Additional identifiers
EudraCT/CTIS number
IRAS 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(s)
Not provided at time of registration
Study design
Randomised controlled trial
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Study setting(s)
Not specified
Study 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
Pharmaceutical study type(s)
Phase
Not Specified
Drug/device/biological/vaccine name(s)
G-CSF, ATG and Cyclosporin A
Primary outcome measure
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 study start date
26/02/2001
Overall study end date
31/12/2007
Reason abandoned (if study stopped)
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(s)
Patient
Age group
Adult
Lower age limit
18 Years
Sex
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
Study 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
ROR
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
Individual participant data (IPD) sharing plan
IPD sharing plan summary
Not provided at time of registration
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 28/04/2011 | Yes | No |