Brief Title
Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF)
Official Title
A RANDOMIZED CONTROLLED STUDY IN NEWLY DIAGNOSED SEVERE APLASTIC ANEMIA PATIENTS RECEIVING ANTITHYMOCYTE GLOBULIN (ATG), CYCLOSPORIN A, WITH OR WITHOUT G-CSF
Brief Summary
The purpose of this study is to examine the effect of G-CSF on disease free survival and
overall survival in aplastic anaemia patients who also receive ATG and Cyclosporin A.
Detailed Description
Open label, randomized, controlled study of G-CSF, ATG and Cyclosporin A versus ATG and
Cyclosporin A. Subjects will be evaluated for hematologic response through day 240. Subjects
who do not demonstrate a partial or complete remission by day 120 will be randomized to
receive either a second course of ATG or continue their current regimen. Subjects who do
demonstrate a partial or complete remission will continue their current regimen through day
240 or maintenance of a complete remission for 30 days. The last day of study treatment will
be day 240.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Failure free survival
Secondary Outcome
Haematological response
Condition
Aplastic Anaemia
Intervention
G-CSF
Study Arms / Comparison Groups
No G-CSF, No 2nd ATG
Description: Patients randomised not to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
205
Start Date
March 2001
Completion Date
November 2010
Primary Completion Date
June 2008
Eligibility Criteria
Inclusion Criteria:
- Severe or very severe aplastic anemia
- Less than 6 months from diagnosis of severe aplastic anemia by bone marrow biopsy
- Ethical - Before randomization is done the subject or legally acceptable
representative must give written informed consent for participation in the study
Exclusion Criteria:
- Eligibility for an HLA-matched sibling donor transplant
- Prior therapy with ATG
- Cyclosporin A <4 weeks before enrollment
- Treatment with G-CSF <2 weeks before enrollment
- Other growth factors <4 weeks before enrollment
- Diagnosis of Fanconi anemia, dyskeratosis congenita or congenital bone marrow failure
syndrome
- Evidence of myelodysplastic disease
- Diagnosis or previous history of carcinoma (except local cervical, basal cell,
squamous cells, or melanoma)
- Subjects who have infection, hepatic, renal cardiac, metabolic or other concurrent
diseases of such severity that death is imminent
- Subject is pregnant (e.g. positive HCG test) or is breast feeding
Gender
All
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
André Tichelli, Prof. MD., ,
Location Countries
Czech Republic
Location Countries
Czech Republic
Administrative Informations
NCT ID
NCT01163942
Organization ID
Flagship AA trial
Secondary IDs
41980964
Responsible Party
Sponsor
Study Sponsor
European Society for Blood and Marrow Transplantation
Collaborators
CHUGAI sanofi-aventis
Study Sponsor
André Tichelli, Prof. MD., Principal Investigator, University Hospital, Basel, Switzerland
Verification Date
July 2010