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    Summary
    EudraCT Number:2019-002754-22
    Sponsor's Protocol Code Number:TCC-201
    National Competent Authority:Portugal - INFARMED
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-06-18
    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 ConcernedPortugal - INFARMED
    A.2EudraCT number2019-002754-22
    A.3Full title of the trial
    ACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period
    ACcomplisH: Um ensaio de Fase 2 multicêntrico, com dupla ocultação, aleatorizado e controlado com placebo de escalonamento da dose para avaliar a segurança, eficácia e farmacocinética das doses subcutâneas de TransCon CNP administrado uma vez por semana durante 52 semanas em crianças pré-púberes com acondroplasia seguido de um Período de Extensão Sem Ocultação
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension Period
    Um ensaio de Fase 2 para avaliar a segurança, eficácia e farmacocinética de TransCon CNP em crianças pré-púberes com acondroplasia seguido de um Período de Extensão Sem Ocultação
    A.3.2Name or abbreviated title of the trial where available
    ACcomplisH
    ACcomplisH
    A.4.1Sponsor's protocol code numberTCC-201
    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 SponsorAscendis Pharma Growth Disorders A/S
    B.1.3.4CountryDenmark
    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 supportAscendis Pharma Growth Disorders A/S
    B.4.2CountryDenmark
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAscendis Pharma A/S
    B.5.2Functional name of contact pointClinical Trial Information Desk
    B.5.3 Address:
    B.5.3.1Street AddressTuborg Boulevard 12
    B.5.3.2Town/ cityHellerup
    B.5.3.3Post codeDK-2900
    B.5.3.4CountryDenmark
    B.5.4Telephone number004570222244
    B.5.6E-mailclinhelpdesk@ascendispharma.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 nameTransCon CNP 3.9 mg CNP-38/vial
    D.3.2Product code TransCon CNP
    D.3.4Pharmaceutical form Powder and solvent 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 INNC-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
    D.3.9.1CAS number 2413551-27-4
    D.3.9.2Current sponsor codeTransCon CNP
    D.3.9.3Other descriptive nameC-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
    D.3.9.4EV Substance CodeSUB193224
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number3.9
    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 and solvent for solution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    D.8 Placebo: 2
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Achondroplasia (ACH) in prepubertal children
    Acondroplasia (ACH) em crianças pré-púberes
    E.1.1.1Medical condition in easily understood language
    Achondroplasia (skeletal growth disorder) in children
    Acondroplasia (distúrbio do crescimento esquelético) em crianças
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 25.0
    E.1.2Level LLT
    E.1.2Classification code 10000452
    E.1.2Term Achondroplasia
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    In prepubertal children with achondroplasia (ACH) at 52 weeks,
    • To determine the safety of once weekly subcutaneous (SC) doses of
    TransCon CNP
    • To evaluate the effect of once weekly SC doses of TransCon CNP on
    annualized height velocity (AHV)
    Em crianças pré-púberes com acondroplasia ao 52 semanas • Determinar a segurança das doses subcutâneas (SC) de TransCon CNP administradas uma vez por semana • Avaliar o efeito das doses SC de TransCon CNP administradas uma vez por semana na velocidade de altura anualizada (VAA)
    E.2.2Secondary objectives of the trial
    • To evaluate the effect of once weekly SC doses of TransCon CNP on body proportionality (upper to lower body segment ratio) in prepubertal children with ACH at 52 weeks
    • To evaluate the pharmacokinetic (PK) properties of once weekly SC doses of TransCon CNP
    • To assess the potential immunogenic response to once weekly SC doses of TransCon CNP
    • Avaliar o efeito das doses SC de TransCon CNP administradas uma vez por semana na proporcionalidade corporal (razão de segmento corporal superior a inferior) em crianças pré-púberes com acondroplasia aos 52 semanas
    • Avaliar as propriedades farmacocinéticas (PK) das doses SC de TransCon CNP administradas uma vez por semana
    • Avaliar a potencial resposta imunogénica às doses SC de TransCon CNPadministradas uma vez por semana
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Clinical diagnosis of ACH with genetic confirmation
    2. Age between 2 to 10 years old (inclusive) at Screening Visit
    3. Prepubertal (Stage 1 breasts for girls or testicular volume < 4ml for boys) at Screening Visit
    4. Able to stand without assistance
    5. Caregiver willing and able to administer subcutaneous injections of study drug
    6. Written, signed informed consent of the parent(s) or legal guardian(s) of the participant and written assent of the participant as required by the institutional review board/human research ethics committee/independent ethics committee (IRB/HREC/IEC).
    1. Diagnóstico clínico de acondroplasia com confirmação genética
    2. Idade entre 2 a 10 anos (inclusive) na Visita de Triagem
    3. Pré-púbere (seios em Fase 1 para as raparigas ou volume testicular < 4 ml para os rapazes) na Visita de Triagem
    4. Capacidade de se levantar sem ajuda
    5. Cuidador disposto e capacitado para administrar injeções subcutâneasdo medicamento em estudo
    6.Consentimento informado por escrito e assinado pelo(s) progenitor(es) ou representante(s) legal(is) do participante e assentimento por escrito do participante,conforme exigido pela comissão de revisão institucional/comissão de éticapara investigação em humanos/comissão de ética independente (CRI/CEIH/CEI)

    E.4Principal exclusion criteria
    1.Clinically significant findings at Screening that:
    •are expected to require surgical intervention during participation in the trial or
    •are musculoskeletal in nature, such as Salter-Harris fractures and severe hip pain or
    •otherwise are considered by investigator or Medical Monitor to make a participant unfit to receive study drug or undergo trial related procedures
    2.Have received treatment (>3 months) of human growth hormone (hGH) or other medications known to affect stature or body proportionality at any time
    3.Have received any dose of medications intended to affect stature or body proportionality within the previous 6 months of Screening Visit
    4.Have received any study drug or device intended to affect stature or body proportionality at any time
    5.History or presence of injury or disease of the growth plate(s), other than ACH, that affects growth potential of long bones
    6.History of any bone-related surgery that affects growth potential of long bones, such as orthopedic reconstructive urgery and osteotomy (Limb-lengthening with full recovery is allowed with a minimum of 12 months of bone healing. Foramen magnum decompression and laminectomy with full recovery are allowed with minimum of 6 months of bone healing. History of 8 plate epiphysiodesis is allowed, but the plates must have been removed prior to Screening with minimum 4 weeks of healing).
    7.Have a form of skeletal dysplasia other than ACH or known medical conditions that result in short stature or abnormal growth.
    8.History or presence of malignant disease, other than basal cell epithelioma/carcinoma or completely resected squamous skin cancer with no recurrence for 12 months per medical records
    9.History or presence of the following:
    •Chronic anemia (resolved iron deficiency anemia is allowed)
    •Significant cardiovascular disease per the judgement of the investigator, such as congenital heart disease (uncomplicated patent ductus arteriosus and atrial or ventricular septal defect with repair are allowed), aortic insufficiency, clinically significant arrhythmias, congestive heart failure with NYHA class II and above, or other conditions that impair regulation of blood pressure or heart rate
    •Condition that impacts hemodynamic stability (such as autonomic dysfunction, orthostatic intolerance)
    •History of chronic renal insufficiency
    •Chronic or recurrent illness that can affect hydration or volume status. This may include conditions associated with decreased nutritional intake
    or increased volume loss
    •Bone fracture within 6 months prior to Screening Visit (within 2 months for fracture of digits)
    •Any disease or condition that, in the opinion of the investigator, may make the participant unlikely to fully complete the trial, may confound interpretation of trial results, or presents undue risk from receiving study drug
    10.Child has significant electrocardiogram abnormalities, including evidence of a previous myocardial infarction, left ventricular hypertrophy, flat T waves (particularly in the inferior leads) or more
    than minor non-specific ST-T wave changes or:
    •QRS>90 milliseconds (msec)
    •QT interval corrected using Fridericia's formula (QTcF)>440 msec
    •PR interval>170 msec
    •Complete right or left bundle branch block
    11.Requires, or anticipated to require, chronic (>4 weeks) or repeated (more than twice per year) treatment with oral corticosteroids during participation in the trial (low and mid-dose inhaled corticosteroids are allowed. High-dose inhaled corticosteroids are not allowed.). See Appendix 4 for definition of high doses of inhaled corticosteroids.
    12.Use of medication known to prolong the QT/QTc interval within 7 days or 5 half-lives (whichever is longer) prior to the Screening Visit (see https://crediblemeds.org/ for the list of medications that are
    known to prolong the QT/QTc interval. Note: Only medications on the
    Known Risk list are excluded, not those on the Possible or Conditional Risk lists)
    13.Ongoing treatment with any medication that affects blood pressure or heart rate
    14.Known hypersensitivity to the components of the study drug [trehalose, tris(hydroxymethyl)aminomethane, succinate and PEG]
    15.Any other reason that in the opinion of the investigator would prevent the child from complying with the trial requirements or completing the trial

    1. Conclusões clinicamente significativas na Triagem que: podem exigir uma intervenção cirúrgica durante a participação no ensaio, ou sejam denatureza musculoesquelética, como fraturas de Salter-Harris e dor da anca intensa, ou de outra forma, considerados pelo investigador ou Monitor Clínico como impróprios para um participante receber o medicamento em estudo ou ser submetido a procedimentos relacionados com o ensaio
    2. Ter recebido tratamento (>3 meses) com hormona de crescimento humano (hGH) ou outros medicamentos destinados a afetar a estatura ou proporcionalidade corporal em qualquer momento
    3. Ter recebido qualquer dose de medicamentos destinados a afetar a estatura ou proporcionalidade corporal nos 6 meses anteriores à Visita de Triagem
    4. Ter recebido qualquer medicamento ou dispositivo em estudo destinado a afetar a estatura ou proporcionalidade corporal em qualquer momento
    5. História ou presença de lesão ou doença da(s) placa(s) de crescimento, que não a acondroplasia, que afete o potencial de crescimento dos ossos longos
    6. História de qualquer cirurgia relacionada com os ossos que afete o potencial de crescimento dos ossos longos, como por exemplo, cirurgia reconstrutiva ortopédica e osteotomia (alongamento dos membros com total recuperação é permitido com um mínimo de 12 meses de cicatrização óssea. Descompressão do forame magno e laminectomia com total recuperação são permitidas com um mínimo de 6 meses de cicatrização óssea. São permitidos antecedentes de 8 placas de epifisiodese, mas as placas terão de ter sido removidas antes da triagem,com um mínimo de 4 semanas de cicatrização).
    7. Ter uma forma de displasia esqueléticas que não seja acondroplasia ou situações clínicas conhecidas que resultem em estatura baixa ou crescimento anormal dos ossos.
    8. História ou presença de doença maligna que não seja carcinoma/epitelioma basocelular ou carcinoma de células escamosas cutâneo completamente ressecado sem qualquer recidiva durante 12 meses, segundo os registos medico
    9.História ou presença das seguintes condições:•Anemia crónica (anemia por deficiência de ferro resolvida é permitido)•Doença cardiovascular importante, segundo o julgamento do investigador, tal como doença cardíaca congénita (ducto arterioso persistente não complicado e defeito do septo atrial ou ventricular com reparação são permitidos), insuficiência aórtica, arritmias clinicamente significativas, insuficiência cardíaca congestiva de Classe II e superior, de acordo com a classificaçãoda NYHA,ou outras condições que comprometam a regulação da tensão arterial ou do ritmo cardíaco•Condição com impacto na estabilidade hemodinâmica (tal como disfunção autonómica, intolerância ortostática)•História de insuficiência renal crónica •Doença crónica ou recorrente que pode afetar a hidratação ou o estado do volume.Isto pode incluir condições associadas a uma reduzida ingestão nutricional ou a uma acrescida perda de volume•Fratura óssea nos 6meses anteriores à Visita de Triagem (dois meses em caso de fratura de dedos)•Qualquer doença ou condição que, na opinião do investigador, possa tornar o participante incapaz de concluir o ensaio, possa confundir a interpretação dos resultados do ensaio ou constitua um risco indevido para receber o medicamento em estudo
    10. A criança tem alterações significativas no eletrocardiograma, incluindo evidências de um enfarte do miocárdio anterior, hipertrofia ventricular esquerda, ondas T planas (especialmente nas derivações inferiores) ou alterações mais do que ligeiras das ondas ST-T não específicas, ou: • QRS >90 milissegundos (msec)• intervalo QT corrigido usando a fórmula de Fridericia (QTcF) >440 msec• Intervalo PR >170 msec• Bloqueio completo do ramo direito ou esquerdo
    11.Necessidade, ou esperada necessidade, de tratamento crónico(>4semanas)ou repetido (mais de duas vezes por ano) com corticosteroides orais durante a participação no ensaio (doses baixas e médias de corticosteroides inaladossão permitidas. Doses elevadas de corticosteroides inalados não são permitidas). Ver 0para definição de doses elevadas de corticosteroides inalados.
    12. Uso de medicamentos conhecidos por prolongar o intervalo QT/QTc em 7 dias ou 5 semividas (consoante o que for mais longo) antes da Visita de Triagem (ver https://crediblemeds.org/ para a lista de medicamentos conhecidos por prolongar o intervalo QT/QTc. Nota: Apenas estão excluídos medicamentos da lista de Risco Conhecido e não medicamentos das listas de Risco Possível ou Condicional)
    13. Tratamento contínuo com qualquer medicamento que afete a tensãoarterial ou ritmo cardíaco
    14. Conhecida hipersensibilidade aos excipientes do medicamento em estudo [trealose, tris(hidroximetil)aminometano, succinato e PEG]
    15. Qualquer outra razão que, na opinião do investigador, impediria a criança de cumprir com as exigências do ensaio ou concluí-lo.
    E.5 End points
    E.5.1Primary end point(s)
    Safety Endpoints
    The following safety endpoints will be assessed for both blinded Randomised Period and Open-Label Extension Period
    • Incidence of AEs
    • All blood chemistry, hematology, lipid panel, and urinalysis parameters
    • Vital sign measurements and physical examination assessments
    • 12-lead ECG
    • Radiographic findings from:
    - Bone age X-ray
    - DXA
    - AP standing lower extremity X-ray
    - AP and lateral spine X-ray
    • Incidence of anti-drug antibodies

    Efficacy Endpoints
    • AHV as measured at 52 weeks of weekly TransCon CNP treatment or placebo

    Pharmacokinetic Endpoints
    • Plasma concentration of Total CNP
    • Plasma concentration of Free CNP
    • Plasma concentration of mPEG and mPEG-linker
    Endpoints de segurança
    Os seguintes endpoints de segurança serão avaliados tanto para o Período de Aleatorização em ocultação como para o Período de Extensão Sem Ocultação
    •Incidência de AA•Todos os parâmetros de bioquímica, hematologia, lipidograma e urinálise•Medições dos sinais vitais e avaliações do exame físico•ECG de 12 derivações•Conclusões radiográficas de:
    Raio-X da idade ósseaDXARaio-X AP das extremidades inferiores em pé Raio-X AP e lateral da coluna
    •Incidência de anticorpos antifármac
    Endpoints de eficácia

    VAA como medida às 52semanasde tratamento com TransCon CNP semanalou placebo
    Endpoints farmacocinéticos
    • Concentração plasmática de CNP total• Concentração plasmática de CNP livre• Concentração plasmática de mPEG e do ligando mPEG
    E.5.1.1Timepoint(s) of evaluation of this end point
    Efficacy will be evaluated at 52 weeks but safety and PK will be
    evaluated at 52 weeks and during the open-label extension period
    A eficácia será avaliada em 52 semanas, mas a segurança e a farmacocinética serão avaliado em 52 semanas e durante o período de extensão de rótulo aberto
    E.5.2Secondary end point(s)
    • Change in upper to lower body segment ratio as measured at 52 weeks of weekly TransCon CNP treatment or placebo
    Alteração na razão do segmento corporal superior a inferior como medido às 52semanasde tratamento com TransCon CNP semanalou placebo
    E.5.2.1Timepoint(s) of evaluation of this end point
    At 52 weeks of weekly TransCon CNP treatment or placebo
    Aos 52 semanas de tratamento com TransCon CNP administrado uma vez por semanaou placebo
    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 Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    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
    Dose escalation trial
    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 trial6
    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 EEA11
    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
    New Zealand
    Switzerland
    Australia
    Canada
    United Kingdom
    United States
    Austria
    Denmark
    Germany
    Ireland
    Portugal
    Spain
    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
    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 months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years4
    E.8.9.2In all countries concerned by the trial months4
    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: 60
    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: 60
    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 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 state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 60
    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)
    Participants who are not continuing in the Open-Label Extension Period
    will attend a follow-up visit 5 weeks after Visit 7 to review any adverse
    events, to collect blood samples for assessment of anti-drug antibodies
    and answer any questions.
    Participantes que não estão continuando no período de extensão de rótulo aberto comparecerá a uma visita de acompanhamento 5 semanas após a Visita 7 para revisar qualquer eventos, para coletar amostras de sangue para avaliação de anticorpos anti-drogase responda a quaisquer perguntas.
    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 Decision2020-08-27
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-05
    P. End of Trial
    P.End of Trial StatusOngoing
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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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