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 |
| Secondary identifying numbers | N/A |
- 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
Scientific
Hematology
University Hospital
Basel
4031
Switzerland
Study information
| Study design | Randomised controlled trial |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Not specified |
| Study type | Treatment |
| 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 |
| 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 |
| Completion date | 31/12/2007 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 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
- Czech Republic
- France
- Germany
- Greece
- Italy
- Netherlands
- Switzerland
- United Kingdom
Study participating centre
Hematology
Basel
4031
Switzerland
4031
Switzerland
Sponsor information
European Group Blood and Marrow Transplantation
Other
Other
P Debyelaan 25
Haematologie
Maastricht
6299HX
Netherlands
"ROR" |
https://ror.org/014wq8057 |
|---|
Funders
Funder type
Industry
Chugai-Aventis
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | 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 |
