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

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
205

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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
        • Hopitaux Universitaires de Geneve
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
European Society for Blood and Marrow Transplantation

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Sanofi

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2001

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 1, 2008

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
November 1, 2010

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 14, 2010

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 15, 2010

First Posted (Estimated)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 16, 2010

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 24, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 21, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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