Clinical trial NCT06693791: Romiplostim N01 for Platelet Recovery in Adult Hematologic Malignancies Patients Undergoing UCBT
Official title: Efficacy and Safety of a Long-acting Thrombopoietin Receptor Agonist for Platelet Recovery in Adult Hematologic Malignancies Patients Undergoing Umbilical Cord Blood Transplantation
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Platelet Recovery After Umbilical Cord Blood Transplantation
Lead sponsor: Anhui Provincial Hospital
Sponsor class: OTHER_GOV
First posted: 2024-11-18
Enrollment: 34
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To evaluate the efficacy and safety of Romiplostim N01 for platelet recovery after umbilical cord blood transplantation (UCBT) in patients with hematological malignancies.

Detailed description: Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) is an important means of curing a wide range of hematologic diseases, and good hematopoietic and immune reconstitution after transplantation is one of the most important factors determining the success of the transplantation.Persistent thrombocytopenia after Allo-HSCT is one of the common post-transplantation complications.This is an investigator-initiated, single-center, single-arm, Phase II clinical trial to evaluate the efficacy and safety of Romiplostim N01 for platelet recovery after UCBT in patients with hematologic tumors. The study was conducted in patients ≥18 years of age with a diagnosis of hematological neoplasia who underwent UCBT. Subjects will be screened and receive roprostin N01 from +1 to +28 days after UCBT after signing an informed consent form, and the study will focus on the cumulative PLT implantation rate at 28 days after transplantation as the primary efficacy measure. The study is planned to enroll 34 patients.

Eligibility criteria: Inclusion Criteria:

* Age ≥12 years old, male or female;
* Patients diagnosed with hematological malignancies and undergoing UCBT;
* ECOG score ≤2;
* Voluntary participation in this clinical trial, patients fully understand the trial content and sign the informed consent

Exclusion Criteria:

* Pregnant or lactating women；
* Known allergy to Romiplostim N01;
* A history of severe thrombotic events or known risk factors for thrombosis or active thromboembolism requiring anticoagulation;
* A history of platelet dysfunction or bleeding prone disease or severe bleeding (requiring more than 2 units of red blood cell infusion or a hematocrit drop of ≥10%) within 7 days prior to screening;
* Chronic active hepatitis B and C;
* Repeat or multiple transplantation or multiple organ transplantation;
* HIV positive, EBV-DNA positive, CMV-DNA positive;
* Have severe infection or complicated with serious heart, liver, lung, kidney, neurological or metabolic diseases;
* A serious adverse event (Level 4 or 5) as defined by the Standard General Terminology for Adverse Events Version 4.0 occurred during the pre-treatment period;
* Participants participate in another clinical study with any exploratory drug or device within 30 days prior to baseline visit; Observational studies are allowed;
* Subjects with cognitive impairment or uncontrolled mental illness;
* Subjects and/or authorized family members to refuse treatment with Romiplostim N01;
* Those who are judged by the researchers as not suitable for inclusion (such as accompanying medical history, which may affect the safety of the subjects or it is estimated that the treatment cannot be adhered to due to financial problems).

Primary outcomes:
measure: Time of platelet engraftment
timeFrame: 28 days
measure: The cumulative incidence of platelet engraftment at 28 days after transplantation
timeFrame: 28 days
Secondary outcomes:
measure: The cumulative incidence of platelet recovery rate at 28 days after transplantation
timeFrame: 28 days
measure: Time of platelet recovery
timeFrame: 28 days
measure: Total platelet transfusion during 4 weeks of treatment
timeFrame: 28 days
measure: Cumulative implantation rate of the granulocyte lineage at 28 days post-transplantation
timeFrame: 28 days
measure: Megakaryocyte levels in bone marrow smears 4 weeks after treatment
timeFrame: 4 weeks
Other outcomes:
Locations:
city: Hefei
country: China
facility: The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)
state: Anhui
Study officials:
References: