Actively Recruiting
Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment-Naive Severe Aplastic Anemia
Led by National Heart, Lung, and Blood Institute (NHLBI) · Updated on 2026-03-09
80
Participants Needed
1
Research Sites
469 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Severe aplastic anemia (SAA) is a rare and serious blood disorder where the immune system attacks bone marrow cells, leading to low blood counts. Researchers are evaluating if starting a lower dose of cyclosporine (CsA) and eltrombopag (EPAG) early, before giving horse anti-thymocyte globulin (h-ATG), is safe and effective for people with untreated SAA. The study focuses on treatment-naive patients aged 3 years and older. Participants begin oral therapy with low-dose CsA and EPAG, with dosing adjusted by age and ethnicity. CsA is taken twice daily for six months, and EPAG is taken for six months starting day one. Some participants may receive h-ATG intravenously for four days at the Clinical Center, unless they have already achieved a complete response after remote oral therapy. CsA doses increase upon starting h-ATG and are adjusted based on response, with a treatment period that may extend up to 24 months. During the study, participants undergo screening tests including medical history, physical exams, blood tests, bone marrow biopsy, and ECGs, with many tests repeated throughout the study. Follow-up visits occur at 3 months, 6 months, and annually for five years. Researchers monitor safety, feasibility, blood count recovery, relapse, survival, and the development of other blood disorders. Weekly phone calls support participants who start treatment remotely until they can visit the Clinical Center.
CONDITIONS
Brief 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 blood counts: absolute neutrophil count less than 500/microliter, platelet count less than 20,000/microliter, or absolute reticulocyte count less than 60,000/microliter
You will not qualify if you...
- Known or suspected Fanconi anemia or other inherited marrow failure syndrome
- Clonal disorders involving chromosome 7 or complex karyotype within 12 weeks prior to study entry
- Prior immunosuppressive therapy or eltrombopag treatment
- Elevated liver enzymes or bilirubin above specified limits
- Liver cirrhosis
- Untreated or uncontrolled HIV infection
- Reduced kidney function with glomerular filtration rate below 40 mL/min/1.73m2
- Allergy to eltrombopag or its components
- Infections not responding to treatment
- Critical illness or other severe diseases that prevent study participation
- Active cancer treatment or medications affecting blood counts
- Inability to understand or provide informed consent
- Difficulty swallowing medication
- Unable to participate in audio/video telecommunication or drug shipment
- Significant cardiac disease or abnormal heart function
- Participation in another investigational study within 30 days
- Known blood clotting risk factors unless benefits outweigh risks
- Women of childbearing potential not using effective contraception
- Pregnant or nursing women
- Sexually active men not using condoms during and after treatment as specified for safety reasons
Note: Pregnant women are not intentionally enrolled but may remain on study if pregnancy occurs during participation for safety follow-up.
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 12 weeks initially, with extension possible; CsA and Eltrombopag treatment may continue up to 24 months based on response
Participants start oral therapy remotely with low-dose Cyclosporine and Eltrombopag while awaiting full work up and transfer to the Clinical Center. After initial 8 weeks, participants who do not achieve a complete response receive standard three-drug regimen including Horse Anti-thymocyte Globulin (h-ATG).
Regular visits coordinated remotely and at the Clinical Center as needed
Duration - Up to 5 years
Participants are monitored for hematological response, relapse, clonal evolution, and overall survival with assessments at 1, 2, 3, 6, and 12 months and yearly thereafter.
Visits at 1, 2, 3, 6, and 12 months, then yearly visits
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.
B
Bhavisha A Patel, M.D.
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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