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| ID | Type | Description | Link |
|---|---|---|---|
| 41980964 | Registry Identifier | ISRCTN |
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Ceased production of the study drug, Lymphoglobulin. Recruitment of patients onto the trial was too slow.
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| Name | Class |
|---|---|
| Sanofi | INDUSTRY |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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, 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. |
|
| 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| G-CSF | Drug | Yes/no addition of G-CSF |
|
| Measure | Description | Time Frame |
|---|---|---|
| Failure free survival | 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). | day 240 |
| Measure | Description | Time Frame |
|---|---|---|
| Haematological response | The proportion of subjects who achieve a hematologic response | day 240 |
| Severe Infections | Incidence of severe infections |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| André Tichelli, Prof. MD. | University Hospital, Basel, Switzerland | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital | Pilsen | Czechia | ||||
| CHU Angers |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31582549 | Derived | Tichelli A, de Latour RP, Passweg J, Knol-Bout C, Socie G, Marsh J, Schrezenmeier H, Hochsmann B, Bacigalupo A, Samarasinghe S, Rovo A, Kulasekararaj A, Roth A, Eikema DJ, Bosman P, Bader P, Risitano A, Dufour C; SAA Working Party of the EBMT. Long-term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony-stimulating factor: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation. Haematologica. 2020 May;105(5):1223-1231. doi: 10.3324/haematol.2019.222562. Epub 2019 Oct 3. | |
| 21233311 |
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| Early retreatment with ATG | Drug | Yes/no early retreatment with ATG |
|
| day 240 |
| Benefit of addition of G-CSF | The benefit due to the addition of G-CSF on death rate (i), days of hospitalization (ii), and duration of antibiotic treatment (iii) | day 240 |
| Complete remission | Time to achieving a complete remission within 120 days | day 120 |
| Relapse rate | The relapse rate among responders | 2year |
| Blood count | Median blood counts among subjects who achieve transfusion independence | day 240 |
| Severity of the disease | The proportion of subjects who have a change in severity of disease (e.g. improvement from very severe to severe aplastic anemia) | day 365 |
| Retreatment with ATG | Proportion of subjects who respond to re-treatment with ATG, | day 240 |
| Safety | The safety of G-CSF in subjects treated with G-CSF, ATG and Cyclosporin A, compared to subjects who receive ATG and Cyclosporin A | 6year |
| Angers |
| France |
| Avicenne Hospital | Bobigny | France |
| University Hospital | Brest | France |
| CHU Clemenceau | Caen | France |
| CHU de Caen | Caen | France |
| Henri Mondor | Créteil | France |
| CHU Limoges | Limoges | France |
| Paoli-Calmettes Institute | Marseille | France |
| CHU Montpellier | Montpellier | France |
| CHU Caremeau | Nîmes | 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 | Hanover | 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 | Nuremberg | 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 | Pátrai | 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 |
| Derived |
| Tichelli A, Schrezenmeier H, Socie G, Marsh J, Bacigalupo A, Duhrsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Hochsmann 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. |
| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
| D001855 | Bone Marrow Diseases |
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| ID | Term |
|---|---|
| D016179 | Granulocyte Colony-Stimulating Factor |
| ID | Term |
|---|---|
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
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