Prospective Phase II Study of Rabbit Antithymocyte Globulin (ATG, Thymoglobuline®, Genzyme) With Ciclosporin for Patients With Acquired Aplastic Anaemia and Comparison With Matched Historical Patients Treated With Horse ATG and Ciclosporin

EudraCT: 2007-000902-55

To assess the tolerability and effectiveness of rabbit antithymocyte globulin (ATG, Thymoglobuline) with ciclosporin in the first line treatment of patients with acquired severe aplastic anaemia, and patients with non-severe aplastic anaemia and who are transfusion dependent.

2008-08-01

2011-10-01

2013-02-01

Completed

Early phase I

35

Inclusion Criteria: 1. Must fulfil definition of aplastic anaemia: There must be at least two of the following: * haemoglobin \< 10g/dl * platelet count \< 50 x 109/l * neutrophil count \< 1.5 x 109/l, and a hypocellular bone marrow on bone marrow biopsy SAA as defined by a hypocellular bone marrow of \<25% cellularity and two of the following: * neutrophil count \< 0.5 x 109/l * platelets \< 20 x 109/l * reticulocytes \< 20 x 109/l NSAA as defined by a hypocellular bone marrow and cytopenia in at least two cell lines and neutrophil count \> 0.5 x 109/l, and red cell and/or platelet transfusion dependence 2. Have acquired aplastic anaemia 3. Time from diagnosis to study registration maximum 6 months 4. No prior treatment except for haemopoietic growth factors given for no more than four weeks, and androgens 5. Age minimum 16 years with no upper age limit Exclusion Criteria: 1. Eligibility for an human leukocyte antigens (HLA)-matched sibling donor transplant for SAA patients 2. Prior therapy with ATG or CSA 3. Haematopoeitic growth factors more than 4 weeks before study enrolment 4. Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome 5. Evidence of myelodysplastic disease 6. Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history of Paroxysmal Nocturnal Hemoglobinuria (PNH) associated thrombosis or a PNH clone \>50% by flow cytometry 7. Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma) 8. Subject is pregnant (e.g. positive Human Chorionic Gonadotropin (HCG) test) or is breast feeding 9. Severe uncontrolled infection or unexplained fever \>38 degrees Celsius 10. Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 35, 'type': 'ACTUAL'}}

2023-09-26