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Efficacy and Safety of Romiplostim N01 for Injection in Promoting Platelet Reconstitution After Allogeneic Hematopoietic Stem Cell Transplantation

Efficacy and Safety of Romiplostim N01 for Injection in Promoting Platelet Reconstitution After Allogeneic Hematopoietic Stem Cell Transplantation: A Prospective, Single-Center, Randomized Controlled Clinical Study

This is a prospective, single-center, randomized controlled, investigator-initiated clinical study. The purpose of this study is to evaluate the efficacy and safety of Romiplostim N01 for Injection in promoting platelet reconstitution after allogeneic hematopoietic stem cell transplantation (allo-HSCT).

Eligible participants will be randomly assigned in a 2:1 ratio to receive either Romiplostim N01 for Injection (experimental group) or the same treatment with the exception of Romiplostim N01 for Injection (control group).In the experimental group, Romiplostim N01 for Injection will be administered subcutaneously once weekly starting from Day 1 after allo-HSCT, with an initial dose of 3.0 μg/kg and dose titration based on platelet counts, up to a maximum of 10 μg/kg once weekly. Treatment will continue until platelet count exceeds 20×10⁹/L for 7 consecutive days without platelet transfusion, or until lack of efficacy after 4 weeks at 10 μg/kg, or at the investigator's discretion. Participants will enter a safety follow-up period for 28 days after treatment completion, with visits as frequently as weekly to collect adverse events, concomitant medications, and supportive care information.

The primary efficacy endpoint is the time to platelet engraftment, defined as the first day of platelet count ≥20×10⁹/L for 7 consecutive days without platelet transfusion.

Descripción general del estudio

Estado

Aún no reclutando

Condiciones

Intervención / Tratamiento

Tipo de estudio

Intervencionista

Inscripción (Estimado)

87

Fase

  • Fase 2

Contactos y Ubicaciones

Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.

Estudio Contacto

Ubicaciones de estudio

    • Henan
      • Luoyang, Henan, Porcelana, 471023
        • The First Affiliated Hospital of Henan University of Science and Technology
        • Contacto:

Criterios de participación

Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.

Criterio de elegibilidad

Edades elegibles para estudiar

18 años y mayores (Adulto, Adulto Mayor)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Descripción

Inclusion Criteria:

  • 1.Subjects must understand study procedures, be willing to comply with the study protocol, and provide written informed consent prior to any study-related procedures.

    2. Age ≥18 years, male or female. 3. Patients with hematological malignancies undergoing allogeneic hematopoietic stem-cell transplantation (allo-HSCT).

    4. Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1. 5. Adequate organ function defined as follows:

  • Serum bilirubin ≤ 2 mg/dL, unless due to benign congenital hyperbilirubinemia;

  • AST, ALT and alkaline phosphatase < 3 × upper limit of normal (ULN);

  • Creatinine clearance ≥ 40 mL/min (calculated by the Cockcroft-Gault formula);

  • Left ventricular ejection fraction (LVEF) ≥ 45% by MUGA scan or resting echocardiogram;

  • Pulmonary function: FEV1 and corrected DLCO ≥45% of predicted value. 6. Participants of reproductive potential must use reliable contraceptive methods throughout the study period (including male or female condoms, contraceptive foam, contraceptive gel, contraceptive diaphragm, contraceptive cream, contraceptive suppositories, abstinence, intrauterine device, etc.). Exceptions include female participants who have undergone hysterectomy, bilateral salpingectomy, bilateral tubal ligation, or are postmenopausal for more than 1 year; and male participants with bilateral vasectomy.

Exclusion Criteria:

  • 1. Subjects who have received romiplostim, eltrombopag, or other TPO-RA thrombopoietic agents within 1 month prior to enrollment.

    2. History of symptomatic or incidental venous thromboembolic events (e.g., deep vein thrombosis or pulmonary embolism), except patients who received and tolerated anticoagulation within the preceding 6 months, completed anticoagulation >6 months ago, or are receiving ongoing anticoagulation. Patients with central venous catheter-related venous thromboembolic events are excluded.

    3. Patients with a history of symptomatic arterial thrombotic events including myocardial infarction, ischemic cerebrovascular accident, or transient ischemic attack within the preceding 6 months.

    4. Patients with severe underlying diseases that may interfere with the conduct of the study, such as unstable angina, renal failure requiring hemodialysis, or active infection requiring intravenous antibiotic therapy.

    5. Abnormal hepatic or renal function: total bilirubin > 2 × upper limit of normal (ULN); AST or ALT > 1.5 × ULN; creatinine > 1.5 × ULN; estimated glomerular filtration rate < 30 mL/min/1.73 m².

    6. Patients with positive hepatitis C antibody plus elevated HCV-RNA; patients with positive hepatitis B surface antigen plus elevated HBV-DNA; patients with severe liver cirrhosis; patients with positive HIV antibody or positive syphilis antibody.

    7. Subjects with known hypersensitivity or intolerance to the active ingredient or excipients of injectable Romiplostim N01.

    8.Participation in any other interventional clinical study of investigational drugs or devices within 1 month prior to screening.

    9. Female subjects who are pregnant or breastfeeding. 10. Subjects who, in the investigator's judgment, are unable to comply with study procedures, or for whom study participation is otherwise deemed inappropriate by the investigator.

Plan de estudios

Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.

¿Cómo está diseñado el estudio?

Detalles de diseño

  • Propósito principal: Tratamiento
  • Asignación: Aleatorizado
  • Modelo Intervencionista: Asignación paralela
  • Enmascaramiento: Ninguno (etiqueta abierta)

Armas e Intervenciones

Grupo de participantes/brazo
Intervención / Tratamiento
Experimental: Romiplostim N01

Participants receive Romiplostim N01 for Injection subcutaneously once weekly starting from Day 1 after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The initial dose is 3.0 μg/kg, with dose titration based on platelet counts, up to a maximum of 10 μg/kg once weekly. Treatment continues until platelet count exceeds 20×10⁹/L for 7 consecutive days without platelet transfusion, or until lack of efficacy after 4 weeks at 10 μg/kg, or at the investigator's discretion.

Subcutaneous injection once weekly, initiated on Day +1 after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The starting dose is 3.0 μg/kg. The dose will be titrated according to peripheral platelet counts up to a maximum weekly dose of 10 μg/kg. Treatment will be discontinued when any of the following criteria are met: platelet count ≥20×10⁹/L sustained for 7 consecutive days without platelet transfusion; inadequate platelet response after 4 consecutive weeks at the maximum dose of 10 μg/kg; or discontinuation at the investigator's clinical discretion.

Sin intervención: Control Group

Participants in the control group receive identical standard allo-HSCT supportive care, with the exception of Romiplostim N01 for Injection

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Platelet engraftment time
Periodo de tiempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant

the first day of platelet count ≥20×10⁹/L sustained for 7 consecutive days in the absence of platelet transfusion

From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Medidas de resultado secundarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Neutrophil engraftment time
Periodo de tiempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant

the first day when absolute neutrophil count (ANC) ≥0.5×10⁹/L sustained for 3 consecutive days in the absence of G-CSF support

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Median time to achieve complete remission (CR)
Periodo de tiempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Defined as the median time to achieve complete remission (CR), where complete remission is defined as platelet count ≥50×10⁹/L sustained for 7 consecutive days without platelet transfusion.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of primary poor graft function (PGF)
Periodo de tiempo: Within 28 days after allo-HSCT

Defined as the incidence of primary poor graft function, a condition occurring within 28 days after transplantation, characterized by full donor chimerism, absence of severe GVHD or disease relapse, successful myeloid and erythroid engraftment (absolute neutrophil count >0.5×10⁹/L and hemoglobin >70 g/L), with sustained platelet count ≤20×10⁹/L.

Within 28 days after allo-HSCT
Incidence of secondary platelet recovery failure
Periodo de tiempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Incidence of secondary platelet recovery failure (defined as a decline in platelet count to <20,000/μL after achievement of initial platelet recovery, lasting for at least 7 days or requiring platelet transfusion within 7 days)

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of graft-versus-host disease
Periodo de tiempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Acute GVHD is graded according to the Glucksberg grading criteria; chronic GVHD is diagnosed and graded based on the National Institutes of Health (NIH) consensus criteria and Chinese expert consensus.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence of primary graft failure
Periodo de tiempo: At Day 30 or Day 42 after cell infusion

① For peripheral blood stem cells (PBS) or non-mobilized bone marrow cells: ANC < 0.5×10⁹/L accompanied by pancytopenia on Day 30 after infusion.

② For umbilical cord blood (UCB): ANC < 0.5×10⁹/L accompanied by pancytopenia on Day 42 after infusion.

At Day 30 or Day 42 after cell infusion
Incidence of secondary graft failure
Periodo de tiempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant

deterioration of hematopoietic function (involving hemoglobin and/or platelets and/or neutrophils) after achievement of hematopoietic reconstitution (including hemoglobin, platelets and/or neutrophils), requiring blood product transfusion or growth factor support.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Comparison of treatment-related costs between Romiplostim N01 group and control group
Periodo de tiempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Comparison of treatment-related costs between the Romiplostim N01 group and the control group.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Platelet transfusion requirements during engraftment
Periodo de tiempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant

Total number of platelet transfusions administered during the platelet engraftment period.

From Day +1 after allogeneic-HSCT up to 1 year post-transplant
Incidence and severity of adverse events (AEs) and adverse drug reactions (ADRs)
Periodo de tiempo: From treatment initiation to 28 days after treatment completion

Adverse events and adverse drug reactions will be recorded and graded according to the Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.

From treatment initiation to 28 days after treatment completion

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Colaboradores

Fechas de registro del estudio

Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados ​​por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.

Fechas importantes del estudio

Inicio del estudio (Estimado)

1 de noviembre de 2026

Finalización primaria (Estimado)

1 de junio de 2028

Finalización del estudio (Estimado)

31 de diciembre de 2028

Fechas de registro del estudio

Enviado por primera vez

11 de septiembre de 2026

Primero enviado que cumplió con los criterios de control de calidad

17 de septiembre de 2026

Publicado por primera vez (Actual)

21 de septiembre de 2026

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

21 de septiembre de 2026

Última actualización enviada que cumplió con los criterios de control de calidad

17 de septiembre de 2026

Última verificación

1 de septiembre de 2026

Más información

Términos relacionados con este estudio

Otros números de identificación del estudio

  • HN-Blood-HSCT-107

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

NO

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .