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

Monash University0 个研究点目标入组 57 人开始时间: 2019年7月23日最近更新: 2024年4月15日

适应症

试验速览

暂无简介。

•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

•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

发起方