Esta página foi traduzida automaticamente e a precisão da tradução não é garantida. Por favor, consulte o versão em inglês para um texto fonte.

Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

22 de julho de 2017 atualizado por: Novartis Pharmaceuticals

A Randomized, Open-label, Multicenter, Two Arm, Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Tipo de estudo

Intervencional

Inscrição (Real)

173

Estágio

  • Fase 2

Contactos e Locais

Esta seção fornece os detalhes de contato para aqueles que conduzem o estudo e informações sobre onde este estudo está sendo realizado.

Locais de estudo

  • Alemanha
      • Berlin, Alemanha, 13353
        • Novartis Investigative Site
      • Dresden, Alemanha, 01307
        • Novartis Investigative Site
      • Goslar, Alemanha, 38642
        • Novartis Investigative Site
      • Hannover, Alemanha, 30170
        • Novartis Investigative Site
      • Leipzig, Alemanha, 04103
        • Novartis Investigative Site
      • Potsdam, Alemanha, 14467
        • Novartis Investigative Site
    • Baden-Württemberg
      • Mannheim, Baden-Württemberg, Alemanha, 68305
        • Novartis Investigative Site
  • Argentina
      • Buenos aires, Argentina, C1221ADC
        • Novartis Investigative Site
  • Arábia Saudita
      • Dammam, Arábia Saudita, 15215
        • Novartis Investigative Site
      • Dammam, Arábia Saudita, 40145
        • Novartis Investigative Site
      • Jeddah, Arábia Saudita, 21589
        • Novartis Investigative Site
      • Riyadh, Arábia Saudita, 11472
        • Novartis Investigative Site
  • Emirados Árabes Unidos
      • Al Ain - Abu Dhabi, Emirados Árabes Unidos
        • Novartis Investigative Site
      • Dubai, Emirados Árabes Unidos, 9115
        • Novartis Investigative Site
  • Espanha
      • Barcelona, Espanha, 08041
        • Novartis Investigative Site
      • Madrid, Espanha, 28033
        • Novartis Investigative Site
  • Estados Unidos
    • California
      • Orange, California, Estados Unidos, 92868-3874
        • Children's Hospital of Orange County Onc Dept
    • Illinois
      • Chicago, Illinois, Estados Unidos, 60611
        • Lurie Children's Hospital of Chicago Onc Dept
    • Massachusetts
      • Boston, Massachusetts, Estados Unidos, 02115
        • Children's Hospital Boston Department of Hematology
    • New York
      • New York, New York, Estados Unidos, 10021
        • Weill Cornell Medical College-Cornell University Onc Dept
    • Pennsylvania
      • Philadelphia, Pennsylvania, Estados Unidos, 19104-4399
        • Children's Hospital of Philadelphia Onc. Dept
  • Federação Russa
    • Russia
      • Moskow, Russia, Federação Russa, 117198
        • Novartis Investigative Site
  • França
      • Lille cedex, França, 59020
        • Novartis Investigative Site
      • Paris, França, 75010
        • Novartis Investigative Site
  • Grécia
    • GR
      • Athens, GR, Grécia, GR-115 27
        • Novartis Investigative Site
      • Patra - RIO, GR, Grécia, 265 04
        • Novartis Investigative Site
      • Thessaloniki, GR, Grécia, 546 42
        • Novartis Investigative Site
  • Itália
      • Napoli, Itália, 80138
        • Novartis Investigative Site
    • BR
      • Brindisi, BR, Itália, 72100
        • Novartis Investigative Site
    • CT
      • Catania, CT, Itália, 95125
        • Novartis Investigative Site
    • FE
      • Cona, FE, Itália, 44100
        • Novartis Investigative Site
    • GE
      • Genova, GE, Itália, 16132
        • Novartis Investigative Site
      • Genova, GE, Itália, 16128
        • Novartis Investigative Site
    • ITA
      • Cagliari, ITA, Itália, 09121
        • Novartis Investigative Site
    • LE
      • Lecce, LE, Itália, 73100
        • Novartis Investigative Site
    • MI
      • Milano, MI, Itália, 20162
        • Novartis Investigative Site
      • Milano, MI, Itália, 20122
        • Novartis Investigative Site
    • PA
      • Palermo, PA, Itália, 90127
        • Novartis Investigative Site
      • Palermo, PA, Itália, 90146
        • Novartis Investigative Site
    • RC
      • Reggio Calabria, RC, Itália, 89100
        • Novartis Investigative Site
    • VR
      • Verona, VR, Itália, 37126
        • Novartis Investigative Site
  • Líbano
    • Beirut
      • Hazmiyeh, Beirut, Líbano, PO BOX 213
        • Novartis Investigative Site
  • Malásia
      • Kuala Lumpur, Malásia, 50589
        • Novartis Investigative Site
      • Pulau Pinang, Malásia, 10990
        • Novartis Investigative Site
  • México
    • Distrito Federal
      • Mexico, Distrito Federal, México, 06726
        • Novartis Investigative Site
  • Reino Unido
      • London, Reino Unido, N19 5NF
        • Novartis Investigative Site
      • London, Reino Unido, NW1 2PJ
        • Novartis Investigative Site
  • Tailândia
      • Bangkok, Tailândia, 10700
        • Novartis Investigative Site
      • Bangkok, Tailândia, 10400
        • Novartis Investigative Site
  • Áustria
      • Linz, Áustria, A-4010
        • Novartis Investigative Site
      • Wien, Áustria, 1140
        • Novartis Investigative Site

Critérios de participação

Os pesquisadores procuram pessoas que se encaixem em uma determinada descrição, chamada de critérios de elegibilidade. Alguns exemplos desses critérios são a condição geral de saúde de uma pessoa ou tratamentos anteriores.

Critérios de elegibilidade

Idades elegíveis para estudo

10 anos e mais velhos (Filho, Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

Key Inclusion Criteria:

  • Male and female patients aged ≥ 10 years
  • Patients with transfusion-dependent thalassemia and iron overload, requiring deferasirox DT at doses of ≥ 30 mg/kg/day as per the investigator's decision OR Patients with very low, low or intermediate (int) risk myelodysplastic syndrome (MDS) and iron overload, requiring deferasirox DT at doses of ≥ 20 mg/kg/day as per the investigator's decision.
  • History of transfusion of at least 20 PRBC units and anticipated to be transfused with at least 8 units of PRBCs annually during the study
  • Serum ferritin > 1000 ng/mL, measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).

Key Exclusion Criteria:

  • Creatinine clearance below the contraindication limit in the locally approved prescribing information. Creatinine clearance will be estimated from serum creatinine at screening Visit 1 and screening Visit 2 and the mean value will be used for eligibility criteria.
  • Serum creatinine > 1.5 xULN at screening measured at screening Visit 1 and screening Visit 2 (the mean value will be used for eligibility criteria).
  • ALT (SGPT) > 5xULN, unless LIC confirmed as >10 mg Fe/dw within 6 months prior to screening visit 1.
  • Significant proteinuria as indicated by a urinary protein/creatinine ratio > 0.5 mg/mg in a non-first void urine sample at screening Visit 1 or screening Visit 2.
  • Patients with significant impaired gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral deferasirox (e.g. ulcerative diseases, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome, or small bowel resection).
  • Liver disease with severity of Child-Pugh Class B or C

Plano de estudo

Esta seção fornece detalhes do plano de estudo, incluindo como o estudo é projetado e o que o estudo está medindo.

Como o estudo é projetado?

Detalhes do projeto

  • Finalidade Principal: Tratamento
  • Alocação: Randomizado
  • Modelo Intervencional: Atribuição Paralela
  • Mascaramento: Nenhum (rótulo aberto)

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Comparador Ativo: Deferasirox dispersible tablet (DFX-DT)
Iron chelation naïve participants received DFX-DT 20 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 5 to 10 mg/kg/day, with a maximum dose of 40 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose.
Medicamento: Deferasirox dispersible tablet
Deferasirox DT was provided as 125 mg, 250 mg and 500 mg dispersible tablets for oral use.
Outros nomes:
  • ICL670, DT (dispersible tablet)
Experimental: Deferasirox film-coated tablet (DFX-FCT)
Participants received DFX-FCT 14 mg/kg/day once daily orally from weeks 1 - 4. After week 4, the dose could be adjusted by +/- 3.5 to 7 mg/kg/day, with a maximum dose of 28 mg/kg/day. Iron chelation pre-treated participants were supposed to start on a dose that was equivalent to their pre-washout dose
Medicamento: Defearisox film-coated tablet
Deferasirox FCT was provided as 90 mg, 180 mg and 360 mg film-coated tablets for oral use.
Outros nomes:
  • ICL670, FCT (film-coated tablet)

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Overall Safety as Measured by Frequency of Adverse Events
Prazo: 28 weeks
The percentage of participants with adverse events, serious adverse events and deaths was assessed.
28 weeks
Overall Safety as Measured by Changes in Laboratory Values From Baseline
Prazo: baseline (BL), 30 weeks
The percentage of participants with post-baseline laboratory values meeting specified criteria for notable/extended range was assessed. The following laboratory parameters were measured: platelet count, absolute neutrophils, serum creatinine , creatinine clearance, urinary protein/urinary creatinine ratio, alanine aminotransferase (ALT) and aspartate aminotransferase (AST). Note that within data categories, creat = creatinine, cons = consecutive, ULN = upper limit of normal and urin = urinary.
baseline (BL), 30 weeks

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Frequency of Selected Gastro-intestinal (GI) Adverse Events
Prazo: 28 weeks
The percentage of participants with any GI adverse event, diarrhea, constipation, nausea, vomiting, abdominal pain was assessed.
28 weeks
Mean Domain Scores of the Modified Satisfaction With Iron Chelation Therapy (Modified SICT)
Prazo: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
The modified SICT consisted of 13 items that represent 3 domains: adherence, satisfaction and concerns. The adherence domain consisted of 7 items, 6 which were measured using a 5-point response scale and was calculated by summing the 6 items. The score range from 6 to 30 and higher scores indicated worse adherence. The satisfaction domain consisted of 3 items, 2 which were measured using a 5-point response scale and was calculated by summing the 2 items. The score range from 2 to 10 and higher scores indicated worse satisfaction. The concerns domain consisted of 3 items to address any concerns or worries with his/her medication. All 3 items were measured on a 5-point response scale and were calculated by summing the 3 items. The score range from 3 to 15 and higher scores indicated fewer concerns. For all three domains, the meaningful difference between two treatment arms was determined to be 1 point.
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Palatability Questionnaire Score
Prazo: weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
The palatability questionnaire consisted of 4 items. The first item measured the taste and aftertaste of the medication and were scored a on a 5-point response scale. The second item offered an additional response option of "no aftertaste". The last 2 items referred to whether the medication was taken, i.e. swallowed or vomited, and how the participant perceived the amount of medication to be taken. The palatability summary score was calculated using a scoring matrix from items 1, 3 and 4 scores and the score ranges from 0 - 11. Higher scores indicated the best palatability. A meaningful difference between two treatment arms was determined to be 1 point.
weeks 2, 3, 13 and 24 (end of treatment or within 7 days of last dose)
Weekly Average of Daily Scores of the Gastrointestinal (GI) Symptom Diary
Prazo: weeks -1, 4, 8, 12, 16, 20, 24
The GI symptom diary consisted of 6 items, five which were scored using a 0 - 10 rating scale with item appropriate anchors to rate the symptom, for example, Pain in your belly: 0 = no pain and 10 = worst pain. The GI diary summary score was created using the 10 point response scale for the 5 items. The GI symptom daily diary had a minimum score of 0 and a maximum score of 50. The weekly average score for the 7 days was calculated for each individual item and the GI summary score was created from these weekly averages. Higher scores indicated worse symptoms. A meaningful difference between two treatment arms was determined to be 0.3 point.
weeks -1, 4, 8, 12, 16, 20, 24
Number of Participants With Weekly Average Compliance of Medication Consumption
Prazo: Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
A compliance questionnaire assessed whether the medication was taken. Weekly average compliance was calculated when there were at least four non-missing daily responses.
Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24
Weekly Dose Violation Rate
Prazo: weeks 1, 4, 8, 12, 16, 20, 24
The dose violation is defined as a dose either missed completely or not taken in accordance with the timing instruction (no later than 12:00 pm. The rate was calculated as [number of dose violations/drug exposure (days)] x 100.
weeks 1, 4, 8, 12, 16, 20, 24
Area Under the Plasma Concentration-time Curve From Time Zero to the Last Quantifiable Concentration (AUClast)
Prazo: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess AUClast.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Observed Maximum Plasma Concentration Following Drug Administration (Cmax)
Prazo: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess Cmax.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Time to Reach the Maximum Plasma Concentration After Drug Administration (Tmax)
Prazo: week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Blood samples were collected to assess Tmax.
week 1, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose; week 3, day 1: pre-dose (0 hour) and 1, 2, 3, 4, 8 and 24 hours post dose
Dererasirox Plasma Concentration
Prazo: Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose
Blood samples were collected to assess deferasirox concentration. Dose-adjusted calculations are presented: (concentration/actual dose)*20 for participants on DFX-DT and (concentration/actual dose)*14 for participants on DFX-FCT.
Week 3, day 1, pre-dose (0 hour (h)) and 2 h post-dose; week 13, day 1, pre-dose (0 hour (h)) and 2 h post-dose; and week 21, day 1, pre-dose (0 hour (h)) and 2 h post-dose

Colaboradores e Investigadores

É aqui que você encontrará pessoas e organizações envolvidas com este estudo.

Patrocinador

Novartis Pharmaceuticals

Publicações e links úteis

A pessoa responsável por inserir informações sobre o estudo fornece voluntariamente essas publicações. Estes podem ser sobre qualquer coisa relacionada ao estudo.

Publicações Gerais

Datas de registro do estudo

Essas datas acompanham o progresso do registro do estudo e os envios de resumo dos resultados para ClinicalTrials.gov. Os registros do estudo e os resultados relatados são revisados ​​pela National Library of Medicine (NLM) para garantir que atendam aos padrões específicos de controle de qualidade antes de serem publicados no site público.

Datas Principais do Estudo

Início do estudo (Real)

8 de julho de 2014

Conclusão Primária (Real)

24 de fevereiro de 2016

Conclusão do estudo (Real)

24 de fevereiro de 2016

Datas de inscrição no estudo

Enviado pela primeira vez

26 de abril de 2014

Enviado pela primeira vez que atendeu aos critérios de CQ

26 de abril de 2014

Primeira postagem (Estimativa)

29 de abril de 2014

Atualizações de registro de estudo

Última Atualização Postada (Real)

25 de julho de 2017

Última atualização enviada que atendeu aos critérios de controle de qualidade

22 de julho de 2017

Última verificação

1 de julho de 2017

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • CICL670F2201

Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .

Ensaios clínicos em Deferasirox dispersible tablet

Pesquisar ensaios semelhantes

Patrocinadores e Colaboradores

Condições médicas

Intervenções de drogas

CROs by country

CROs in Switzerland

Condições

Doenças Raras

Intervenções de drogas

Suplementos Alimentares

Patrocinador / Colaboradores

Localizações

Se inscrever