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)

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

ISRCTN

Registry ID

ISRCTN41980964

Enrollment

340

Registered

2005-10-21

Start date

2001-02-26

Completion date

Unknown

Last updated

2015-01-13

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Severe or very severe aplastic anaemia Haematological Disorders Anaemia

Interventions

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 demonst

Eligibility

Sex/Gender

All

Inclusion criteria

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

Exclusion criteria

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

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
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	—

Measure

Time frame

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

—

Countries

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

Outcome results

None listed

Source: ISRCTN (via WHO ICTRP) · Data processed: Feb 4, 2026