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

NCT ID: NCT01163942

Last Updated: 2024-02-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

205 participants

Study Classification

INTERVENTIONAL

Study Start Date

2001-03-31

Study Completion Date

2010-11-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Aplastic Anaemia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

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.

Group Type ACTIVE_COMPARATOR

G-CSF

Intervention Type DRUG

Yes/no addition of G-CSF

Early retreatment with ATG

Intervention Type DRUG

Yes/no early retreatment with ATG

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.

Group Type ACTIVE_COMPARATOR

G-CSF

Intervention Type DRUG

Yes/no addition of G-CSF

Early retreatment with ATG

Intervention Type DRUG

Yes/no early retreatment with ATG

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.

Group Type ACTIVE_COMPARATOR

G-CSF

Intervention Type DRUG

Yes/no addition of G-CSF

Early retreatment with ATG

Intervention Type DRUG

Yes/no early retreatment with ATG

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.

Group Type ACTIVE_COMPARATOR

G-CSF

Intervention Type DRUG

Yes/no addition of G-CSF

Early retreatment with ATG

Intervention Type DRUG

Yes/no early retreatment with ATG

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

G-CSF

Yes/no addition of G-CSF

Intervention Type DRUG

Early retreatment with ATG

Yes/no early retreatment with ATG

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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
Minimum Eligible Age

2 Years

Maximum Eligible Age

90 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Sanofi

INDUSTRY

Sponsor Role collaborator

European Society for Blood and Marrow Transplantation

NETWORK

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

André Tichelli, Prof. MD.

Role: PRINCIPAL_INVESTIGATOR

University Hospital, Basel, Switzerland

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

University Hospital

Pilsen, , Czechia

Site Status

CHU Angers

Angers, , France

Site Status

Avicenne Hospital

Bobigny, , France

Site Status

University Hospital

Brest, , France

Site Status

CHU Clemenceau

Caen, , France

Site Status

CHU de Caen

Caen, , France

Site Status

Henri Mondor

Créteil, , France

Site Status

CHU Limoges

Limoges, , France

Site Status

Paoli-Calmettes Institute

Marseille, , France

Site Status

CHU Montpellier

Montpellier, , France

Site Status

CHU Caremeau

Nîmes, , France

Site Status

St. Antoine

Paris, , France

Site Status

St. Louis Hospital

Paris, , France

Site Status

CHU Reims

Reims, , France

Site Status

CHU Toulouse

Toulouse, , France

Site Status

Bretonneau Hospital

Tours, , France

Site Status

G. Roussy Institute

Villejuf, , France

Site Status

Benjamin Franklin Hospital

Berlin, , Germany

Site Status

Charite Hospital

Berlin, , Germany

Site Status

Evangelisches Waldkrankenhaus

Berlin, , Germany

Site Status

Evangelisches Krankenhaus Diakonie

Bremen, , Germany

Site Status

University Hospital

Cologne, , Germany

Site Status

University Hospital Carl Gustav Carus

Dresden, , Germany

Site Status

St. Johannes-Hospital

Duisburg, , Germany

Site Status

University Hospital Heinrich Heine

Düsseldorf, , Germany

Site Status

Universitätsklinik

Essen, , Germany

Site Status

University Hospital

Frankfurt, , Germany

Site Status

University Hospital Georg August

Göttingen, , Germany

Site Status

Marien Hopistal

Hagen, , Germany

Site Status

University Hospital

Halle, , Germany

Site Status

Asklepios Klinik Altona

Hamburg, , Germany

Site Status

Hannover Medical School

Hanover, , Germany

Site Status

University Hospital

Heidelberg, , Germany

Site Status

Universitäts Klinikum

Ludwigshaven, , Germany

Site Status

Sana Klinikum

Lübeck, , Germany

Site Status

Harlachin

München, , Germany

Site Status

Klinkum Rechts der Isar

München, , Germany

Site Status

Krakenhaus München Schwabing

München, , Germany

Site Status

Klinikum Nord

Nuremberg, , Germany

Site Status

Klinikum Oldenburg

Oldenburg, , Germany

Site Status

Brüderkrankenhaus St. Josef

Paderborn, , Germany

Site Status

Klinikum Ernst von Bergmann

Potsdam, , Germany

Site Status

University Hospital

Regensburg, , Germany

Site Status

University Hospital

Rostock, , Germany

Site Status

Klinikum Stuttgart

Stuttgart, , Germany

Site Status

University Clinic Tübingen

Tübingen, , Germany

Site Status

University Hospital Ulm

Ulm, , Germany

Site Status

Deutsche Klinik für Diagnostik

Wiesbaden, , Germany

Site Status

University Hospital

Wiesbaden, , Germany

Site Status

Helios Klinikum Wuppertal

Wuppertal, , Germany

Site Status

Athens General Pediatric Hospital

Athens, , Greece

Site Status

University Hospital

Pátrai, , Greece

Site Status

Gaslini Children's Hospital

Genova, , Italy

Site Status

San Martino

Genova, , Italy

Site Status

San Raffaele Hospital

Milan, , Italy

Site Status

University Hospital

Padua, , Italy

Site Status

Groningen University Hospital

Groningen, , Netherlands

Site Status

Leiden University Medical Centre

Leiden, , Netherlands

Site Status

Erasmus MC

Rotterdam, , Netherlands

Site Status

Lund Unversity

Lund, , Sweden

Site Status

Huddinge University Hospital

Stockholm, , Sweden

Site Status

University Hospital

Basel, , Switzerland

Site Status

Hopitaux Universitaires de Geneve

Geneva, , Switzerland

Site Status

Monklands Hospital

Airdrie, , United Kingdom

Site Status

Heartlands Hospital

Birmingham, , United Kingdom

Site Status

Bristol Haematology & Oncology Centre

Bristol, , United Kingdom

Site Status

Royal Cornwall Hospitals

Cornwall, , United Kingdom

Site Status

The Leeds Teaching Hospitals

Leeds, , United Kingdom

Site Status

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

London, , United Kingdom

Site Status

St. Bartholomew's Hospital

London, , United Kingdom

Site Status

Wishaw General

Wishaw, , United Kingdom

Site Status

Countries

Review the countries where the study has at least one active or historical site.

Czechia France Germany Greece Italy Netherlands Sweden Switzerland United Kingdom

References

Explore related publications, articles, or registry entries linked to this study.

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.

Reference Type DERIVED
PMID: 31582549 (View on PubMed)

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.

Reference Type DERIVED
PMID: 21233311 (View on PubMed)

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

41980964

Identifier Type: REGISTRY

Identifier Source: secondary_id

Flagship AA trial

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Thymoglobulin (ATG) Dose Finding Study
NCT00409695 TERMINATED PHASE1/PHASE2