Condition category
Haematological Disorders
Date applied
21/09/2005
Date assigned
21/10/2005
Last edited
18/01/2011
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

Contact information

Type

Scientific

Primary contact

Prof André Tichelli

ORCID ID

Contact details

Hematology
University Hospital
Basel
4031
Switzerland

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

N/A

Study information

Scientific title

Acronym

SAA-G-CSF

Study hypothesis

In patients with severe/very severe aplastic anaemia (who are not eligible for bone marrow transplantation), this study aims to evaluate the effect of G-CSF on failure free survival and mortality in patients also receiving ATG and Cyclosporin A

Ethics approval

Not provided at time of registration

Study design

Randomised controlled trial

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Not specified

Trial type

Treatment

Patient information sheet

Condition

Severe or very severe aplastic anaemia

Intervention

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.

Intervention type

Drug

Phase

Not Specified

Drug names

G-CSF, ATG and Cyclosporin A

Primary outcome measures

To evaluate the effect of G-CSF on failure free survival and mortality in study subjects also receiving ATG and Cyclosporin A. Also time to hematologic response (failure defined as death, non-response or requirement of further treatment).

Secondary outcome measures

1. The proportion of subjects who achieve a hematologic response
2. The incidence of severe infections
3. The benefit due to the addition of G-CSF on death rate, days of hospitalization, and duration of antibiotic treatment
4. Time to achieving a complete remission within 120 days
5. Proportion of subjects who achieve a complete remission within 120 days
6. The relapse rate among responders
7. Median blood counts among subjects who achieve transfusion independence
8. The proportion of subjects who have a change in severity of disease (e.g. improvement from very severe to severe aplastic anemia)
9. Proportion of subjects who respond to re-treatment with ATG
10. The safety of G-CSF in subjects treated with G-CSF, ATG and Cyclosporin A, compared to subjects who receive ATG and Cyclosporin A

Overall trial start date

26/02/2001

Overall trial end date

31/12/2007

Reason abandoned

Eligibility

Participant inclusion criteria

1. Severe or very severe aplastic anaemia
2. Less than 6 months from diagnosis of severe aplastic anaemia by bone marrow biopsy
3. For patients in the UK and in Germany there are minimum age restrictions: 16 and 18 years respectively

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

340

Participant exclusion criteria

1. Eligibility for a human leukocyte antigen (HLA) matched sibling donor transplant
2. Prior therapy with ATG
3. Cyclosporin A <4 weeks before enrollment
4. Treatment with G-CSF <2 weeks before enrollment
5. Other growth factors <4 weeks before enrollment
6. Diagnosis of Fanconi anaemia, dyskeratosis congenita or congenital bone marrow failure syndrome
7. Evidence of myelodysplastic disease
8. Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)
9. Subjects who have infection, hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that death is imminent
10. Pregnant or breast feeding females

Recruitment start date

26/02/2001

Recruitment end date

31/12/2007

Locations

Countries of recruitment

Czech Republic, France, Germany, Greece, Italy, Netherlands, Switzerland, United Kingdom

Trial participating centre

Hematology
Basel
4031
Switzerland

Sponsor information

Organisation

European Group Blood and Marrow Transplantation

Sponsor details

P Debyelaan 25
Haematologie
Maastricht
6299HX
Netherlands

Sponsor type

Other

Website

Funders

Funder type

Industry

Funder name

Chugai-Aventis

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Not provided at time of registration

Intention to publish date

Participant level data

Not provided at time of registration

Results - basic reporting

Publication summary

1. 2011 results in http://www.ncbi.nlm.nih.gov/pubmed/21233311

Publication citations

  1. Results

    Tichelli A, Schrezenmeier H, Socié G, Marsh J, Bacigalupo A, Dührsen U, Franzke A, Hallek M, Thiel E, Wilhelm M, Höchsmann 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, 117, 17, 4434-4441, doi: 10.1182/blood-2010-08-304071.

Additional files

Editorial Notes