Avatrombopag Usage in NSAA

NCT04728789 | Unknown Phase 2

• 1 other identifier: Avatrombopag-1
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

This is a multicenter, open-label, prospective one arm study to explore the efficacy and safety of Avatrombopag in non-severe aplastic anemia. Patients meeting the inclusion and exclusion criteria would be recruited. Treatment of Avatrombopag would be started with 20mg/day. The dosage would be increased by 20 mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The dosage could range from 20mg/week to 60mg/day. All patients would receive treatment for at least 6 months except that the platelet \<20×10e9/L at the dosage of 60mg/day for 4 weeks or the platelet ≥200×10e9/L at the dosage of 20mg/week for 2 weeks. The hematological response rate and safety will be recorded and compared at every month after starting the study treatment. The patients would be followed up for at least 6 months.

Conditions

Aplastic Anemia; Drug Effect

Outcome Measures

Primary Outcomes (1)

• ORR at 6 Months

  Overall Response Rate (ORR) Defined as the Number of Participants Who Met the Criteria of Either Complete Response (CR) or Partial Response (PR) at 6 months

  6 months

Secondary Outcomes (5)

• ORR at 3 Months

  3 months

• Percentage of patients with clonal evolution at 6 months

  6 months

• percentage of side effects at 6 months

  6 months

• Total volume of Platelet Transfusions

  1 month

• Total volume of Red Blood Cells Transfusions

  1 month

Study Arms (1)

Avatrombopag treatment group

EXPERIMENTAL

Avatrombopag would be started with 20mg/day. The dosage would be increased by 20 mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L. The dosage could range from 20mg/week to 60mg/day.

Drug: Avatrombopag 20 MG

Interventions

Avatrombopag 20 MG DRUG

Avatrombopag 20mg/week to 60mg/day, starting at 20mg/day and increased by 20 mg/day every 2 weeks if the platelet count remains less than 20×10e9/L and reduced if the platelet count reaches over than 150×10e9/L.

Avatrombopag treatment group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients diagnosed to be non-severe aplastic anemia
• Patients have at least one of the followings: ①absolute neutrophil count \< 1.5×109/L, ②platelet count \< 30×109/L, ③ hemoglobin level \< 100g/L
• Patients have no response or relapsed following at least one treatment course in a period time of \> 6 months of immunosuppression containing CsA or CsA+anti-thymocyte globulin (ATG);
• Eastern Cooperative Oncology Group (ECOG) performance status 0-2;
• Patients able to understand and comply with protocol requirements and instructions and have signed and dated informed consent.

You may not qualify if:

• Congenital aplastic anemia;
• Presence of chromosomal aberration;
• Evidence of a clonal hematologic bone marrow disorder (MDS, AML) on cytogenetics;
• Presence with PNH clone ≥50%;
• Patients received HSCT before;
• Uncontrolled infection or bleeding with standard treatment;
• Allergic to Avatrombopag or accessories;
• HIV, HCV or HBV active infection or liver cirrhosis or portal hypertension;
• Patient with QTcF (Fridericia's QT correction formula) at screening \<450 msec, or\<480 msec with bundle branch block, as determined via the mean of a triplicate ECG and assessed at site, unstable angina pectoris, uncontrolled hypertension(\>180/100mmHg)，pulmonary artery hypertension;
• Have any concomitant malignancies within 5 years expect for local basal cell carcinoma of the skin;
• Past history of thromboembolic event, heart attack or stroke (including anti-phospholipid antibody syndrome) and current use of anticoagulants;
• Pregnant or nursing (lactating) woman;
• Have attended other clinical trials within 3 months;

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, 100730, China

Location

Related Publications (1)

• Wan Z, Chen M, Han B. Avatrombopag, a promising novel thrombopoietin receptor agonist for refractory/relapsed/intolerant non-severe aplastic anemia: a phase 2 single-arm clinical trial. Ann Med. 2023 Dec;55(1):2224044. doi: 10.1080/07853890.2023.2224044.

  PMID: 37318085 DERIVED

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

avatrombopag

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: December 1, 2020
• First Posted: January 28, 2021
• Study Start: February 1, 2021
• Primary Completion: December 31, 2022
• Study Completion: June 30, 2023
• Last Updated: January 28, 2021

Record last verified: 2021-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Time Frame: 10 years
• Access Criteria: email request

Locations

CN (1)