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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.

Přehled studie

Postavení

Zatím nenabíráme

Podmínky

Intervence / Léčba

Typ studie

Intervenční

Zápis (Odhadovaný)

87

Fáze

  • Fáze 2

Kontakty a umístění

Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.

Studijní kontakt

Studijní místa

    • Henan
      • Luoyang, Henan, Čína, 471023
        • The First Affiliated Hospital of Henan University of Science and Technology
        • Kontakt:

Kritéria účasti

Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.

Kritéria způsobilosti

Věk způsobilý ke studiu

18 let a starší (Dospělý, Starší dospělý)

Přijímá zdravé dobrovolníky

Ne

Pohlaví způsobilá ke studiu

Všechno

Popis

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.

Studijní plán

Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.

Jak je studie koncipována?

Detaily designu

  • Primární účel: Léčba
  • Přidělení: Randomizované
  • Intervenční model: Paralelní přiřazení
  • Maskování: Žádné (otevřený štítek)

Zbraně a zásahy

Skupina účastníků / Arm
Intervence / Léčba
Experimentální: 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.

Žádný zásah: Control Group

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

Co je měření studie?

Primární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Platelet engraftment time
Časové okno: 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

Sekundární výstupní opatření

Měření výsledku
Popis opatření
Časové okno
Neutrophil engraftment time
Časové okno: 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)
Časové okno: 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)
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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
Časové okno: 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)
Časové okno: 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

Spolupracovníci a vyšetřovatelé

Zde najdete lidi a organizace zapojené do této studie.

Sponzor

Spolupracovníci

Termíny studijních záznamů

Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.

Hlavní termíny studia

Začátek studia (Odhadovaný)

1. listopadu 2026

Primární dokončení (Odhadovaný)

1. června 2028

Dokončení studie (Odhadovaný)

31. prosince 2028

Termíny zápisu do studia

První předloženo

11. září 2026

První předloženo, které splnilo kritéria kontroly kvality

17. září 2026

První zveřejněno (Aktuální)

21. září 2026

Aktualizace studijních záznamů

Poslední zveřejněná aktualizace (Aktuální)

21. září 2026

Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality

17. září 2026

Naposledy ověřeno

1. září 2026

Více informací

Termíny související s touto studií

Další identifikační čísla studie

  • HN-Blood-HSCT-107

Plán pro data jednotlivých účastníků (IPD)

Plánujete sdílet data jednotlivých účastníků (IPD)?

NE

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Ne

Studuje produkt zařízení regulovaný americkým úřadem FDA

Ne

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