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)

• Register: ISRCTN
• Last refreshed on: 13 January 2015
• Main ID: ISRCTN41980964
• Date of registration: 21/10/2005
• Prospective Registration: No
• Primary sponsor: European Group Blood and Marrow Transplantation
• Public title: 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)
• Date of first enrolment: 26/02/2001
• Target sample size: 340
• Recruitment status: Completed
• URL: http://isrctn.com/ISRCTN41980964
• Study type: Interventional
• Study design: Randomised controlled trial (Treatment)

Countries of recruitment

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

Contacts

• Name: André Tichelli

Key inclusion & exclusion 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: 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

Age minimum:
Age maximum:
Gender: Both

Health Condition(s) or Problem(s) studied

Severe or very severe aplastic anaemia
Haematological Disorders
Anaemia

Intervention(s)

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.

Primary Outcome(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).

Secondary Outcome(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

Secondary ID(s)

N/A

Source(s) of Monetary Support

Chugai-Aventis