A Randomized Controlled Study in Newly Diagnosed Severe Aplastic Anemia Patients Receiving Antithymocyte Globulin (ATG), Cyclosporin A, with or without Granulocyte Colony Stimulating Factor (G-CSF)

ISRCTN	ISRCTN41980964
DOI	https://doi.org/10.1186/ISRCTN41980964
Protocol serial number	N/A
Sponsor	European Group Blood and Marrow Transplantation
Funder	Chugai-Aventis

Submission date: 21/09/2005
Registration date: 21/10/2005
Last edited: 18/01/2011

Recruitment status: No longer recruiting
Overall study status: Completed
Condition category: Haematological Disorders

Prospectively registered

Protocol

Statistical analysis plan

Results

Individual participant data

Plain English summary of protocol

Not provided at time of registration

Contact information

Prof André Tichelli
Scientific

Hematology
University Hospital
Basel
4031
Switzerland

Study information

Primary study design	Interventional
Study design	Randomised controlled trial
Secondary study design	Randomised controlled trial
Scientific title
Study acronym	SAA-G-CSF
Study objectives	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(s)	Not provided at time of registration
Health condition(s) or problem(s) studied	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 / device / biological / vaccine name(s)	G-CSF, ATG and Cyclosporin A
Primary outcome measure(s)	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).
Key secondary outcome measure(s)	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
Completion date	31/12/2007

Eligibility

Participant type(s)	Patient
Age group	Adult
Lower age limit	18 Years
Sex	All
Target sample size at registration	340
Key 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
Key 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
Date of first enrolment	26/02/2001
Date of final enrolment	31/12/2007

Locations

Countries of recruitment

United Kingdom
Czech Republic
France
Germany
Greece
Italy
Netherlands
Switzerland

Study participating centre

Hematology

Basel
4031
Switzerland

Results and Publications

Individual participant data (IPD) Intention to share	No
IPD sharing plan summary	Not provided at time of registration
IPD sharing plan

Study outputs

Output type	Details	Date created	Date added	Peer reviewed?	Patient-facing?
Results article	results	28/04/2011		Yes	No