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Evaluating the Safety and Efficacy of Romiplostim (AMG 531) in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)

20 de dezembro de 2019 atualizado por: Amgen

A Dose-finding Study Evaluating the Safety and Efficacy of AMG 531 in Thrombocytopenic Subjects With Immune Thrombocytopenic Purpura (ITP)

The primary objective of this study is to evaluate the safety and tolerability of romiplostim in thrombocytopenic patients with ITP.

Visão geral do estudo

Status

Concluído

Condições

Intervenção / Tratamento

Tipo de estudo

Intervencional

Inscrição (Real)

45

Estágio

  • Fase 2

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

18 anos a 65 anos (Adulto, Adulto mais velho)

Aceita Voluntários Saudáveis

Não

Gêneros Elegíveis para o Estudo

Tudo

Descrição

Inclusion Criteria:

  • Diagnosis of ITP according to American Society of Hematology (ASH) guidelines at least 3 months before enrollment
  • Have completed at least 1 prior treatment for ITP

  • Two (including day -2) of the 3 platelet counts taken during the screening and pre-treatment periods must have fulfilled the following:

    • less than 30 x 10^9/L for those subjects not receiving any ITP therapy,
    • less than 50 x 10^9/L for those subjects receiving any ITP therapy
  • Eastern Cooperative Oncology Group performance status of 0 to 2
  • Serum creatinine concentration ≤ 2 mg/dL (≤ 176.8 µmol/L)
  • Adequate liver function, as evidenced by a serum bilirubin ≤ 1.5 times the laboratory normal range
  • Hemoglobin greater than 10.0 g/dL
  • Written informed consent

Exclusion Criteria:

  • Considered a substantial risk for adverse outcomes because of a clinically important trend (as determined by the investigator) detected in the platelet counts during the screening period
  • Any known history of bone marrow stem cell disorder
  • Any active malignancy. If prior history of cancer other than basal cell carcinoma or cervical carcinoma in situ, no treatment or active disease within 5 years before randomization
  • Documented diagnosis of arterial thrombosis (ie, stroke, transient ischemic attack, or myocardial infarction) in the previous year; history of venous thrombosis (ie, deep vein thrombosis, pulmonary embolism) and receiving anticoagulation therapy
  • Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure [New York Heart Association (NYHA) greater than class II], uncontrolled hypertension [diastolic greater than 100 mmHg] or cardiac arrhythmia)
  • Have 3 or more of the following predisposing factors for thromboembolic events: diabetes; smoker using oral contraceptives; hypercholesteremia (> 240 mg/dL); treatment for hypertension
  • Known positive test for human immunodeficiency virus (HIV) infection or hepatitis C virus
  • Received any treatment for ITP (except for a constant dose schedule of corticosteroids) within 4 weeks before the screening visit
  • Received intravenous (IV) immunoglobulin (Ig) or WinRho within 2 weeks before the screening visit
  • Received hematopoietic growth factors, including interleukin (IL)-11 (Neumega®) within 4 weeks before the screening visit
  • Past or present participation in any study evaluating polyethylene glycol recombinant human magakaryopoiesis differentiating factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), or related platelet product
  • Received any alkylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
  • Received any monoclonal antibody (eg, rituximab) within 16 weeks before the screening visit or anticipated use during the time of the proposed study
  • Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA approved for any indication before the screening period
  • Less than 2 months since major surgery (including laparoscopic splenectomy)
  • Pregnant or breast feeding
  • Subjects of reproductive potential who are not using adequate contraceptive precautions, in the judgment of the investigator

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 sequencial
  • Mascaramento: Dobro

Armas e Intervenções

Grupo de Participantes / Braço
Intervenção / Tratamento
Experimental: Part A: Romiplostim 0.2 µg/kg
Participants received 0.2 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE
Experimental: Part A: Romiplostim 0.5 µg/kg
Participants received 0.5 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE
Experimental: Part A: Romiplostim 1.0 µg/kg
Participants received 1.0 µg/kg romiplostim subcutaneously on day 1 and on day 15 or 22 depending on platelet counts.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE
Experimental: Part A: Romiplostim 3 µg/kg
Participants received 3.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE
Experimental: Part A: Romiplostim 6 µg/kg
Participants received 6.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE
Experimental: Part A: Romiplostim 10 µg/kg
Participants received 10.0 µg/kg romiplostim subcutaneously on day 1 and day 15 or 22, depending on platelet counts.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE
Comparador de Placebo: Part B: Placebo
Participants received placebo subcutaneously once a week for 6 weeks.
Medicamento: Placebo
Administrado por injeção subcutânea
Experimental: Part B: Romiplostim 1.0 µg/kg
Participants received 1.0 µg/kg subcutaneously once a week for 6 weeks.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE
Experimental: Part B: Romiplostim 3.0 µg/kg
Participants received 3.0 µg/kg subcutaneously once a week for 6 weeks.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE
Experimental: Part B: Romiplostim 6.0 µg/kg
Participants received 6.0 µg/kg subcutaneously once a week for 6 weeks.
Medicamento: Romiplostim
Administered by subcutaneous injection
Outros nomes:
  • AMG 531
  • NPLATE

O que o estudo está medindo?

Medidas de resultados primários

Medida de resultado
Descrição da medida
Prazo
Number of Participants With Adverse Events
Prazo: From first dose of study drug through 8 weeks (Part A) or 6 weeks (Part B) after last dose of study drug; 78 days
From first dose of study drug through 8 weeks (Part A) or 6 weeks (Part B) after last dose of study drug; 78 days
Number of Participants With Anti-romiplostim or Anti-endogenous Thrombopoietin Neutralizing Antibodies
Prazo: Assessed on day 29 (Part A only), day 43 (Part B only), and day 78
The development of antibodies to romiplostim or to endogenous thrombopoietin (eTPO) was assessed using a neutralizing bioassay. Participants positive for neutralizing antibodies at any of the assessments during the study are reported.
Assessed on day 29 (Part A only), day 43 (Part B only), and day 78

Medidas de resultados secundários

Medida de resultado
Descrição da medida
Prazo
Number of Participants Who Achieved Targeted Therapeutic Platelet Level in Part A
Prazo: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)

Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and between 50 to 450 x 10⁹ cells/L.

Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L Over Baseline in Part A
Prazo: After first dose (day 1 to day 15 or 22), and after second dose (day 15 or 22 to day 78)
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
After first dose (day 1 to day 15 or 22), and after second dose (day 15 or 22 to day 78)
Number of Participants With a Peak Platelet Count ≥ 100 x 10⁹ Cells/L in Part A
Prazo: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Number of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part A
Prazo: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders.
After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Peak Platelet Count After Each Dose in Part A
Prazo: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Platelet count data after the use of rescue medication were not included.
After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Change From Baseline in Peak Platelet Count After Each Dose in Part A
Prazo: Baseline and after first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Platelet count data after the use of rescue medication were not included.
Baseline and after first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Time to Peak Platelet Count After Each Dose in Part A
Prazo: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Platelet count data after the use of rescue medication were not included.
After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Duration Within the Targeted Therapeutic Platelet Range In Part A
Prazo: After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)

Targeted therapeutic platelet level was defined as a platelet count that was double the baseline level and ≥ 50 and ≤ 450 × 10⁹ cells/L.

Platelet count data after the use of rescue medication were not included.
After first dose (day 1 to day 15 or 22) and after second dose (day 15 or 22 to day 78)
Percentage of Participants Who Achieved Targeted Therapeutic Platelet Level In Part B
Prazo: Day 1 to day 78

Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and within the range of greater than or equal to 50 x 10⁹ cells/L and less than or equal to 450 x 10⁹ cells/L.

Platelet count data after use of rescue medication were not included in the analysis.
Day 1 to day 78
Percentage of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L Over Baseline in Part B
Prazo: Day 1 to day 78
Platelet count data after administration of rescue medication were not included in the analysis. Participants with no platelet count data were considered non-responders.
Day 1 to day 78
Percentage of Participants With a Peak Platelet Count of ≥ 100 x 10⁹ Cells/L in Part B
Prazo: Day 1 to day 78
Platelet count data after administration of rescue medication were not included in the analysis. Participants with no platelet count data were considered non-responders.
Day 1 to day 78
Percentage of Participants With a Peak Platelet Count of > 450 x 10⁹ Cells/L in Part B
Prazo: Day 1 to day 78
Platelet count data after administration of rescue medication were not included in the analysis. Participants with no platelet count data were considered non-responders.
Day 1 to day 78
Percentage of Participants With a Peak Platelet Count of > 500 x 10⁹ Cells/L in Part B
Prazo: Day 1 to day 78
Platelet count data after administration of rescue medication were not included in the analysis. Participants with no platelet count data were considered non-responders.
Day 1 to day 78
Peak Platelet Count in Part B
Prazo: Day 1 to day 78
Platelet count data after administration of rescue medication were not included in the analysis.
Day 1 to day 78
Change From Baseline in Peak Platelet Count in Part B
Prazo: Baseline and day 1 to day 78
Platelet count data after administration of rescue medication were not included in the analysis.
Baseline and day 1 to day 78
Time to Peak Platelet Count in Part B
Prazo: Day 1 to day 78
Platelet count data after administration of rescue medication were not included in the analysis. Time to peak platelet count was analyzed using the Kaplan-Meier method.
Day 1 to day 78
Duration Within the Targeted Therapeutic Platelet Range in Part B
Prazo: Day 1 to day 78

Targeted therapeutic platelet level was defined as a doubling of baseline platelet counts and within the range of greater than or equal to 50 × 10⁹ cells/L and less than or equal to 450 × 10⁹ cells/L.

Platelet count data after administration of rescue medication were not included in the analysis.
Day 1 to day 78

Colaboradores e Investigadores

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

Patrocinador

Amgen

Publicações e links úteis

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Publicações Gerais

Links úteis

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)

1 de julho de 2002

Conclusão Primária (Real)

17 de junho de 2004

Conclusão do estudo (Real)

17 de junho de 2004

Datas de inscrição no estudo

Enviado pela primeira vez

20 de maio de 2005

Enviado pela primeira vez que atendeu aos critérios de CQ

20 de maio de 2005

Primeira postagem (Estimativa)

23 de maio de 2005

Atualizações de registro de estudo

Última Atualização Postada (Real)

10 de janeiro de 2020

Última atualização enviada que atendeu aos critérios de controle de qualidade

20 de dezembro de 2019

Última verificação

1 de dezembro de 2019

Mais Informações

Termos relacionados a este estudo

Palavras-chave

Termos MeSH relevantes adicionais

Outros números de identificação do estudo

  • 20000137

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