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

April 21, 2026 updated by: European Society for Blood and Marrow Transplantation

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.

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

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

Enrollment (Actual)

205

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Czechia
      • Pilsen, Czechia
        • University Hospital
  • France
      • Angers, France
        • CHU Angers
      • Bobigny, France
        • Avicenne Hospital
      • Brest, France
        • University Hospital
      • Caen, France
        • CHU de Caen
      • Caen, France
        • CHU Clemenceau
      • Créteil, France
        • Henri Mondor
      • Limoges, France
        • CHU Limoges
      • Marseille, France
        • Paoli-Calmettes Institute
      • Montpellier, France
        • CHU Montpellier
      • Nîmes, France
        • CHU Caremeau
      • Paris, France
        • St. Antoine
      • Paris, France
        • St. Louis Hospital
      • Reims, France
        • Chu Reims
      • Toulouse, France
        • CHU Toulouse
      • Tours, France
        • Bretonneau Hospital
      • Villejuf, France
        • G. Roussy Institute
  • Germany
      • Berlin, Germany
        • Charite Hospital
      • Berlin, Germany
        • Benjamin Franklin Hospital
      • Berlin, Germany
        • Evangelisches Waldkrankenhaus
      • Bremen, Germany
        • Evangelisches Krankenhaus Diakonie
      • Cologne, Germany
        • University Hospital
      • Dresden, Germany
        • University Hospital Carl Gustav Carus
      • Duisburg, Germany
        • St. Johannes-Hospital
      • Düsseldorf, Germany
        • University Hospital Heinrich Heine
      • Essen, Germany
        • Universitätsklinik
      • Frankfurt, Germany
        • University Hospital
      • Göttingen, Germany
        • University Hospital Georg August
      • Hagen, Germany
        • Marien Hopistal
      • Halle, Germany
        • University Hospital
      • Hamburg, Germany
        • Asklepios Klinik Altona
      • Hanover, Germany
        • Hannover Medical School
      • Heidelberg, Germany
        • University Hospital
      • Ludwigshaven, Germany
        • Universitäts Klinikum
      • Lübeck, Germany
        • Sana Klinikum
      • München, Germany
        • Harlachin
      • München, Germany
        • Klinkum Rechts der Isar
      • München, Germany
        • Krakenhaus München Schwabing
      • Nuremberg, Germany
        • Klinikum Nord
      • Oldenburg, Germany
        • Klinikum Oldenburg
      • Paderborn, Germany
        • Brüderkrankenhaus St. Josef
      • Potsdam, Germany
        • Klinikum Ernst von Bergmann
      • Regensburg, Germany
        • University Hospital
      • Rostock, Germany
        • University Hospital
      • Stuttgart, Germany
        • Klinikum Stuttgart
      • Tübingen, Germany
        • University Clinic Tübingen
      • Ulm, Germany
        • University Hospital Ulm
      • Wiesbaden, Germany
        • Deutsche Klinik für Diagnostik
      • Wiesbaden, Germany
        • University Hospital
      • Wuppertal, Germany
        • Helios Klinikum Wuppertal
  • Greece
      • Athens, Greece
        • Athens General Pediatric Hospital
      • Pátrai, Greece
        • University Hospital
  • Italy
      • Genova, Italy
        • Gaslini Children's Hospital
      • Genova, Italy
        • San Martino
      • Milan, Italy
        • San Raffaele Hospital
      • Padova, Italy
        • University Hospital
  • Netherlands
      • Groningen, Netherlands
        • Groningen University Hospital
      • Leiden, Netherlands
        • Leiden University Medical Centre
      • Rotterdam, Netherlands
        • Erasmus MC
  • Sweden
      • Lund, Sweden
        • Lund Unversity
      • Stockholm, Sweden
        • Huddinge University Hospital
  • Switzerland
      • Basel, Switzerland
        • University Hospital
      • Geneva, Switzerland
        • Hôpitaux Universitaires de Genève
  • United Kingdom
      • Airdrie, United Kingdom
        • Monklands Hospital
      • Birmingham, United Kingdom
        • Heartlands Hospital
      • Bristol, United Kingdom
        • Bristol Haematology & Oncology Centre
      • Cornwall, United Kingdom
        • Royal Cornwall Hospitals
      • Leeds, United Kingdom
        • The Leeds Teaching Hospitals
      • London, United Kingdom
        • St. Bartholomew's Hospital
      • London, United Kingdom, Sw17 0RE
        • St George's Hospital/ St George's University of London
      • Wishaw, United Kingdom
        • Wishaw General

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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).
day 240

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Haematological response
Time Frame: day 240
The proportion of subjects who achieve a hematologic response
day 240
Severe Infections
Time Frame: day 240
Incidence of severe infections
day 240
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)
day 240
Complete remission
Time Frame: day 120
Time to achieving a complete remission within 120 days
day 120
Relapse rate
Time Frame: 2year
The relapse rate among responders
2year
Blood count
Time Frame: day 240
Median blood counts among subjects who achieve transfusion independence
day 240
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)
day 365
Retreatment with ATG
Time Frame: day 240
Proportion of subjects who respond to re-treatment with ATG,
day 240
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
6year

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

European Society for Blood and Marrow Transplantation

Collaborators

Sanofi

Investigators

  • Principal Investigator: André Tichelli, Prof. MD., University Hospital, Basel, Switzerland

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2001

Primary Completion (Actual)

June 1, 2008

Study Completion (Actual)

November 1, 2010

Study Registration Dates

First Submitted

July 14, 2010

First Submitted That Met QC Criteria

July 15, 2010

First Posted (Estimated)

July 16, 2010

Study Record Updates

Last Update Posted (Actual)

April 24, 2026

Last Update Submitted That Met QC Criteria

April 21, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Flagship AA trial
  • 41980964 (Registry Identifier: ISRCTN)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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