Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease

NCT05823376 | Recruiting

• 1 other identifier: AVA&MDS-001
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the efficacy and safety of Avatrombopag for platelet recovery after unrelated cord blood transplantation (UCBT) in patients with bone marrow failure disease

Conditions

Platelet Recovery After Umbilical Cord Blood Transplantation

Keywords

Avatrobopag

Outcome Measures

Primary Outcomes (2)

• Time of platelet engraftment

  Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10\^9/L.

  28 days

• The cumulative incidence of platelet engraftment at 28 days after transplantation

  Platelet engraftment is defined as independence from platelet transfusion for at least 7 days with a platelet count of more than ≥20 × 10\^9/L.

  28 days

Secondary Outcomes (7)

• The cumulative incidence of platelet recovery rate at 28 days after transplantation

  28 days

• Total platelet transfusion during 4 weeks of treatment

  28 days

• neutrophil reconstitution

  28 days

• erythroid reconstitution

  28 days

• Megakaryocyte levels in bone marrow smears 4 weeks after treatment

  4 weeks

Study Arms (1)

Avatrombopag group

The duration of the study is 4 weeks, from +1 day to +28 days after UCBT, patients will be given Avatrombopag with a recommended dose of 40mg per day. After 28 days of administration, the patients may continue, change or discontinue medication based on the patients' platelet status.

Drug: Avatrombopag

Interventions

Avatrombopag DRUG

Indications for drug reduction and withdrawal: excluding blood transfusion factors, the reduction will be 20mg/d when PLT≥50×10\^9/L; Drug administration will be stopped when PLT≥100×10\^9/L.

Avatrombopag group

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients diagnosed with bone marrow failure and receiving umbilical cord blood transplantation

You may qualify if:

• Age ≥12 years old, male or female;
• Patients diagnosed with bone marrow failure disease including aplastic anemia (AA), Fanconi anemia (FA), paroxysmal nocturnal hemoglobinuria (PNH) and undergoing UCBT;
• ECOG score ≤2;
• Voluntary participation in this clinical trial, patients fully understand the trial content and sign the informed consent；

You may not qualify if:

• Pregnant or lactating women；
• Known allergy to Avatrombopag;
• 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 Avatrombopag;

Sponsors & Collaborators

• Anhui Provincial Hospital: lead

Study Sites (1)

The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)

Hefei, Anhui, 230036, China

RECRUITING

MeSH Terms

Interventions

avatrombopag

Study Officials

• Xiaoyu Zhu, ph.D

  The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital)

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaoyu Zhu, ph.D

CONTACT

15255456091; xiaoyuz@ustc.edu.cn

Aijie Huang, M.D

CONTACT

15221056490; huangaijie@mail.ustc.edu.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 28 Days
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Chief physician

Study Record Dates

• First Submitted: March 24, 2023
• First Posted: April 21, 2023
• Study Start: May 4, 2023
• Primary Completion: January 31, 2025
• Study Completion: February 28, 2025
• Last Updated: December 31, 2024

Record last verified: 2024-12

Locations

CN (1)