Status:

COMPLETED

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

Lead Sponsor:

European Society for Blood and Marrow Transplantation

Collaborating Sponsors:

Genzyme, a Sanofi Company

Conditions:

Aplastic Anemia

Eligibility:

All Genders

16+ years

Phase:

PHASE2

Brief Summary

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 ...

Detailed Description

Traditionally horse antithymocyte globulin (ATG) has been the preferred animal source of ATG as first line treatment for acquired aplastic anaemia (AA) patients who are ineligible for bone marrow tran...

Eligibility Criteria

Inclusion

  • 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
  • Have acquired aplastic anaemia
  • Time from diagnosis to study registration maximum 6 months
  • No prior treatment except for haemopoietic growth factors given for no more than four weeks, and androgens
  • Age minimum 16 years with no upper age limit

Exclusion

  • Eligibility for an human leukocyte antigens (HLA)-matched sibling donor transplant for SAA patients
  • Prior therapy with ATG or CSA
  • Haematopoeitic growth factors more than 4 weeks before study enrolment
  • Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome
  • Evidence of myelodysplastic disease
  • Paroxysmal nocturnal haemoglobinuria with evidence of significant haemolysis, history of Paroxysmal Nocturnal Hemoglobinuria (PNH) associated thrombosis or a PNH clone \>50% by flow cytometry
  • Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)
  • Subject is pregnant (e.g. positive Human Chorionic Gonadotropin (HCG) test) or is breast feeding
  • Severe uncontrolled infection or unexplained fever \>38 degrees Celsius
  • Subjects who have hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that life expectancy is less than 3 months

Key Trial Info

Start Date :

August 1 2008

Trial Type :

INTERVENTIONAL

Allocation :

ACTUAL

End Date :

February 1 2013

Estimated Enrollment :

35 Patients enrolled

Trial Details

Trial ID

NCT00471848

Start Date

August 1 2008

End Date

February 1 2013

Last Update

September 26 2023

Active Locations (14)

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Page 1 of 4 (14 locations)

1

Henri Mondor Hospital

Créteil, France

2

Hopital St. Louis

Paris, France, 75475

3

University Hospital Essen

Essen, Germany

4

University Hospital Eppendorf

Hamburg, Germany