Efficacy and Safety of Avatrombopag Combined With IST for the Treatment of HAAA and SAA With Abnormal Liver Function

NCT05571332 | Unknown

• 1 other identifier: IIT2021008-EC-1(2)
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

This is a multicenter, single-arm clinical study. The objective was to evaluate the efficacy and safety of Avatrombopag combined with IST in very/sever aplastic anemia patients with abnormal liver function or HAAA patients treated for the first time. The design was: Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. Cyclosporine 3 mg/kg orally in two divided doses, with cyclosporine trough concentrations maintained at 200-250 ng/ml for 3 months to achieve maximum efficacy, and Avatrombopag, which was administered in the dose of 40 mg orally once daily for a total of 12 weeks. Thirty-nine patients are expected to be enrolled in this study. Evaluation endpoint: complete response rate at 12 weeks of treatment.

Conditions

Aplastic Anemia

Keywords

Avatrombopag; aplastic anemia; abnormal liver function; hepatitis

Outcome Measures

Primary Outcomes (2)

• CR rate at 12 weeks of treatment

  Percentage of the total number of patients receiving treatment who received a complete response at 12 weeks of treatment

  12 weeks of treatment

• ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading at 12 weeks of treatment

  Incidence of Treatment-Emergent AE by CTCAE

  12 weeks of treatment

Secondary Outcomes (1)

• OR rate at 12 weeks of treatment

  12 weeks of treatment

Study Arms (1)

Avatrombopag+CsA+ p-ATG

EXPERIMENTAL

Patients received p-ATG for 5 consecutive days (day 1-5), at a dose of 20 mg/kg/day. CSA is started at 3 mg/kg orally in two doses. Concentrations maintained at 200-250 ng/ml to achieve maximum efficacy and then tapered by 25 mg every 3 months; Avatrombopag: 40 mg orally once daily for a total of 12 weeks. A total of 39 patients were expected to be included.

Drug: Avatrombopag 20 MG Oral Tablet

Interventions

Avatrombopag 20 MG Oral Tablet DRUG

p-ATG and CsA in combination with Avatrombopag to treat

Also known as: porcine ATG, Cyclosporine(CsA)

Avatrombopag+CsA+ p-ATG

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• patients with V/SAA with a definite diagnosis.
• age between 18-70 years, male or female.
• Subjects must complete all screening assessments as outlined in the trial protocol.
• Able to swallow or administer the drug orally.
• No prior application of TPO receptor agonists (including Thrombopoietin, Eltrombopag, Hetrombopag, etc.) or application of TPO receptor agonists for treatment with ≤ 5 total doses and ≤ 7 days of TPO receptor agonist drugs such as Eltrombopag, Hetrombopag, etc.
• Diagnosis as HAAA or abnormal liver function. ALT and AST more than 1.5 times of upper limit.
• Informed consent must be signed prior to the start of all specific study procedures, in consideration of the patient's condition, or by a member of the patient's immediate family if the patient's signature is not conducive to the treatment of the condition.

You may not qualify if:

• Known diagnosis of congenital hematopoietic failure disorders (e.g. Fanconi anemia) and other causes of allogeneic cytopenias and bone marrow hypoproliferative disorders (e.g. hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated allogeneic cytopenias, etc.);
• Patients with uncontrolled bleeding and/or infection despite standard treatment.
• Patients with previous history of hematopoietic stem cell transplantation; previous history of thrombosis.
• Patients with concurrent malignancy or potential cancer on immunosuppressive therapy.
• Those who are considered unsuitable for enrollment by the investigator.

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

Related Publications (2)

• Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica. 2008 Apr;93(4):489-92. doi: 10.3324/haematol.12855. No abstract available.

  PMID: 18379007 RESULT

• Scheinberg P. Activity of eltrombopag in severe aplastic anemia. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):450-456. doi: 10.1182/asheducation-2018.1.450.

  PMID: 30504345 RESULT

MeSH Terms

Conditions

Anemia, Aplastic; Liver Diseases; Hepatitis

Interventions

avatrombopag; Tablets

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Study Officials

• Wenrui Yang

  Anemia Therapeutic center

  STUDY DIRECTOR

Central Study Contacts

Fengkui Zhang, Dr.

CONTACT

+8602223909229; zhangfenkui@ihcams.ac.cn

Wenrui Yang, Dr.

CONTACT

+8602223909223; yangwenrui@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 28, 2022
• First Posted: October 7, 2022
• Study Start: June 28, 2022
• Primary Completion: December 28, 2023
• Study Completion: June 28, 2024
• Last Updated: October 7, 2022

Record last verified: 2022-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Follow-up and publication of the paper are planned for December 2024
• Access Criteria: After the paper is written and published

Locations

CN (1)