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.
Panoramica dello studio
Stato
Stato
Condizioni
Condizioni
Intervento / Trattamento
Intervento / Trattamento
Tipo di studio
Tipo di studio
Iscrizione (Stimato)
Iscrizione
Fase
Fase
- Fase 2
Contatti e Sedi
Contatto studio
Contatto studio
- Nome: Haiping Yang
- Numero di telefono: 69823566
- Email: 13938820189@163.com
Luoghi di studio
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Henan
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Luoyang, Henan, Cina, 471023
- The First Affiliated Hospital of Henan University of Science and Technology
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Contatto:
- Haiping Yang
- Numero di telefono: 69823566
- Email: 13938820189@163.com
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Criteri di partecipazione
Criteri di ammissibilità
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
Inclusion Criteria:
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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:
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Serum bilirubin ≤ 2 mg/dL, unless due to benign congenital hyperbilirubinemia;
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AST, ALT and alkaline phosphatase < 3 × upper limit of normal (ULN);
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Creatinine clearance ≥ 40 mL/min (calculated by the Cockcroft-Gault formula);
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Left ventricular ejection fraction (LVEF) ≥ 45% by MUGA scan or resting echocardiogram;
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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:
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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.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Nessuno (etichetta aperta)
Numero di armi
Armi e interventi
Gruppo di partecipanti / ArmGruppo di partecipanti / Arm |
Intervento / TrattamentoIntervento / Trattamento |
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Sperimentale: 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. |
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Nessun intervento: Control Group
Participants in the control group receive identical standard allo-HSCT supportive care, with the exception of Romiplostim N01 for Injection |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Platelet engraftment time
Lasso di tempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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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
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Misure di risultato secondarie
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Neutrophil engraftment time
Lasso di tempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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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
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Median time to achieve complete remission (CR)
Lasso di tempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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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
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Incidence of primary poor graft function (PGF)
Lasso di tempo: Within 28 days after allo-HSCT
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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
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Incidence of secondary platelet recovery failure
Lasso di tempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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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
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Incidence of graft-versus-host disease
Lasso di tempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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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
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Incidence of primary graft failure
Lasso di tempo: At Day 30 or Day 42 after cell infusion
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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
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Incidence of secondary graft failure
Lasso di tempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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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
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Comparison of treatment-related costs between Romiplostim N01 group and control group
Lasso di tempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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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
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Platelet transfusion requirements during engraftment
Lasso di tempo: From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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Total number of platelet transfusions administered during the platelet engraftment period. |
From Day +1 after allogeneic-HSCT up to 1 year post-transplant
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Incidence and severity of adverse events (AEs) and adverse drug reactions (ADRs)
Lasso di tempo: From treatment initiation to 28 days after treatment completion
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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
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Collaboratori e investigatori
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Collaboratori
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Inizio studio (Stimato)
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Date di iscrizione allo studio
Primo inviato
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo inviato che soddisfa i criteri di controllo qualità
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Aggiornamenti dei record di studio
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Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
Altri numeri di identificazione dello studio
- HN-Blood-HSCT-107
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