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Immunosuppressant

Eltrombopag + Immunosuppression for Severe Aplastic Anemia

Phase 1 & 2
Recruiting
Led By Neal S Young, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Age greater than or equal to 2 years old
Severe aplastic anemia characterized by Bone marrow cellularity less than 30 percent (excluding lymphocytes)
Must not have
Subjects with known liver cirrhosis in severity that would preclude tolerability of cyclosporine and eltrombopag as evidenced by albumin < 35g/L
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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 3 months to 5 years
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing the safety and effectiveness of adding eltrombopag to standard immunosuppressive therapy for severe aplastic anemia.

Who is the study for?
This trial is for individuals aged 2 years and older with severe aplastic anemia who haven't been treated yet. They must have specific low blood cell counts, weigh over 12 kg, and not have certain liver issues or infections. People with prior immunosuppressive therapy, pregnancy, or severe concurrent diseases can't participate.
What is being tested?
The trial tests the addition of a drug called Eltrombopag to standard treatment (horse-ATG and cyclosporine) for severe aplastic anemia. Participants are divided into groups receiving Eltrombopag at different times and durations alongside their standard care.
What are the potential side effects?
Eltrombopag may cause liver problems, headaches, nausea, diarrhea, coughing up blood or bleeding more easily than normal. Horse-ATG can lead to allergic reactions and cyclosporine might result in kidney damage or high blood pressure.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 2 years old.
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My bone marrow is very underactive.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My liver is too weak for certain medications due to low albumin levels.
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I do not have severe health issues that would stop me from tolerating the treatment.
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I have been diagnosed with Fanconi anemia.
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I have previously received immunosuppressive therapy.
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My infection is not getting better despite treatment.
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I am not currently on chemotherapy or drugs affecting blood for my cancer.
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I understand the study's purpose and can give informed consent.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~3 months to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and 3 months to 5 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Rate of Complete Hematologic Response
Secondary study objectives
Rate of Response at 3 and 12 Months Then Yearly; Rate of Relapse; Rate of Clonal Evolution to PNH, MDS and AML; Rate of Survival; Rate of Response for Relapse Subjects That Re-start Treatment and Effects of CsA Dose Starting at Month 6 to Month 24.

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

4Treatment groups
Experimental Treatment
Group I: Extrension CohortExperimental Treatment1 Intervention
Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18 months, and receive eltrombopag day 1 to month 6
Group II: Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6Experimental Treatment1 Intervention
Receive horse ATG days 1- 4, receive CsA day 1 to month 6 at higher dose, then reduced dose for 18months, and receive eltrombopag day 1 to month 6
Group III: Cohort 2: hATG, CsA, EPAG Day 14 to Month 3Experimental Treatment1 Intervention
Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 3
Group IV: Cohort 1: hATG, CsA, EPAG Day 14 to Month 6Experimental Treatment1 Intervention
Receive horse ATG days 1- 4, receive CsA day 1 to month 6, and receive eltrombopag day 14 to month 6

Find a Location

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,936 Previous Clinical Trials
47,792,446 Total Patients Enrolled
11 Trials studying Aplastic Anemia
922 Patients Enrolled for Aplastic Anemia
NovartisIndustry Sponsor
1,637 Previous Clinical Trials
2,773,563 Total Patients Enrolled
2 Trials studying Aplastic Anemia
89 Patients Enrolled for Aplastic Anemia
Neal S Young, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
18 Previous Clinical Trials
2,797 Total Patients Enrolled
1 Trials studying Aplastic Anemia
27 Patients Enrolled for Aplastic Anemia

Media Library

Cyclosporine (Immunosuppressant) Clinical Trial Eligibility Overview. Trial Name: NCT01623167 — Phase 1 & 2
Aplastic Anemia Research Study Groups: Extrension Cohort, Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6, Cohort 2: hATG, CsA, EPAG Day 14 to Month 3, Cohort 1: hATG, CsA, EPAG Day 14 to Month 6
Aplastic Anemia Clinical Trial 2023: Cyclosporine Highlights & Side Effects. Trial Name: NCT01623167 — Phase 1 & 2
Cyclosporine (Immunosuppressant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01623167 — Phase 1 & 2
~16 spots leftby Nov 2025
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