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

• Register: ClinicalTrials.gov
• Last refreshed on: 4 March 2024
• Main ID: NCT01163942
• Date of registration: 14/07/2010
• Prospective Registration: No
• Primary sponsor: European Society for Blood and Marrow Transplantation
• Public title: Randomized Study In Severe Aplastic Anemia Patients Receiving Atg, Cyclosporin A, With Or Without G-CSF (SAA-G-CSF) SAA-G-CSF
• Scientific title: A RANDOMIZED CONTROLLED STUDY IN NEWLY DIAGNOSED SEVERE APLASTIC ANEMIA PATIENTS RECEIVING ANTITHYMOCYTE GLOBULIN (ATG), CYCLOSPORIN A, WITH OR WITHOUT G-CSF
• Date of first enrolment: March 2001
• Target sample size: 205
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT01163942
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

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

Contacts

• Name: André Tichelli, Prof. MD.
• Affiliation: University Hospital, Basel, Switzerland

Key inclusion & exclusion criteria

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



Age minimum: 2 Years
Age maximum: 90 Years
Gender: All

Health Condition(s) or Problem(s) studied

Aplastic Anaemia

Intervention(s)

Drug: Early retreatment with ATG

Drug: G-CSF

Primary Outcome(s)

Failure free survival [Time Frame: day 240]

Secondary Outcome(s)

Haematological response [Time Frame: day 240]

Blood count [Time Frame: day 240]

Complete remission [Time Frame: day 120]

Relapse rate [Time Frame: 2year]

Severe Infections [Time Frame: day 240]

Benefit of addition of G-CSF [Time Frame: day 240]

Retreatment with ATG [Time Frame: day 240]

Severity of the disease [Time Frame: day 365]

Safety [Time Frame: 6year]

Secondary ID(s)

Flagship AA trial

41980964

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

CHUGAI sanofi-aventis