Prospective Phase II Pilot 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: Aquired aplastic anaemia and transfusion dependent non-severe aplastic anaemia; MedDRA version: 9.1Level: LLTClassification code 10002274Term: Anemia aplastic

• Registration Number: EUCTR2007-000902-55-FR
• 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: 2007-10-04
• Last Update Posted: 8 August 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• 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

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 haemopoietic growth factors given for no more than 4 weeks, and 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

Primary Outcome Measures

Name	Time	Method
Primary end point(s): is response at 6 months post treatment;Main Objective: To assess the tolerability and efficacy of rabbit antithymocyte globulin (ATG, Thymoglobuline®) with Ciclosporin (CSA) in the first line treatment of patients with acquired severe aplastic anaemia (SAA), and patients with non-severe aplastic anaemia (NSAA) and who are transfusion dependent.;Secondary Objective: To compare the response rate of the combination of rabbit ATG (Thymoglobuline® and CSA from this pilot study with the response rate observed in a series of matched AA patients; treated after 1994 with the combination of horse ATG (Lymphoglobuline®) and CSA; obtained from the EBMT database (comparative study).