A RANDOMIZED CONTROLLED STUDY IN NEWLY DIAGNOSED SEVERE APLASTIC ANEMIA PATIENTS RECEIVING ANTITHYMOCYTE GLOBULIN (ATG), CYCLOSPORIN A, WITH OR WITHOUT G-CSF
Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF)
Sponsors
Source
European Group for Blood and Marrow Transplantation
Oversight Info
Has Dmc
Yes
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.
Overall Status
Terminated
Start Date
2001-03-01
Completion Date
2010-11-01
Primary Completion Date
2008-06-01
Phase
Phase 3
Study Type
Interventional
Primary Outcome
Measure |
Time Frame |
|
Failure free survival |
day 240 |
Secondary Outcome
Measure |
Time Frame |
|
Haematological response |
day 240 |
|
Severe Infections |
day 240 |
|
Benefit of addition of G-CSF |
day 240 |
|
Complete remission |
day 120 |
|
Relapse rate |
2year |
|
Blood count |
day 240 |
|
Severity of the disease |
day 365 |
|
Retreatment with ATG |
day 240 |
|
Safety |
6year |
Enrollment
205
Condition
Intervention
Intervention Type
Drug
Intervention Name
Description
Yes/no addition of G-CSF
Arm Group Label
No G-CSF, No 2nd ATG
No G-CSF, yes 2nd ATG
Yes G-CSF, No 2nd ATG
Yes G-CSF, Yes 2nd ATG
Intervention Type
Drug
Intervention Name
Description
Yes/no early retreatment with ATG
Arm Group Label
No G-CSF, No 2nd ATG
No G-CSF, yes 2nd ATG
Yes G-CSF, No 2nd ATG
Yes G-CSF, Yes 2nd ATG
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
Minimum Age
N/A
Maximum Age
N/A
Healthy Volunteers
No
Overall Official
Last Name |
Role |
Affiliation |
|
André Tichelli, Prof. MD. |
Principal Investigator |
University Hospital, Basel, Switzerland |
Location
Facility |
|
University Hospital Pilsen Czech Republic |
|
CHU Angers Angers France |
|
Avicenne Hospital Bobigny France |
|
University Hospital Brest France |
|
CHU Clemenceau Caen France |
|
CHU de Caen Caen France |
|
Henri Mondor Creteil France |
|
CHU Limoges Limoges France |
|
Paoli-Calmettes Institute Marseille France |
|
CHU Montpellier Montpellier France |
|
CHU Caremeau Nimes France |
|
St. Antoine Paris France |
|
St. Louis Hospital Paris France |
|
CHU Reims Reims France |
|
CHU Toulouse Toulouse France |
|
Bretonneau Hospital Tours France |
|
G. Roussy Institute Villejuf France |
|
Benjamin Franklin Hospital Berlin Germany |
|
Charite Hospital Berlin Germany |
|
Evangelisches Waldkrankenhaus Berlin Germany |
|
Evangelisches Krankenhaus Diakonie Bremen Germany |
|
University Hospital Cologne Germany |
|
University Hospital Carl Gustav Carus Dresden Germany |
|
St. Johannes-Hospital Duisburg Germany |
|
University Hospital Heinrich Heine Düsseldorf Germany |
|
Universitätsklinik Essen Germany |
|
University Hospital Frankfurt Germany |
|
University Hospital Georg August Göttingen Germany |
|
Marien Hopistal Hagen Germany |
|
University Hospital Halle Germany |
|
Asklepios Klinik Altona Hamburg Germany |
|
Hannover Medical School Hannover Germany |
|
University Hospital Heidelberg Germany |
|
Universitäts Klinikum Ludwigshaven Germany |
|
Sana Klinikum Lübeck Germany |
|
Harlachin München Germany |
|
Klinkum Rechts der Isar München Germany |
|
Krakenhaus München Schwabing München Germany |
|
Klinikum Nord Nürnberg Germany |
|
Klinikum Oldenburg Oldenburg Germany |
|
Brüderkrankenhaus St. Josef Paderborn Germany |
|
Klinikum Ernst von Bergmann Potsdam Germany |
|
University Hospital Regensburg Germany |
|
University Hospital Rostock Germany |
|
Klinikum Stuttgart Stuttgart Germany |
|
University Clinic Tübingen Tübingen Germany |
|
University Hospital Ulm Ulm Germany |
|
Deutsche Klinik für Diagnostik Wiesbaden Germany |
|
University Hospital Wiesbaden Germany |
|
Helios Klinikum Wuppertal Wuppertal Germany |
|
Athens General Pediatric Hospital Athens Greece |
|
University Hospital Patras Greece |
|
Gaslini Children's Hospital Genova Italy |
|
San Martino Genova Italy |
|
San Raffaele Hospital Milan Italy |
|
University Hospital Padova Italy |
|
Groningen University Hospital Groningen Netherlands |
|
Leiden University Medical Centre Leiden Netherlands |
|
Erasmus MC Rotterdam Netherlands |
|
Lund Unversity Lund Sweden |
|
Huddinge University Hospital Stockholm Sweden |
|
University Hospital Basel Switzerland |
|
Hopitaux Universitaires de Geneve Geneva Switzerland |
|
Monklands Hospital Airdrie United Kingdom |
|
Heartlands Hospital Birmingham United Kingdom |
|
Bristol Haematology & Oncology Centre Bristol United Kingdom |
|
Royal Cornwall Hospitals Cornwall United Kingdom |
|
The Leeds Teaching Hospitals Leeds United Kingdom |
|
St George's Hospital/ St George's University of London London Sw17 0RE United Kingdom |
|
St. Bartholomew's Hospital London United Kingdom |
|
Wishaw General Wishaw United Kingdom |
Location Countries
Country
Czech Republic
France
Germany
Greece
Italy
Netherlands
Sweden
Switzerland
United Kingdom
Verification Date
2010-07-01
Lastchanged Date
N/A
Firstreceived Date
N/A
Responsible Party
Responsible Party Type
Sponsor
Has Expanded Access
No
Condition Browse
Secondary Id
41980964
Number Of Arms
4
Intervention Browse
Mesh Term
Cyclosporins
Cyclosporine
Lenograstim
Arm Group
Arm Group Label
No G-CSF, No 2nd ATG
Arm Group Type
Active Comparator
Description
Patients randomised not to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
Arm Group Label
No G-CSF, yes 2nd ATG
Arm Group Type
Active Comparator
Description
Patients randomised not to receive G-CSF (alongside ATG and CSA) but they do receive early retreatment in case of no response.
Arm Group Label
Yes G-CSF, No 2nd ATG
Arm Group Type
Active Comparator
Description
Patients randomised to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
Arm Group Label
Yes G-CSF, Yes 2nd ATG
Arm Group Type
Active Comparator
Description
Patients randomised to receive G-CSF (alongside ATG and CSA) and to receive early retreatment in case of no response.
Firstreceived Results Date
N/A
Why Stopped
Ceased production of the study drug, Lymphoglobulin. Recruitment of patients onto the trial was
too slow.
Acronym
SAA-G-CSF
Firstreceived Results Disposition Date
N/A
Study Design Info
Allocation
Randomized
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)
Study First Submitted
July 14, 2010
Study First Submitted Qc
July 15, 2010
Study First Posted
July 16, 2010
Last Update Submitted
April 2, 2015
Last Update Submitted Qc
April 2, 2015
Last Update Posted
April 3, 2015
ClinicalTrials.gov processed this data on August 31, 2018
Conditions
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conditions include any health issue worth studying, such as lifespan, quality of life, health risks, etc.
Interventions
Interventions refer to the drug, vaccine, procedure, device, or other potential treatment being studied.
Interventions can also include less intrusive possibilities such as surveys, education, and interviews.
Study Phase
Most clinical trials are designated as phase 1, 2, 3, or 4, based on the type of questions
that study is seeking to answer:
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.
In Phase 1 (Phase I) clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
In Phase 2 (Phase II) clinical trials, the study drug or treatment is given to a larger group of people (100-300) to see if it is effective and to further evaluate its safety.
In Phase 3 (Phase III) clinical trials, the study drug or treatment is given to large groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
In Phase 4 (Phase IV) clinical trials, post marketing studies delineate additional information including the drug's risks, benefits, and optimal use.
These phases are defined by the Food and Drug Administration in the Code of Federal Regulations.