Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia (NCT01623167) | Clinical Trial Compass
Active — Not RecruitingPhase 1/2
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
United States207 participantsStarted 2012-07-02
Plain-language summary
Background:
* Severe aplastic anemia is a rare and serious blood disorder. It happens when the immune system starts to attack the bone marrow cells. This causes the bone marrow to stop making red blood cells, platelets, and white blood cells. Standard treatment for this disease is horse-ATG and cyclosporine, which suppress the immune system and stop it from attacking the bone marrow. However, this treatment does not work in all people. Some people still have poor blood cell counts even after treatment.
* Eltrombopag is a drug designed to mimic a protein in the body called thrombopoietin. It helps the body to make more platelets. It may also cause the body to make more red and white blood cells. Studies have shown that eltrombopag may be useful when added to standard treatment for severe aplastic anemia. It may help improve poor blood cell counts.
Objectives:
\- To test the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia.
Eligibility:
\- Individuals at least 2 years of age who have severe aplastic anemia that has not yet been treated.
Design:
* Participants will be screened with a physical exam, medical history, and blood tests. Blood and urine samples will be collected.
* Participants will start treatment with horse-ATG and cyclosporine. Treatment will be given according to the standard of care for the disease.
* Cohort 1: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 6 months.
* Cohort 2: After 14 days, participants will start taking eltrombopag. They will take eltrombopag for up to 3 months.
* Cohort 3 and Extension Cohort: Participants will start taking eltrombopag on Day 1. They will take eltrombopag for up to 6 months.
* Participants may receive other medications to prevent infections during treatment.
* Treatment will be monitored with frequent blood tests. Participants will also fill out questionnaires about their symptoms and their quality of life.
Who can participate
Age range
2 Years – 95 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Severe aplastic anemia characterized by Bone marrow cellularity less than 30 percent (excluding lymphocytes)
. Age greater than or equal to 2 years old
. Weight greater than 12 kg
Exclusion criteria
. Known diagnosis of Fanconi anemia
. Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry. Patients with super severe neutropenia (ANC less than 200 /microL) will not be excluded initially if cytogenetics are not available or pending. If evidence of a clonal disorder consistent with myelodysplasia is later identified, the patient will go off study.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Rate of Complete Hematologic Response
Timeframe: 6 months
Trial details
NCT IDNCT01623167
SponsorNational Heart, Lung, and Blood Institute (NHLBI)
. Prior immunosuppressive therapy with any ATG, alemtuzumab, or high dose cyclophosphamide
. SGOT or SGPT \>5 times the upper limit of normal
. Subjects with known liver cirrhosis in severity that would preclude tolerability of cyclosporine and eltrombopag as evidenced by albumin \< 35g/L
. Hypersensitivity to eltrombopag or its components
. Infection not adequately responding to appropriate therapy
. 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