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No longer recruiting (closed or complete)Last updated: 26 February 2026

DIAAMOND-Ava FIRST: This phase II trial is trying to understand whether the addition of a new drug (avatrombopag) to standard immunosuppressive therapy (IST) can effectively treat patients with severe aplastic anaemia who have not had prior treatmentAvatrombopag plus up Front ImmunosuppReSsive Therapy in treatment-naive severe aplastic anaemia (AA) - a Bayesian Optimal Phase II study

Trial purpose

Medical clipboardCancer treatment

Tumor type

Blood Cancers Haematological

Age

People18+

Trial acronym

DIAAMOND-Ava FIRST

Clinical summary

Summary

Eligible patients will receive oral Avatrombopag at a maximum dose of 60 mg per day for up to 180 days. The Avatrombopag dose will be adjusted every 2 weeks guided by reticulocyte count, platelet count/platelet transfusion and neutrophil count.

Conditions

This trial is treating people with severe aplastic anaemia

Eligibility

Inclusion

1. Severe or very severe aplastic anaemia characterised by bone marrow cellularity <30% (excluding lymphocytes) and at least two of the following:
a. Absolute neutrophil count <0.5 x10^9/L
b. Platelet count <20 x 10^9/L
c. Absolute reticulocyte count <60 x 10^9/L
2. No prior ATG-based immunosuppressive therapy
3. Age >18 years
4. Negative pregnancy test for women of child bearing potential

Exclusion

1. Planned for a sibling allogeneic stem cell transplant
2. Evidence of a myelodysplastic syndrome, defined according to the World Health Organization 2017 criteria. Patients with AA with cytogenetic abnormalities that are recurrent in MDS, who do not meet the WHO diagnostic criteria for MDS, are also excluded. Patients with del(20q), +8 and –Y are not included in this category and are therefore eligible for this trial.
3. Known diagnosis or clinical suspicion of inherited bone marrow failure syndrome (IBMFS), including but not limited to Fanconi Anaemia, Dyskeratosis Congenita, Shwachman-Diamond Syndrome and Diamond-Blackfan Anaemia
4. Previous history of stem cell transplantation
5. Cancer diagnosis within the last 5 years (except for patients with resected basal cell carcinoma or squamous cell carcinoma of the skin)
6. Previous history of melanoma
7. Pregnant or breast feeding patients2,3
8. Active CMV disease
9. Participants with known hypersensitivity to any of the component medications (avatrombopag, cyclosporine, horse or rabbit ATG)
10. Concurrent hepatic, renal or cardiac disease of such severity that it would in the investigator’s opinion, preclude the patient’s ability to tolerate protocol therapy
11. Death anticipated within 14 days

Exclusion

  • You have been diagnosed with a prior or secondary type of cancer.
  • You have certain types of non-cancer medical conditions.
  • You have had certain treatments, surgical procedures or drugs.
Message

Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.

Participating hospitals

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More information

Trial Identifiers

Information on this page is partially produced from ANZCTR *. View further details about this trial on the registry via the links below:

Trial sponsor

Monash University

Scientific Title

Avatrombopag plus up Front ImmunosuppReSsive Therapy in treatment-naive severe aplastic anaemia (AA) - a Bayesian Optimal Phase II study

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