Actively Recruiting
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-03-09
80
Participants Needed
1
Research Sites
710 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Severe aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells. Standard treatment for SSA is a combination of 3 drugs (Cyclosporine \[CsA\], Eltrombopag \[EPAG\], and horse anti-thymocyte globulin \[h-ATG\]). Researchers want to see if starting people at a lower dose of CsA with EPAG before giving them h-ATG is helpful. Objective: To learn if early initiation of oral therapy with CsA and EPAG is safe and effective in people who have SAA and have not been treated with a course of immunosuppressive therapy and EPAG. Eligibility: People ages 3 and older with SAA Design: Participants will be screened with: * medical history * physical exam * electrocardiogram * blood tests * family history * bone marrow biopsy * current medicines. Participants may be screened remotely via telephone conference. Participants will take a lower oral dose of CsA and EPAG. They will take CsA twice a day for 6 months. They will take EPAG for 6 months. Those who cannot visit the NIH Clinical Center within 72 hours will start taking the drugs at home. They will have weekly telephone calls with NIH staff until they visit the Clinical Center. Participants may get h-ATG at the Clinical Center for 4 days. For this, they will have a central line placed. It is a plastic tube inserted into a neck, chest, or arm vein. Participants will repeat most screening tests throughout the study. Participants will have follow-up visits at the Clinical Center at 3 months, 6 months, and annually for 5 years after the start of the study....
CONDITIONS
Official Title
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 3 years or older
- Weight greater than 12 kilograms
- Diagnosis of severe aplastic anemia with bone marrow cellularity less than 30% (excluding lymphocytes)
- At least two of the following: absolute neutrophil count below 500/microliter, platelet count below 20,000/microliter, or absolute reticulocyte count below 60,000/microliter
You will not qualify if you...
- Known or suspected Fanconi anemia or other inherited bone marrow failure syndromes
- Presence of clonal disorders involving chromosome 7 or complex karyotype within 12 weeks before study entry
- Previous treatment with immunosuppressive therapy or eltrombopag
- Liver enzymes (SGOT or SGPT) more than 2.5 times the normal upper limit or total bilirubin more than 1.5 times normal
- Liver cirrhosis
- Untreated or uncontrolled HIV infection with low CD4 count or use of interacting antiretroviral drugs
- Kidney function with GFR below 40 mL/min/1.73m2
- Allergy to eltrombopag or its components
- Infections not responding to treatment
- Severe health conditions that prevent safe participation or likely death within 7-10 days
- Active cancer treatment or use of drugs affecting blood counts
- Inability to understand or give informed consent
- Difficulty swallowing
- Unable to use audio/video telecommunication
- Unable to receive study medication by shipment
- Significant or uncontrolled heart conditions or abnormal ECG findings
- Participation in another investigational study within 30 days prior to enrollment
- Known blood clotting risk factors unless benefits outweigh risks
- Women of child-bearing potential not using effective contraception
- Pregnant or nursing women
- Sexually active males not using condoms during and shortly after treatment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
O
Olga J Rios, R.N.
CONTACT
B
Bhavisha A Patel, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
2
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