Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF)

This study has been terminated.
(Ceased production of the study drug, Lymphoglobulin. Recruitment of patients onto the trial was too slow.)
Sponsor:
Collaborator:
CHUGAI sanofi-aventis
Information provided by:
European Group for Blood and Marrow Transplantation
ClinicalTrials.gov Identifier:
NCT01163942
First received: July 14, 2010
Last updated: July 22, 2010
Last verified: July 2010

July 14, 2010
July 22, 2010
March 2001
June 2008   (final data collection date for primary outcome measure)
Failure free survival [ Time Frame: day 240 ] [ Designated as safety issue: No ]
To evaluate the effect of G-CSF on failure free survival and mortality in study subjects also receiving ATG and Cyclosporin A & time to hematologic response (failure defined as death, non-response or requirement of further treatment).
Same as current
Complete list of historical versions of study NCT01163942 on ClinicalTrials.gov Archive Site
  • Haematological response [ Time Frame: day 240 ] [ Designated as safety issue: No ]
    The proportion of subjects who achieve a hematologic response
  • Severe Infections [ Time Frame: day 240 ] [ Designated as safety issue: No ]
    Incidence of severe infections
  • Benefit of addition of G-CSF [ Time Frame: day 240 ] [ Designated as safety issue: No ]
    The benefit due to the addition of G-CSF on death rate (i), days of hospitalization (ii), and duration of antibiotic treatment (iii)
  • Complete remission [ Time Frame: day 120 ] [ Designated as safety issue: No ]
    Time to achieving a complete remission within 120 days
  • Relapse rate [ Time Frame: 2year ] [ Designated as safety issue: No ]
    The relapse rate among responders
  • Blood count [ Time Frame: day 240 ] [ Designated as safety issue: No ]
    Median blood counts among subjects who achieve transfusion independence
  • Severity of the disease [ Time Frame: day 365 ] [ Designated as safety issue: No ]
    The proportion of subjects who have a change in severity of disease (e.g. improvement from very severe to severe aplastic anemia)
  • Retreatment with ATG [ Time Frame: day 240 ] [ Designated as safety issue: No ]
    Proportion of subjects who respond to re-treatment with ATG,
  • Safety [ Time Frame: 6year ] [ Designated as safety issue: Yes ]
    The safety of G-CSF in subjects treated with G-CSF, ATG and Cyclosporin A, compared to subjects who receive ATG and Cyclosporin A
Same as current
Not Provided
Not Provided
 
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

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.

Interventional
Phase 3
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Aplastic Anaemia
  • Drug: G-CSF
    Yes/no addition of G-CSF
  • Drug: Early retreatment with ATG
    Yes/no early retreatment with ATG
  • Active Comparator: No G-CSF, No 2nd ATG
    Patients randomised not to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
    Interventions:
    • Drug: G-CSF
    • Drug: Early retreatment with ATG
  • Active Comparator: No G-CSF, yes 2nd ATG
    Patients randomised not to receive G-CSF (alongside ATG and CSA) but they do receive early retreatment in case of no response.
    Interventions:
    • Drug: G-CSF
    • Drug: Early retreatment with ATG
  • Active Comparator: Yes G-CSF, No 2nd ATG
    Patients randomised to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
    Interventions:
    • Drug: G-CSF
    • Drug: Early retreatment with ATG
  • Active Comparator: Yes G-CSF, Yes 2nd ATG
    Patients randomised to receive G-CSF (alongside ATG and CSA) and to receive early retreatment in case of no response.
    Interventions:
    • Drug: G-CSF
    • Drug: Early retreatment with ATG
Tichelli A, Schrezenmeier H, Socié G, Marsh J, Bacigalupo A, Dührsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Höchsmann B, Barrois A, Champion K, Passweg JR. A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G-CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2011 Apr 28;117(17):4434-41. doi: 10.1182/blood-2010-08-304071. Epub 2011 Jan 13.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
205
November 2010
June 2008   (final data collection date for primary outcome measure)

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
Both
Not Provided
No
Contact information is only displayed when the study is recruiting subjects
Germany,   Greece,   France,   United Kingdom,   Netherlands,   Italy,   Switzerland,   Sweden,   Czech Republic
 
NCT01163942
Flagship AA trial, 41980964
Yes
André Tichelli, University Hospital, Basel
European Group for Blood and Marrow Transplantation
CHUGAI sanofi-aventis
Principal Investigator: André Tichelli, Prof. MD. University Hospital, Basel, Switzerland
European Group for Blood and Marrow Transplantation
July 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP