Trial | NCT01163942  Report issue

Title

Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF)
A RANDOMIZED CONTROLLED STUDY IN NEWLY DIAGNOSED SEVERE APLASTIC ANEMIA PATIENTS RECEIVING ANTITHYMOCYTE GLOBULIN (ATG), CYCLOSPORIN A, WITH OR WITHOUT G-CSF

  • Phase

    Phase 3

  • Study Type

    Interventional

  • Status

    Terminated

  • Study Participants

    205
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.
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 Started
Mar 31
2001
Primary Completion
Jun 30
2008
Study Completion
Nov 30
2010
Last Update
Apr 03
2015
Estimate

Drug G-CSF

Yes/no addition of G-CSF

Drug Early retreatment with ATG

Yes/no early retreatment with ATG

Yes G-CSF, No 2nd ATG Active Comparator

Patients randomised to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.

No G-CSF, No 2nd ATG Active Comparator

Patients randomised not to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.

No G-CSF, yes 2nd ATG Active Comparator

Patients randomised not to receive G-CSF (alongside ATG and CSA) but they do receive early retreatment in case of no response.

Yes G-CSF, Yes 2nd ATG Active Comparator

Patients randomised to receive G-CSF (alongside ATG and CSA) and to receive early retreatment in case of no response.

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
No Results Posted