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Early Oral Therapy for Severe Aplastic Anemia (SAA)

This study is looking at a new way to treat severe aplastic anemia (SAA), a serious blood disorder where your body doesn't make enough new blood cells. Standard treatment uses three drugs: cyclosporine, eltrombopag, and horse anti-thymocyte globulin (h-ATG). Researchers want to see if starting with lower doses of cyclosporine and eltrombopag orally (by mouth) before giving h-ATG is safe and helps people who haven't been treated before. The study aims to enroll 80 participants aged 3 to 99 years old who have SAA. The main goal is to check the safety and feasibility of this early oral treatment approach before the standard h-ATG treatment begins, over a period of up to 12 weeks.

Study design
This is an interventional study, meaning participants will receive specific treatments. It aims to enroll 80 participants.
What's involved
Participants will receive cyclosporine and eltrombopag orally, with h-ATG given later if needed. Dosing will be adjusted based on age and response.
Compensation
Not stated in the trial record.
Follow-up
The primary safety and feasibility are measured for up to 12 weeks from the start of oral treatment. Cyclosporine dosing is adjusted based on response up to 24 months.

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NCT04304820

Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)

Recruiting
PHASE2Ages 3–99InterventionalTreatment
National Heart, Lung, and Blood Institute (NHLBI)
~80 participants
Updated 2026-03-09 on ClinicalTrials.gov
What's tested:EltrombopagCyclosporineHorse-Anti-thymocyte-Globulin

At a glance

Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants Meeting Safety and Feasibility Criteria During Study Treatment Period With Oral Treatment (Low Dose Cyclosporine and Eltrombopag) Until the Start of Standard of Care Treatment (Horse Anti-thymocyte-globulin)
Measured over Up to 12 Weeks from the initiation of oral treatment (Low Dose Cyclosporine and Eltrombopag)

Conditions

Severe Aplastic Anemia

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Severe Aplastic Anemia

Where it's being run

1 sites across 1 states
Maryland1

Study leadership

  • Bhavisha A Patel, M.D. · PRINCIPAL_INVESTIGATOR · National Heart, Lung, and Blood Institute (NHLBI)

Who to contact

Olga J Rios, R.N.
[email protected]
(301) 496-4462
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Eligibility criteria

Inclusion

Bone marrow cellularity \<30% (excluding lymphocytes) AND At least two of the following:
Absolute neutrophil count \<500/microliter
Platelet count \<20,000/microliter
Absolute reticulocyte count \<60,000/microliter

Exclusion

Total abstinence (when this is in line with the preferred and usual lifestyle of the subject. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject
Barrier methods of contraception: Condom or Occlusive cap. For the UK: with spermicidal foam/gel/film/cream/ vaginal suppository
Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate \<1%), for example hormone vaginal ring or transdermal hormone contraception.
In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking study treatment. 21. Female subjects who are nursing or pregnant (positive serum or urine B-human chorionic gonadotrophin (B-hCG) pregnancy test) at screening or pre-dose on Day 1 22. Sexually active males unless they use a condom during intercourse while taking the drug during treatment, and for 7 days after stopping treatment (and for an additional 12 weeks \[for genotoxic compounds\]) and should not father a child in this period. A condom is required to be used also by vasectomized men as well as during intercourse with a male partner in order to prevent delivery of the drug via semen.

What this trial measures

  • Number of Participants Meeting Safety and Feasibility Criteria During Study Treatment Period With Oral Treatment (Low Dose Cyclosporine and Eltrombopag) Until the Start of Standard of Care Treatment (Horse Anti-thymocyte-globulin)Up to 12 Weeks from the initiation of oral treatment (Low Dose Cyclosporine and Eltrombopag)

    For the primary endpoint of this study, the treatment is defined to be "feasible" to a participant if he/she meets the safety and feasibility criteria. Safety and feasibility criteria is a composite measure defined as TRSAE, mis- and altered diagnosis, and non-compliance with the regimen or failure to establish care at the National Institutes of Health Clinical Center. Study treatment period is defined as the duration from the initiation of oral treatment (low dose Cyclosporine and Eltrombopag) to the start of standard of care treatment (Day 1 of horse anti-thymocyte-globulin).