A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment

NCT07786922 | Not Yet Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: Sobi.AVA-AA-350
• Study Type: interventional
• Target: 26
• Locations: 0 countries
• Sites: N/A

Brief Summary

This research study is testing a medicine called avatrombopag in adults with aplastic anemia, a rare condition where the bone marrow does not produce enough blood cells. The study will include about 26 participants from Japan, South Korea, and Taiwan whose disease has not responded to standard treatment, who cannot receive standard treatment, or whose disease has returned after previous treatment. The goal is to learn how well avatrombopag works and how safe it is for these patients.

Conditions

Aplastic Anemia

Keywords

Aplastic anemia; Bone marrow failure; Low blood cell counts; Platelet count increase; Blood cell production; Thrombopoietin receptor agonist; Avatrombopag; Treatment-resistant disease (Refractory Disease); Relapsed disease; Immunosuppressive therapy; Rare blood disorder; Blood transfusion reduction; Multicenter clinical study

Outcome Measures

Primary Outcomes (1)

• The effect of avatrombopag on the proportion of trial participants achieving a hematological response at Week 26 in trial participants diagnosed with AA.

  at Week 26

Secondary Outcomes (6)

• Time to first hematological response defined as the number of days from baseline to first improvement in at least one of the three blood cell lineages (RBCs, platelets, and neutrophils).

  From baseline until the date of first documented progression up to 78 weeks

• Hematological response at Weeks 1, 2, 3, 4, 6, 8, 10, 12, 14, 18, 22, and 26 in the Primary Investigation Phase (Core Phase) and every 4 weeks in the Extension Phase.

  Up to 78 weeks

• Duration of hematological response: Time from first documented response to loss of response on two consecutive scheduled assessments or at End of treatment (EOT).

  Up to 78 weeks

• Changes from baseline in Quality of Life (QOL) assessed using the EORTC QLQ-C30 questionnaire at Weeks 4, 8, 12, 18, and 26 in the Primary Investigation Phase (Core Phase) and every 4 weeks in the Extension Phase.

  Up to 78 weeks

• Transfusion requirements (RBC and platelet units) at Weeks 1, 2, 3, 4, 6, 8, 10, 12, 14, 18, 22, and 26 in the Primary Investigation Phase (Core Phase) and every 4 weeks in the Extension Phase.

  Up to 78 weeks

Study Arms (1)

Avatrombopag

EXPERIMENTAL

Avatrombopag 20 mg oral tablet

Drug: Avatrombopag 20 mg Oral Tablet

Interventions

Avatrombopag 20 mg Oral Tablet DRUG

Trial participants will receive avatrombopag as 20 mg film-coated tablets. On-site administration by the trial team is required on (1) Day 1/Visit 2 (first dose) and (2) the first day a participant is titrated to 80 mg once daily (earliest Visit 6).

Avatrombopag

Eligibility Criteria

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

You may qualify if:

• Primary Investigation Phase (Core Phase)
• Patients must be able to provide informed consent.
• Age ≥18.
• Diagnosis of aplastic anemia confirmed by peripheral blood and bone-marrow aspirate/biopsy.
• Refractory to or relapsed after at least one course of immunosuppressive therapy including horse or rabbit anti-thymocyte globulin (ATG); or ineligible for ATG treatment and refractory to or relapsed after CyA.
• Thrombocytopenia defined as a platelet count of ≤ 30 × 109/L.
• An Eastern Cooperative Oncology Group (ECOG) performance status (PS) score (refer to Appendix D) of 0 to 2 at screening.
• Women of childbearing potential must have a negative pregnancy test at screening and baseline.
• Patients who agree to use an effective method of contraception, as defined in Section 6.4.4, from the time of informed consent until 30 days after the final dose of avatrombopag.
• Extension Phase
• \. No significant safety or tolerability concerns with the trial participant's participation in the Primary Investigation Phase (Core Phase) as determined by the Investigator.

You may not qualify if:

• Primary Investigation Phase (Core Phase)
• Patients with bone marrow fibrosis MF-2 or MF-3 at screening, graded according to the WHO/European Consensus Reticulin Fibrosis Grading System (MF-0 to MF-3; Appendix E) documented on a bone marrow aspirate/biopsy obtained during screening.
• Patients with \>2% bone marrow blasts documented on a bone marrow aspirate/biopsy obtained during screening.
• Patients with MDS-defining cytogenetic abnormalities per WHO 2022 (5th Edition), including -7/del(7q), -5/del(5q), complex (≥3) or monosomal karyotype, 3q26/EVI1 rearrangements, and other recognized MDS/AML-defining lesions, or with unequivocal dysplasia/blast excess; isolated +8, -Y, del(20q), or small (\<10%) non-dysplastic clones are eligible with enhanced surveillance.
• Patients with a history of cirrhosis, portal hypertension, or chronic active hepatitis.
• Patients with clinically significant cardiac disease (class III or IV of the New York Heart Association classification); unstable angina pectoris; myocardial infarction within 6 months before enrollment; cardiac disease accompanied by angioplasty or stenting within 6 months before enrollment; or clinically significant cardiac arrhythmias, including history of torsades de pointes; uncontrollable hypertension.
• Patients with known diagnosis or clinical suspicion of inherited bone marrow failure syndrome, including but not limited to Fanconi Anaemia.
• Patients with thrombocytopenia due to any other causes (e.g., myelodysplastic syndrome \[MDS\], idiopathic thrombocytopenic purpura, human immunodeficiency virus \[HIV\], hepatitis C virus \[HCV\], systemic lupus erythematosus \[SLE\], or cirrhosis).
• Patients with concurrent occurrence of hemolytic predominant paroxysmal nocturnal haemoglobinuria (PNH). Hemolytic predominant is defined as lactate dehydrogenase \>1.5 times the upper limit of the laboratory normal range.
• Patients with a clinically significant PNH clone size, defined as a granulocyte or monocyte PNH clone ≥50% or any clone size considered by the Investigator to confer increased thrombosis risk (e.g., rapid expansion or laboratory evidence of active hemolysis).
• Patients with PNH being treated with a complement-inhibiting therapy, including C5 inhibitors, C3 inhibitors, or proximal complement pathway inhibitors.
• Patients with a history of malignant disease within the past 5 years, or with concurrent malignant disease or receiving cytotoxic chemotherapy for a reason other than AA treatment (except for basal cell carcinoma or squamous cell carcinoma of the skin, or in situ carcinoma of the cervix).
• Patients with medical history of thromboembolism within 6 months or current use of anticoagulants. Patients with antiphospholipid antibody syndrome.
• Pregnant or breastfeeding women, and women of childbearing potential who are unwilling or unable to use effective contraception as defined in Section 6.4.4, or who have a positive pregnancy test at screening or baseline.
• Patients with known allergy to avatrombopag or any of its excipients.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• Sobi, Inc.: collaborator
• CMIC Co, Ltd. Japan: collaborator

MeSH Terms

Conditions

Anemia, Aplastic; Bone Marrow Failure Disorders; Disease; Recurrence

Interventions

avatrombopag; Tablets

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Diseases; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Disease Attributes

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Central Study Contacts

Study Physician

CONTACT

+4686972000; medical.info@sobi.com

Study Physician Study Director Sobi, Inc.

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 30, 2026
• First Posted: August 26, 2026
• Study Start: July 31, 2026
• Primary Completion (Estimated): June 30, 2028
• Study Completion (Estimated): December 31, 2029
• Last Updated: August 26, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share