di estendere la durata del trattamento in studio e il periodo di raccolta dei dati (compresa l'altezza finale dell'adulto) oltre i 24 mesi. Eventuali modifiche significative al protocollo verranno apportate tramite una modifica sostanziale del protocollo.
    Nessun intervento sarà fornito ai partecipanti allo studio al termine della loro partecipazione allo 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 Decision2022-05-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-01-20
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2022-11-18
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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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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