Aplastic AnemiaPhase 2/3NCT07786922
A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment
A Phase 2/3 study testing Avatrombopag 20 mg Oral Tablet in people with Aplastic Anemia, run by Swedish Orphan Biovitrum.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Aplastic Anemia
- What is being tested?
- Avatrombopag 20 mg Oral Tablet
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- No sites listed yet
- Study dates
- 31 Jul 2026to 31 Dec 2029
- Phase
- Phase 2/3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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
- 1. No significant safety or tolerability concerns with the trial participant's participation in the Primary Investigation Phase (Core Phase) as determined by the Investigator.
Who cannot join
- 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.
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- 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.
- Patients with creatinine clearance ≤30 mL/min calculated using the Cockroft and Gault formula.
- Patients receiving any medication or treatment for AA, including the following before avatrombopag treatment initiation:
- Use of ATG (either horse or rabbit) within 90 days of Day 1/Baseline.
- Use of CyA or anabolic steroid within 6 weeks of Day 1/Baseline. However, patients who have been receiving CyA or anabolic steroid at least 8 weeks before Day 1/Baseline may be enrolled if the blood cell count is stable at screening and the dosage regimen is maintained stable for 6 weeks prior to the initiation of avatrombopag treatment and during the trial treatment.
- Any prior hematopoietic stem cell transplantation
- Patients with a history of use of polyethylene glycol-conjugated recombinant human megakaryocyte growth and development factor, recombinant human TPO, or romiplostim.
- Patients received eltrombopag within 7 days of Day 1/Baseline.
- Patients received treatment with another investigational drug within 30 days or 5 half-lives (whichever is longer) before Day 1/Baseline.
- Any clinically relevant abnormality which makes the patient unsuitable for participation in the trial, in the opinion of the Investigator.
- Patients who are considered unable or unwilling to comply with the trial protocol requirements, as determined by the Investigator.
- Extension Phase
- Patients for whom participation in the Extension Phase is considered inappropriate, based on the Investigator's judgment.
- Patients considered unable or unwilling to comply with the trial protocol requirements, as determined by the Investigator.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
Study Physician Study Director Sobi, Inc. contact
About this study
Official title: An Open-label, Single-arm, Multicenter Phase 2/3 Clinical Trial Evaluating Avatrombopag in Adult Patients With Aplastic Anemia (AA) Refractory to or Ineligible for Immunosuppressive Therapy or With Relapsed AA After Immunosuppressive Therapy
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.
Read the detailed description
This phase 2/3 open-label trial will evaluate the efficacy and safety of avatrombopag in adult patients with AA refractory to or ineligible f or immunosuppressive therapy or with relapsed AA after immunosuppressive therapy. Approximately 26 patients in Japan, South Korea and Taiwan who meet all the eligibility requirements will be enrolled to evaluate the efficacy and safety of avatrombopag. The trial will consist of 3 phases: Screening Phase, Primary Investigation Phase (Core Phase), and Extension Phase. The Screening Phase lasts 6 weeks. The Primary Investigation Phase (Core Phase) will be 26 weeks in duration, and the Extension Phase will be conducted for at least 52 weeks and continue until product is commercially available in Japan, South Korea and Taiwan, respectively.
Informed consent must be obtained from each patient in writing prior to screening. Their eligibility should be confirmed at screening and prior to the first avatrombopag administration.
All trial participants will receive an initial dose of 60 mg avatrombopag once daily with food. The dose and dosing frequency will be adjusted upwards or downwards (maximum dose: 80 mg once daily; minimum dose: 20 mg three times a week) based on their individual responses for platelet counts. However, trial participants who are being treated with moderate or strong dual inhibitors of CYP2C9 and CYP3A4/5 will receive an initial dose of 60 mg avatrombopag three times a week with food, and their dose and dosing frequency can be adjusted (maximum dose: 80 mg three times a week; minimum dose: 20 mg once weekly) based on their individual platelet counts.
The overall goal of dose adjustment is to identify the minimum dose that maintains platelet counts within the target range of ≥50×10⁹/L and <200×10⁹/L.
Trial participants will have visits weekly (Visits 2, 3, 4, 5, 6), bi-weekly (Visits 7, 8, 9, 10, 11), then every 4 weeks (Visits 12, 13, 14) during the 26-week Primary Investigation Phase (Core Phase) to collect the required data on platelet count, reticulocyte count (absolute and/or %), hemoglobin level, neutrophil count (absolute neutrophil count; ANC), bleeding events, and other adverse events (AEs). All trial participants who complete the Primary Investigation Phase (Core Phase) can continue to receive avatrombopag in the Extension Phase until product is commercially available. Safety and efficacy data will be collected every 4 weeks in the Extension Phase, which will be conducted for at least 52 weeks.
- Conditions
- Aplastic Anemia
- Being tested
- Avatrombopag 20 mg Oral Tablet
- 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
- Medical subject headings
- Anemia, Aplastic, Bone Marrow Failure Disorders, Disease, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Avatrombopag 20 mg Oral TabletDrug
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).
| Study group | Role | What they receive |
|---|---|---|
| Avatrombopag | Experimental | Avatrombopag 20 mg oral tablet Receives: Drug: Avatrombopag 20 mg Oral Tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
The effect of avatrombopag on the proportion of trial participants achieving a hematological response at Week 26 in trial participants diagnosed with AA.
Measured: 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).
Measured: 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.
Measured: 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).
Measured: 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.
Measured: 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.
Measured: Up to 78 weeks
Need for medical resource utilization, including the number, reason for, and duration of hospitalizations and admissions to intensive care units 1, 2, 3, 4, 6, 8, 10, 12, 14, 18, 22, and 26 in the Primary Investigation Phase (Core Phase)and every 4 weeks
Measured: Up to 78 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2026
- Study start31 Jul 2026 (estimated)
- Primary completion30 Jun 2028 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 26 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sobi, Inc., CMIC Co, Ltd. Japan
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07786922
- Sponsor's protocol ID
- Sobi.AVA-AA-350
- Status last verified
- Aug 2026
- First submitted
- 30 Jul 2026
- Last updated
- 26 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: Yes, Qualified researchers may request access to patient level data and related trial documents. Patient level data will be anonymized and trial documents, if applicable will be redacted to protect the privacy of trial participants.