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Aplastic AnaemiaPhase 3SAA-G-CSFNCT01163942

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

A Phase 3 study testing G-CSF and Early retreatment with ATG in people with Aplastic Anaemia, run by European Society for Blood and Marrow Transplantation at 71 sites in 9 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 to 90 years
Conditions the study is for
Aplastic Anaemia
What is being tested?
G-CSF and Early retreatment with ATG
Could I receive a placebo?
No, everyone receives an active treatment
How many people
205 participants
Where
71 sitesin 9 countries
Study dates
Mar 2001to Nov 2010
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 to 90 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 10Show fewer
  • 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

Trial locations and contacts

71 sites in 9 countries.

71 sites in 9 countries

Germany33 sites

  • Benjamin Franklin Hospital

    Berlin

  • Charite Hospital

    Berlin

  • Evangelisches Waldkrankenhaus

    Berlin

  • Evangelisches Krankenhaus Diakonie

    Bremen

  • University Hospital

    Cologne

  • University Hospital Carl Gustav Carus

    Dresden

  • St. Johannes-Hospital

    Duisburg

  • University Hospital Heinrich Heine

    Düsseldorf

  • Universitätsklinik

    Essen

  • University Hospital

    Frankfurt

  • University Hospital Georg August

    Göttingen

  • Marien Hopistal

    Hagen

  • University Hospital

    Halle

  • Asklepios Klinik Altona

    Hamburg

  • Hannover Medical School

    Hanover

  • University Hospital

    Heidelberg

  • Sana Klinikum

    Lübeck

  • Universitäts Klinikum

    Ludwigshaven

  • Harlachin

    München

  • Klinkum Rechts der Isar

    München

  • Krakenhaus München Schwabing

    München

  • Klinikum Nord

    Nuremberg

  • Klinikum Oldenburg

    Oldenburg

  • Brüderkrankenhaus St. Josef

    Paderborn

  • Klinikum Ernst von Bergmann

    Potsdam

  • University Hospital

    Regensburg

  • University Hospital

    Rostock

  • Klinikum Stuttgart

    Stuttgart

  • University Clinic Tübingen

    Tübingen

  • University Hospital Ulm

    Ulm

  • Deutsche Klinik für Diagnostik

    Wiesbaden

  • University Hospital

    Wiesbaden

  • Helios Klinikum Wuppertal

    Wuppertal

Other trials in Germany

France16 sites

  • CHU Angers

    Angers

  • Avicenne Hospital

    Bobigny

  • University Hospital

    Brest

  • CHU Clemenceau

    Caen

  • CHU de Caen

    Caen

  • Henri Mondor

    Créteil

  • CHU Limoges

    Limoges

  • Paoli-Calmettes Institute

    Marseille

  • CHU Montpellier

    Montpellier

  • CHU Caremeau

    Nîmes

  • St. Antoine

    Paris

  • St. Louis Hospital

    Paris

  • CHU Reims

    Reims

  • CHU Toulouse

    Toulouse

  • Bretonneau Hospital

    Tours

  • G. Roussy Institute

    Villejuf

Other trials in France

United Kingdom8 sites

  • Monklands Hospital

    Airdrie

  • Heartlands Hospital

    Birmingham

  • Bristol Haematology & Oncology Centre

    Bristol

  • Royal Cornwall Hospitals

    Cornwall

  • The Leeds Teaching Hospitals

    Leeds

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

    London, Sw17 0RE

  • St. Bartholomew's Hospital

    London

  • Wishaw General

    Wishaw

Other trials in United Kingdom

Italy4 sites

  • Gaslini Children's Hospital

    Genova

  • San Martino

    Genova

  • San Raffaele Hospital

    Milan

  • University Hospital

    Padova

Other trials in Italy

Netherlands3 sites

  • Groningen University Hospital

    Groningen

  • Leiden University Medical Centre

    Leiden

  • Erasmus MC

    Rotterdam

Other trials in Netherlands

Greece2 sites

  • Athens General Pediatric Hospital

    Athens

  • University Hospital

    Pátrai

Other trials in Greece

Sweden2 sites

  • Lund Unversity

    Lund

  • Huddinge University Hospital

    Stockholm

Other trials in Sweden

Switzerland2 sites

  • University Hospital

    Basel

  • Hopitaux Universitaires de Geneve

    Geneva

Other trials in Switzerland

Czechia1 site

  • University Hospital

    Pilsen

Other trials in Czechia

About this study

Official title: 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.

Read the 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.

Being tested
G-CSF, Early retreatment with ATG
Medical subject headings
Anemia, Aplastic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • G-CSFDrug

    Yes/no addition of G-CSF

  • Early retreatment with ATGDrug

    Yes/no early retreatment with ATG

Study groups in NCT01163942
Study groupRoleWhat they receive
No G-CSF, No 2nd ATGActive comparatorPatients randomised not to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
Receives: Drug: G-CSF; Drug: Early retreatment with ATG
No G-CSF, yes 2nd ATGActive comparatorPatients randomised not to receive G-CSF (alongside ATG and CSA) but they do receive early retreatment in case of no response.
Receives: Drug: G-CSF; Drug: Early retreatment with ATG
Yes G-CSF, No 2nd ATGActive comparatorPatients randomised to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.
Receives: Drug: G-CSF; Drug: Early retreatment with ATG
Yes G-CSF, Yes 2nd ATGActive comparatorPatients randomised to receive G-CSF (alongside ATG and CSA) and to receive early retreatment in case of no response.
Receives: Drug: G-CSF; Drug: Early retreatment with ATG

What is being measured

The primary outcome is the main question the study is designed to answer.

  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).

    Measured: day 240

Secondary outcomes (9)

  1. Haematological response

    The proportion of subjects who achieve a hematologic response

    Measured: day 240

  2. Severe Infections

    Incidence of severe infections

    Measured: day 240

  3. 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)

    Measured: day 240

  4. Complete remission

    Time to achieving a complete remission within 120 days

    Measured: day 120

  5. Relapse rate

    The relapse rate among responders

    Measured: 2year

  6. Blood count

    Median blood counts among subjects who achieve transfusion independence

    Measured: day 240

  7. 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)

    Measured: day 365

  8. Retreatment with ATG

    Proportion of subjects who respond to re-treatment with ATG,

    Measured: day 240

  9. 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

    Measured: 6year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Jul 2010 (estimated)
  2. Study startMar 2001
  3. Primary completionJun 2008
  4. Study completionNov 2010

Why it stopped: Ceased production of the study drug, Lymphoglobulin. Recruitment of patients onto the trial was too slow.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
205 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01163942
Sponsor's protocol ID
Flagship AA trial
Other IDs
41980964
Acronym
SAA-G-CSF
Status last verified
Apr 2026
First submitted
14 Jul 2010
Last updated
24 Apr 2026
Results posted
No
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 31582549

  • 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.

    PubMed 21233311

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing G-CSF and Early retreatment with ATG in people with Aplastic Anaemia, run by European Society for Blood and Marrow Transplantation at 71 sites in 9 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 24 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 71 sites in Germany, France, United Kingdom, Italy, Netherlands and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Ceased production of the study drug, Lymphoglobulin. Recruitment of patients onto the trial was too slow.). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Failure free survival (time frame: day 240). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01163942, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2026.

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