Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00471848
• Date of registration: 09/05/2007
• Prospective Registration: Yes
• Primary sponsor: European Group for Blood and Marrow Transplantation
• Public title: Rabbit Antithymocyte Globulin (Thymoglobuline) With Ciclosporin for Patients With Acquired Aplastic Anaemia RATGAA07
• Scientific title: 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
• Date of first enrolment: August 2008
• Target sample size: 35
• Recruitment status: Active, not recruiting
• URL: http://clinicaltrials.gov/show/NCT00471848
• Study type: Interventional
• Study design: Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 2

Countries of recruitment

France, Germany, Italy, Saudi Arabia, Switzerland, United Kingdom

Contacts

• Name: Judith Marsh, Prof. MD.
• Affiliation: King's College Hospital London

Key inclusion & exclusion criteria

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 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 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



Age minimum: 16 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Aplastic Anemia

Intervention(s)

Drug: rabbit antithymocyte globulin

Primary Outcome(s)

Response [Time Frame: at 6months]

Secondary Outcome(s)

Failure free and overall survival [Time Frame: at 2 years]

Secondary ID(s)

EudraCT: 2007-000902-55

RATGAA07

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Genzyme, a Sanofi Company