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 Ciclospori

Phase 1

• Conditions: MedDRA version: 14.0 Level: LLT Classification code 10002274 Term: Anemia aplastic System Organ Class: 10005329 - Blood and lymphatic system disorders; Acquired severe aplastic anaemia and transfusion dependent non-severe aplastic anaemia

• Registration Number: EUCTR2007-000902-55-GB
• Lead Sponsor: EBMT (European group for Blood and Marrow Transplantation)

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2007-06-26
• Last Update Posted: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 35

Inclusion Criteria

1.To define 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
1.1 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 16
1.2 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
1.3 Acquired AA
2.Time from diagnosis to study registration = 6mths
3. No prior treatment except for haematopoietic growth factors given for no more than 4 weeks, or prior treatment with androgens.
4. Age = 16yrs (= 18yrs in Germany in accordance with German law), with no upper age limit

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Eligibility for an 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 enrollment
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 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 HCG test) or is breast feeding
9. Severe uncontrolled infection or unexplained fever > 38oC
10. Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified