Trial record 4 of 16 for:
Aplastic Anemia | Recruiting, Not yet recruiting, Available Studies | NIH, U.S. Fed
Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
This study is currently recruiting participants.
Verified July 28, 2017 by National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) )
First received: June 14, 2012
Last updated: August 4, 2017
Last verified: July 28, 2017
- 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.
- To test the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia.
- Individuals at least 2 years of age who have severe aplastic anemia that has not yet been treated.
- 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.
Drug: Horse Anti-Thymocyte Globulin (ATG)
Drug: Cyclosporine A (CSA)
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
||Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia
Primary Outcome Measures:
- The primary endpoint will be the rate of complete hematologic response at six months.
Secondary Outcome Measures:
- Secondary endpoints are relapse, robust hematologic blood count recovery at 3, 6, and 12 months, survival, clonal evolution to myelodysplasia and leukemia, and marrow stem cell content.
| Estimated Enrollment:
| Study Start Date:
||June 7, 2012
| Estimated Study Completion Date:
||January 30, 2024
| Estimated Primary Completion Date:
||January 30, 2018 (Final data collection date for primary outcome measure)
|Ages Eligible for Study:
||2 Years and older (Child, Adult, Senior)
|Sexes Eligible for Study:
|Accepts Healthy Volunteers:
Severe aplastic anemia characterized by Bone marrow cellularity less than 30 percent (excluding lymphocytes)
At least two of the following:
- Absolute neutrophil count less than 500/microL
- Platelet count less than 20,000/microL
Absolute reticulocyte count less than 60,000/microL
- Age greater than or equal to 2 years old
- Weight greater than 12 kg
- 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.
- 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
- Potential subjects with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
- Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy if of childbearing potential during the course of this study
- Inability to understand the investigational nature of the study or to give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01623167
|National Institutes of Health Clinical Center, 9000 Rockville Pike
|Bethesda, Maryland, United States, 20892 |
|Contact: For more information at the NIH Clinical Center contact Patient Recruitment and Public Liaison Office (PRPL) 800-411-1222 ext TTY8664111010 email@example.com |
National Heart, Lung, and Blood Institute (NHLBI)
||Danielle M Townsley, M.D.
||National Heart, Lung, and Blood Institute (NHLBI)
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, Weinstein B, Valdez J, Lotter J, Feng X, Desierto M, Leuva H, Bevans M, Wu C, Larochelle A, Calvo KR, Dunbar CE, Young NS. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med. 2017 Apr 20;376(16):1540-1550. doi: 10.1056/NEJMoa1613878.
Hosokawa K, Muranski P, Feng X, Keyvanfar K, Townsley DM, Dumitriu B, Chen J, Kajigaya S, Taylor JG, Hourigan CS, Barrett AJ, Young NS. Identification of novel microRNA signatures linked to acquired aplastic anemia. Haematologica. 2015 Dec;100(12):1534-45. doi: 10.3324/haematol.2015.126128. Epub 2015 Sep 9.
||National Heart, Lung, and Blood Institute (NHLBI)
History of Changes
|Other Study ID Numbers:
|Study First Received:
||June 14, 2012
||August 4, 2017
Keywords provided by National Institutes of Health Clinical Center (CC) ( National Heart, Lung, and Blood Institute (NHLBI) ):
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on September 21, 2017
Blood Platelet Disorders
Bone Marrow Diseases
Molecular Mechanisms of Pharmacological Action
Physiological Effects of Drugs