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

NCT01163942 | Terminated Phase 3 DMC

• 2 other identifiers: Flagship AA trial41980964
• Study Type: interventional
• Target: 205
• Locations: 9 countries
• Sites: 71

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.

Conditions

Aplastic Anaemia

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (9)

• Haematological response

  day 240

• Severe Infections

  day 240

• Benefit of addition of G-CSF

  day 240

• Complete remission

  day 120

• Relapse rate

  2year

Study Arms (4)

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.

Drug: G-CSF; Drug: Early retreatment with ATG

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.

Drug: G-CSF; Drug: 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.

Drug: G-CSF; Drug: Early retreatment with ATG

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.

Drug: G-CSF; Drug: Early retreatment with ATG

Interventions

G-CSF DRUG

Yes/no addition of G-CSF

No G-CSF, No 2nd ATG; No G-CSF, yes 2nd ATG; Yes G-CSF, No 2nd ATG; Yes G-CSF, Yes 2nd ATG

Early retreatment with ATG DRUG

Yes/no early retreatment with ATG

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

• Age: 2 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• European Society for Blood and Marrow Transplantation: lead
• Sanofi: collaborator

Study Sites (71)

University Hospital

Pilsen, Czechia

Location

CHU Angers

Angers, France

Location

Avicenne Hospital

Bobigny, France

Location

University Hospital

Brest, France

Location

CHU Clemenceau

Caen, France

Location

CHU de Caen

Caen, France

Location

Henri Mondor

Créteil, France

Location

CHU Limoges

Limoges, France

Location

Paoli-Calmettes Institute

Marseille, France

Location

CHU Montpellier

Montpellier, France

Location

CHU Caremeau

Nîmes, France

Location

St. Antoine

Paris, France

Location

St. Louis Hospital

Paris, France

Location

CHU Reims

Reims, France

Location

CHU Toulouse

Toulouse, France

Location

Bretonneau Hospital

Tours, France

Location

G. Roussy Institute

Villejuf, France

Location

Benjamin Franklin Hospital

Berlin, Germany

Location

Charite Hospital

Berlin, Germany

Location

Evangelisches Waldkrankenhaus

Berlin, Germany

Location

Evangelisches Krankenhaus Diakonie

Bremen, Germany

Location

University Hospital

Cologne, Germany

Location

University Hospital Carl Gustav Carus

Dresden, Germany

Location

St. Johannes-Hospital

Duisburg, Germany

Location

University Hospital Heinrich Heine

Düsseldorf, Germany

Location

Universitätsklinik

Essen, Germany

Location

University Hospital

Frankfurt, Germany

Location

University Hospital Georg August

Göttingen, Germany

Location

Marien Hopistal

Hagen, Germany

Location

University Hospital

Halle, Germany

Location

Asklepios Klinik Altona

Hamburg, Germany

Location

Hannover Medical School

Hanover, Germany

Location

University Hospital

Heidelberg, Germany

Location

Universitäts Klinikum

Ludwigshaven, Germany

Location

Sana Klinikum

Lübeck, Germany

Location

Harlachin

München, Germany

Location

Klinkum Rechts der Isar

München, Germany

Location

Krakenhaus München Schwabing

München, Germany

Location

Klinikum Nord

Nuremberg, Germany

Location

Klinikum Oldenburg

Oldenburg, Germany

Location

Brüderkrankenhaus St. Josef

Paderborn, Germany

Location

Klinikum Ernst von Bergmann

Potsdam, Germany

Location

University Hospital

Regensburg, Germany

Location

University Hospital

Rostock, Germany

Location

Klinikum Stuttgart

Stuttgart, Germany

Location

University Clinic Tübingen

Tübingen, Germany

Location

University Hospital Ulm

Ulm, Germany

Location

Deutsche Klinik für Diagnostik

Wiesbaden, Germany

Location

University Hospital

Wiesbaden, Germany

Location

Helios Klinikum Wuppertal

Wuppertal, Germany

Location

Athens General Pediatric Hospital

Athens, Greece

Location

University Hospital

Pátrai, Greece

Location

Gaslini Children's Hospital

Genova, Italy

Location

San Martino

Genova, Italy

Location

San Raffaele Hospital

Milan, Italy

Location

University Hospital

Padova, Italy

Location

Groningen University Hospital

Groningen, Netherlands

Location

Leiden University Medical Centre

Leiden, Netherlands

Location

Erasmus MC

Rotterdam, Netherlands

Location

Lund Unversity

Lund, Sweden

Location

Huddinge University Hospital

Stockholm, Sweden

Location

University Hospital

Basel, Switzerland

Location

Hopitaux Universitaires de Geneve

Geneva, Switzerland

Location

Monklands Hospital

Airdrie, United Kingdom

Location

Heartlands Hospital

Birmingham, United Kingdom

Location

Bristol Haematology & Oncology Centre

Bristol, United Kingdom

Location

Royal Cornwall Hospitals

Cornwall, United Kingdom

Location

The Leeds Teaching Hospitals

Leeds, United Kingdom

Location

St George's Hospital/ St George's University of London

London, Sw17 0RE, United Kingdom

Location

St. Bartholomew's Hospital

London, United Kingdom

Location

Wishaw General

Wishaw, United Kingdom

Location

Related Publications (2)

• 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.

  PMID: 31582549 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.

  PMID: 21233311 DERIVED

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Granulocyte Colony-Stimulating Factor

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Study Officials

• André Tichelli, Prof. MD.

  University Hospital, Basel, Switzerland

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NETWORK
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 14, 2010
• First Posted: July 16, 2010
• Study Start: March 1, 2001
• Primary Completion: June 1, 2008
• Study Completion: November 1, 2010
• Last Updated: April 24, 2026

Record last verified: 2026-04

Locations

GR (2); FR (16); IT (4); CH (2); NL (3); CZ (1); DE (33); SE (2); GB (8)