Avatrombopag in Relapsed or Refractory Severe Aplastic Anaemia – a Bayesian Optimal Phase II study

Phase 2

Active, not recruiting

Conditions: Refractory/relapsed severe aplastic anaemia
Blood - Haematological diseases

Registration Number: ACTRN12619001043123

Lead Sponsor: Monash University

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Active, not recruiting

Sex: All

Target Recruitment: 24

Inclusion Criteria

1. Refractory severe aplastic anaemia with an incomplete response following at least one course of horse or rabbit ATG given greater than 6 months ago. Incomplete response defined as any one of the following:
oAbsolute neutrophil count <0.5 x10^9/L
oPlatelet count <20 x 10^9/L
oAbsolute reticulocyte count <60 x 10^9/L or ongoing requirement for red cell transfusion support (if not due to independent medical condition)

Relapsed severe aplastic anaemia, defined as the occurrence of any of the following, after a haematological response to a prior course of horse or rabbit ATG given greater than 6 months ago:
•meeting again the criteria for SAA
•requirement for transfusion support (if not due to independent medical conditions)
•decrease in any of the peripheral blood counts as follows
•absolute neutrophils < 0.5 x 10^9/L
•platelets <20 x 10^9/L
2. Age >18
3. Negative pregnancy test for women of child bearing potential

Exclusion Criteria

1.Evidence of a myelodysplastic syndrome, defined according to the World Health Organization 2017 criteria. Patients with AA with cytogenetic abnormalities, which 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.
2.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
3.Cancer diagnosis within the last 5 years (except for patients with resected basal cell carcinoma or squamous cell carcinoma of the skin)
4.Previous history of melanoma
5.Pregnant or breast feeding patients2
6.Participants with known hypersensitivity to avatrombopag
7.Severe renal impairment (defined as creatinine clearance =30m/min)
8.Treatment with horse or rabbit ATG within 6 months of trial entry. Concurrent treatment with Cyclosporine A is permitted.
9.Death anticipated within 14 days