Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF)

European Society for Blood and Marrow Transplantation

Status and phase: Terminated; Phase 3

Conditions: Aplastic Anaemia

Treatments: Drug: Early retreatment with ATG; Drug: G-CSF

Study type: Interventional

Funder types: NETWORK; Other

Identifiers

NCT01163942

41980964 (Registry Identifier)

Flagship AA trial

Details and patient eligibility

About

The purpose of this study is to examine the effect of G-CSF on disease free survival and overall survival in aplastic anaemia patients who also receive ATG and Cyclosporin A.

Full description

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.

Enrollment: 205 patients

Sex: All

Ages: 2 to 90 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Severe or very severe aplastic anemia
• Less than 6 months from diagnosis of severe aplastic anemia by bone marrow biopsy
• Ethical - Before randomization is done the subject or legally acceptable representative must give written informed consent for participation in the study

Exclusion criteria

• Eligibility for an HLA-matched sibling donor transplant
• Prior therapy with ATG
• Cyclosporin A <4 weeks before enrollment
• Treatment with G-CSF <2 weeks before enrollment
• Other growth factors <4 weeks before enrollment
• Diagnosis of Fanconi anemia, dyskeratosis congenita or congenital bone marrow failure syndrome
• Evidence of myelodysplastic disease
• Diagnosis or previous history of carcinoma (except local cervical, basal cell, squamous cells, or melanoma)
• Subjects who have infection, hepatic, renal cardiac, metabolic or other concurrent diseases of such severity that death is imminent
• Subject is pregnant (e.g. positive HCG test) or is breast feeding

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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205 participants in 4 patient groups

No G-CSF, No 2nd ATG

Active Comparator group

Description:

Patients randomised not to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.

Treatment:

Drug: Early retreatment with ATG

Drug: G-CSF

No G-CSF, yes 2nd ATG

Active Comparator group

Description:

Patients randomised not to receive G-CSF (alongside ATG and CSA) but they do receive early retreatment in case of no response.

Treatment:

Drug: Early retreatment with ATG

Drug: G-CSF

Yes G-CSF, No 2nd ATG

Active Comparator group

Description:

Patients randomised to receive G-CSF (alongside ATG and CSA) and to not receive early retreatment in case of no response.

Treatment:

Drug: Early retreatment with ATG

Drug: G-CSF

Yes G-CSF, Yes 2nd ATG

Active Comparator group

Description:

Patients randomised to receive G-CSF (alongside ATG and CSA) and to receive early retreatment in case of no response.

Treatment:

Drug: Early retreatment with ATG

Drug: G-CSF