Aplastic Anemia Clinical Trial
— ATGOfficial title:
Protocol for Comparison of Two Different Regimens of Rabbit ATG-Fresenius With Cyclosporin in the Treatment of Acquired Aplastic Anaemia
Acquired Aplastic anemia is one of the most frequent reason of bone marrow failure in East
(Pakistan).
- The first treatment option is Allogenic Bone Marrow transplantation which is an
expansive treatment option and also require a full matched HLA identical donor, hence
hardly 25% of our affected patients get opportunity for BMT.
- The second line treatment option caters a large chunk of patients (severe and
non-severe AA) along with those who lack HLA identical donor.
Previously many protocols had been used in past for ATG+CsA Treatment, this treatment
protocol especially addresses the two different regimens of ATG to study its efficacy,
durability and long-term effects. Following doses would be used:
- CsA+ATG @ 10mg/kg for 3 days
- CsA+ATG @ 10mg/kg for 5 days
Status | Recruiting |
Enrollment | 60 |
Est. completion date | December 2017 |
Est. primary completion date | December 2017 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 2 Years to 65 Years |
Eligibility |
Inclusion Criteria: i. Severe aplastic anemia characterized by: Bone marrow cellularity <30% (excluding lymphocytes) AND At least two of the following: a. Absolute neutrophil count < 500/ uL b. Platelet count < 20,000/ uL c. Absolute reticulocyte count <60,000/ uL i. Age > 2 years old ii. Weight > 9 kg Exclusion Criteria: i. Diagnosis of Fanconi's anemia ii. Evidence of a clonal disorder on cytogenetics. Patients with very severe neutropenia (ANC < 200 /uL) will be excluded iii. Failure of BMT iv. Prior immunosuppressive therapy with ATG, ALG, alemtuzumab, or high dose cyclophosphamide v. Infection not adequately responding to appropriate therapy vi. Serologic evidence of HIV infection vii. Failure to discontinue the herbal supplements or Other alternative approach of treatment within 2 weeks of enrolment viii. Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to tolerate protocol therapy, or that death within 7-10 days is likely ix. History of carcinoma that is not considered cured (except local cervical, basal cell, or squamous cell) x. Current pregnancy, or unwillingness to take oral contraceptives or refrain from pregnancy if of childbearing potential xi. Not able to understand the investigational nature of the study or give informed consent |
Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
Pakistan | National Institute of Blood Diseases and Bone Marrow Transplantation | Karachi |
Lead Sponsor | Collaborator |
---|---|
National Institute of Blood Disease Center, Pakistan |
Pakistan,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | absolute blood counts not meeting the criteria of Aplastic Anemia | Response is defined as blood counts no longer meeting the standard ("Camitta") criteria for severe pancytopenia in SAA, equivalent to 2 of the following values obtained on 2 serial blood count measurement at least one week apart at landmark time points (3, 6, and 12 months) . -To document the number of doses required by each of the two dose schedule to produce a rise in neutrophils and platelet count to achieve a nadir absolute lymphocyte count of 200 cmm Absolute neutrophil count > 500/ microL Platelet count > 20,000/ microL Reticulocyte count > 60,000/ microL |
6 months | Yes |
Secondary | transfusion dependency after ATG treatment | To document the short term safety of ATG-Fresenius at 8 weeks,12 weeks and then at 26 weeks in each arm Number of blood units required till 8 weeks and 26 weeks Number of platelet doses required till 8 weeks and 26 weeks |
2 years | Yes |
Secondary | sustained improvement of blood counts post ATG | To document the the change in absolute neutrophil, reticulocyte counts and platelet count from the baseline in each arm at 8 weeks, 12 weeks and then at 26 weeks | 2years | Yes |
Secondary | short-term safety of ATG and clonal evolution | to document the short term safety of ATG and development of any clonal evolution to PNH, myelodysplasia or acute leukaemia in patient | 26 weeks | Yes |
Secondary | response rates of ATG treatment | to document the response rates of treatment with ATG+ Cyclosporin in patients with non-severe and severe Aplastic anemia at 6,12 and 24 months | 2 years | No |
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