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Study of addition of fludarabine to the standard regimen of Anti-thymocyte globulin (ATG) and cyclosporine in patients with aplastic anemia (condition that occurs when the body stops producing enough new blood cells)

Phase 2
Conditions
Health Condition 1: null- PATIENTS WITH APLASIC ANEMIA
Registration Number
CTRI/2014/06/004676
Lead Sponsor
INVESTIGATOR INITIATED STUDY
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Open to Recruitment
Sex
Not specified
Target Recruitment
20
Inclusion Criteria

Adult patients with SAA or VSAA who give consent for the trial [Definition of SAA is as per Camitta et al â?? Bone marrow cellularity < 25% with 2 out of the following 3 criteria â?? Neutrophils < 0.5 x 109/L, Platelet count < 20 x 109/L, reticulocyte count < 20 x 109/L]; VSAA defined as Neutrophils < 0.2 x 109/L with all other criteria of SAA

Exclusion Criteria

1. Children 15 years of age

2. Patients with any abnormalities suggestive of congenital bone marrow failure syndromes such as Fanconiâ??s anemia or Dyskeratosis congenita

3. Patients who had active infection requiring admission within 2 weeks prior to ATG

4. Pregnancy

5. Creatinine 1.4 mg/dl

6. Prior history of treatment with ATG

7. Seropositivity for HIV, Hepatitis B or C

8. Any other significant co-morbidities that make death likely in the next 4 weeks.

Study & Design

Study Type
Interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
To assess if the addition of fludarabine to the standard protocol of Anti-thymocyte globulin (ATG) and cyclosporine can improve the 6 month remission rates in patients with severe aplastic anemia (SAA) and very severe aplastic anemia (VSAA)Timepoint: Incidence of infection in first 3 months. <br/ ><br>12 months response rates and long term follow up.
Secondary Outcome Measures
NameTimeMethod
1. To study the 12 month response rates and long term rates of relapse and incidence of clonal evolution <br/ ><br>2. To study whether there is a difference in the incidence of infection in the first 3 months of treatment compared with a historical cohort of age matched patients treated with ATG and cyclosporine. <br/ ><br>Timepoint: 12 MONHTS
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