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

Aplastic Anaemia Clinical Trial

— SAA-G-CSF  

Official title:

A RANDOMIZED CONTROLLED STUDY IN NEWLY DIAGNOSED SEVERE APLASTIC ANEMIA PATIENTS RECEIVING ANTITHYMOCYTE GLOBULIN (ATG), CYCLOSPORIN A, WITH OR WITHOUT G-CSF

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01163942
• Other study ID #: Flagship AA trial
• Secondary ID: 41980964
• Status: Terminated
• Phase: Phase 3
• Start date: March 2001
• Est. completion date: November 2010

Study information

• Verified date: February 2024
• Source: European Society for Blood and Marrow Transplantation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 205
• Est. completion date: November 2010
• Est. primary completion date: June 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 90 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anaemia

Intervention

Drug:
• G-CSF
Yes/no addition of G-CSF
• Early retreatment with ATG
Yes/no early retreatment with ATG

Locations

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

Sponsors (2)

Lead Sponsor	Collaborator
European Society for Blood and Marrow Transplantation	CHUGAI sanofi-aventis

Countries where clinical trial is conducted

Czechia,  France,  Germany,  Greece,  Italy,  Netherlands,  Sweden,  Switzerland,  United Kingdom, 

Outcome

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