- ICH GCP
- Registro de ensaios clínicos dos EUA
- Ensaio Clínico NCT07612319
Romiplostim for Oral TPO-RA Resistant ITP
A Prospective Cohort Study of Romiplostim in Immune Thrombocytopenia Patients Resistant to Oral Thrombopoietin Receptor Agonists
This study is a prospective, multicenter, open-label, single-arm cohort study to evaluate the effectiveness and safety of romiplostim in patients with immune thrombocytopenia (ITP) who have not responded to oral thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag, hetrombopag, or avatrombopag.
ITP is a blood disorder in which the immune system attacks and destroys the body's own platelets, leading to low platelet counts and an increased risk of bleeding. While oral TPO-RAs are effective for many patients, approximately 20-30% of patients do not respond adequately or lose response over time. For these patients, alternative treatments are urgently needed.
Participants in this study will discontinue their current oral TPO-RA and receive romiplostim as a weekly subcutaneous injection. The starting dose is 3 µg/kg, and the dose may be adjusted weekly based on platelet counts (maximum 10 µg/kg). The total treatment and follow-up period is 24 weeks.
The primary outcome measure is the durable platelet response rate at week 24, defined as maintaining platelet counts at ≥50×10⁹/L for at least two scheduled visits during weeks 22-24 without rescue therapy. Secondary outcomes include the sustained response rate at week 12, complete response rate at week 24, time to first response, improvement in bleeding events, and quality of life assessment.
Safety outcomes include monitoring for thromboembolic events, bone marrow fibrosis, hepatotoxicity, injection site reactions, and other adverse events according to CTCAE v5.0.
Approximately 60 participants will be enrolled across multiple centers in China. This study aims to provide evidence for romiplostim as a treatment option for ITP patients who have failed oral TPO-RAs.
Visão geral do estudo
Status
Intervenção / Tratamento
Tipo de estudo
Inscrição (Estimado)
Estágio
- Não aplicável
Contactos e Locais
Contato de estudo
- Nome: The First Affiliated Hospital of Soochow University
- Número de telefone: 86-0512-67781521
- E-mail: tianhong0718@163.com
Locais de estudo
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Jiangsu
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Suzhou, Jiangsu, China, 215000
- Recrutamento
- The First Affiliated Hospital of Soochow University
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Descrição
Inclusion Criteria:
Age ≥18 years Diagnosed with primary immune thrombocytopenia (ITP) according to current guidelines
Failed prior oral TPO-RA therapy (eltrombopag, hetrombopag, or avatrombopag), defined as:
Platelet count persistently <30×10⁹/L after ≥4 weeks of treatment at the maximum recommended or tolerated dose, OR Prior achieved response (platelet ≥50×10⁹/L) then lost response with platelet count <30×10⁹/L on two consecutive visits (≥1 week apart), OR Intolerance to oral TPO-RA leading to discontinuation (with documented reason) Baseline platelet count <30×10⁹/L (at least two measurements, ≥5 days apart) ECOG performance status 0-2 Willing and able to receive weekly subcutaneous injections and comply with study procedures For women of childbearing potential: negative pregnancy test at screening and agreement to use effective contraception during the study and for 3 months after the last dose Able to provide written informed consent
Exclusion Criteria:
Secondary ITP (e.g., associated with autoimmune diseases, lymphoproliferative disorders, drug-induced, or infections such as HIV/HCV/HBV) Known hypersensitivity to romiplostim or any of its excipients Prior treatment with romiplostim Received rituximab or other B-cell depleting therapy within 6 months prior to screening Received immunosuppressants (e.g., cyclosporine, mycophenolate mofetil, azathioprine, danazol) within 4 weeks prior to screening Splenectomy within 8 weeks prior to screening Use of corticosteroids (prednisone >10 mg/day or equivalent) or IVIG within 2 weeks prior to screening History of bone marrow fibrosis (MF grade ≥2) or known reticulin fibrosis History of arterial or venous thromboembolic event (e.g., myocardial infarction, stroke, deep vein thrombosis, pulmonary embolism) within 6 months prior to screening Known thrombophilic conditions (e.g., antiphospholipid syndrome, protein C/S deficiency, ATIII deficiency) Severe hepatic impairment (ALT or AST >3×ULN, or total bilirubin >1.5×ULN) Severe renal impairment (creatinine clearance <30 mL/min) New York Heart Association (NYHA) functional class III or IV heart failure Malignancy within 5 years prior to screening (except non-melanoma skin cancer, cervical carcinoma in situ, or ductal carcinoma in situ of the breast) Pregnancy or breastfeeding Participation in another interventional clinical trial within 30 days prior to screening Any other condition that, in the investigator's judgment, would make the participant unsuitable for the study
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: N / D
- Modelo Intervencional: Atribuição de grupo único
- Mascaramento: Nenhum (rótulo aberto)
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
|
Experimental: Romiplostim Treatment Arm
|
Romiplostim is administered as a subcutaneous injection once weekly for 24 weeks. The starting dose is 3 µg/kg. Platelet count is assessed weekly, and the dose is adjusted based on platelet count and bleeding symptoms as follows: If platelet count <50×10⁹/L: increase dose by 1 µg/kg (maximum dose: 10 µg/kg per week) If platelet count 50-200×10⁹/L: maintain the lowest dose that reduces bleeding risk If platelet count 200-400×10⁹/L: decrease dose by 1 µg/kg If platelet count >400×10⁹/L: withhold dosing. After platelet count decreases to <200×10⁹/L, resume at a dose 1 µg/kg lower than the dose before withholding. |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
|
Durable Platelet Response Rate at Week 24
Prazo: 24 weeks
|
Defined as the proportion of participants achieving platelet count ≥50×10⁹/L for at least two scheduled visits during weeks 22-24, without receiving rescue therapy (IVIG, high-dose corticosteroids, or platelet transfusion) during weeks 20-24.
|
24 weeks
|
Colaboradores e Investigadores
Patrocinador
Colaboradores
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Estimado)
Conclusão do estudo (Estimado)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
- Citopenia
- Processos Patológicos
- Doenças autoimunes
- Doenças do sistema imunológico
- Hemorragia
- Manifestações de pele
- Doenças Hematológicas
- Distúrbios da Coagulação Sanguínea
- Distúrbios hemorrágicos
- Distúrbios das plaquetas sanguíneas
- Microangiopatias Trombóticas
- Púrpura
- Trombocitopenia
- Condições Patológicas, Sinais e Sintomas
- Sinais e sintomas
- Doenças hemic e linfáticas
- Púrpura Trombocitopênica Idiopática
- Púrpura Trombocitopênica
- Romiplostim
Outros números de identificação do estudo
- 20251200
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
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