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

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ClinicalTrials.gov Identifier: NCT01163942
Recruitment Status : Terminated (Ceased production of the study drug, Lymphoglobulin. Recruitment of patients onto the trial was too slow.)
First Posted : July 16, 2010
Last Update Posted : April 3, 2015
Sponsor:
Collaborator:
CHUGAI sanofi-aventis
Information provided by (Responsible Party):
European Group for Blood and Marrow Transplantation

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.

Condition or disease Intervention/treatment Phase
Aplastic Anaemia Drug: G-CSF Drug: Early retreatment with ATG Phase 3

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 205 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A RANDOMIZED CONTROLLED STUDY IN NEWLY DIAGNOSED SEVERE APLASTIC ANEMIA PATIENTS RECEIVING ANTITHYMOCYTE GLOBULIN (ATG), CYCLOSPORIN A, WITH OR WITHOUT G-CSF
Study Start Date : March 2001
Actual Primary Completion Date : June 2008
Actual Study Completion Date : November 2010

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
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.
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, 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.
Drug: G-CSF
Yes/no addition of G-CSF

Drug: Early retreatment with ATG
Yes/no 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.
Drug: G-CSF
Yes/no addition of G-CSF

Drug: Early retreatment with ATG
Yes/no 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.
Drug: G-CSF
Yes/no addition of G-CSF

Drug: Early retreatment with ATG
Yes/no early retreatment with ATG




Primary Outcome Measures :
  1. Failure free survival [ Time Frame: day 240 ]
    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).


Secondary Outcome Measures :
  1. Haematological response [ Time Frame: day 240 ]
    The proportion of subjects who achieve a hematologic response

  2. Severe Infections [ Time Frame: day 240 ]
    Incidence of severe infections

  3. Benefit of addition of G-CSF [ Time Frame: day 240 ]
    The benefit due to the addition of G-CSF on death rate (i), days of hospitalization (ii), and duration of antibiotic treatment (iii)

  4. Complete remission [ Time Frame: day 120 ]
    Time to achieving a complete remission within 120 days

  5. Relapse rate [ Time Frame: 2year ]
    The relapse rate among responders

  6. Blood count [ Time Frame: day 240 ]
    Median blood counts among subjects who achieve transfusion independence

  7. Severity of the disease [ Time Frame: day 365 ]
    The proportion of subjects who have a change in severity of disease (e.g. improvement from very severe to severe aplastic anemia)

  8. Retreatment with ATG [ Time Frame: day 240 ]
    Proportion of subjects who respond to re-treatment with ATG,

  9. Safety [ Time Frame: 6year ]
    The safety of G-CSF in subjects treated with G-CSF, ATG and Cyclosporin A, compared to subjects who receive ATG and Cyclosporin A



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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01163942


  Show 71 Study Locations
Sponsors and Collaborators
European Group for Blood and Marrow Transplantation
CHUGAI sanofi-aventis
Investigators
Principal Investigator: André Tichelli, Prof. MD. University Hospital, Basel, Switzerland

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: European Group for Blood and Marrow Transplantation
ClinicalTrials.gov Identifier: NCT01163942     History of Changes
Other Study ID Numbers: Flagship AA trial
41980964 ( Registry Identifier: ISRCTN )
First Posted: July 16, 2010    Key Record Dates
Last Update Posted: April 3, 2015
Last Verified: July 2010

Additional relevant MeSH terms:
Anemia
Anemia, Aplastic
Hematologic Diseases
Bone Marrow Diseases
Cyclosporins
Cyclosporine
Lenograstim
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antifungal Agents
Anti-Infective Agents
Dermatologic Agents
Antirheumatic Agents
Calcineurin Inhibitors
Adjuvants, Immunologic