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Immunosuppressant

Cyclosporine + Eltrombopag for Aplastic Anemia

Phase 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 >= 3 years old
Must not have
Inability to swallow
Female subjects who are nursing or pregnant at screening or pre-dose on Day 1
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 8 weeks from the initiation of oral treatment
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing whether a lower dose of cyclosporine with eltrombopag is safe and effective in people with severe aplastic anemia who have not been treated with immunosuppressive therapy and eltrombopag.

Who is the study for?
This trial is for people aged 3 and older with severe aplastic anemia, a condition where the bone marrow doesn't make enough blood cells. Participants must have specific low blood cell counts, weigh over 12Kg, and not have HIV, certain infections or cancers, heart issues, liver cirrhosis or be on conflicting medications. Pregnant women or those who can't use contraception are excluded.
What is being tested?
The study tests if starting oral Cyclosporine (CsA) and Eltrombopag (EPAG) early before horse anti-thymocyte globulin (h-ATG) helps treat severe aplastic anemia. Patients take CsA twice daily and EPAG for six months; some may receive h-ATG at NIH Clinical Center.
What are the potential side effects?
Possible side effects include immune system suppression leading to increased infection risk from Cyclosporine; Eltrombopag might cause liver issues or cataracts; h-ATG could lead to allergic reactions, fever, chills or muscle aches.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am at least 3 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I cannot swallow.
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I am not pregnant or nursing.
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I do not have serious heart conditions or recent heart issues.
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The study drug cannot be shipped to me.
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I have liver cirrhosis.
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I use a highly effective form of birth control.
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I understand the study's purpose and can give informed consent.
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I do not have heart issues like uncontrolled high blood pressure or a history of heart disease.
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I am currently on chemotherapy or medication affecting my blood.
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My kidney function is reduced with a GFR less than 40 mL/min.
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I have or am suspected to have Fanconi anemia or a similar bone marrow failure condition.
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My tests show a genetic issue with chromosome 7 or a complex genetic pattern.
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I have risk factors for blood clots.
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My infection is not getting better despite treatment.
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I have HIV and either not on treatment, have a low CD4 count, or my treatment interacts with the study drugs.
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I have previously received immunosuppressive therapy or eltrombopag.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~8 weeks from the initiation of oral treatment
This trial's timeline: 3 weeks for screening, Varies for treatment, and 8 weeks from the initiation of oral treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Composite measure of TRSAE, mis- and altered diagnosis, and non-compliance with the regimen or failure to establish care at the NIH CC

Side effects data

From 2014 Phase 3 trial • 92 Patients • NCT01520909
17%
Nasopharyngitis
16%
Rhinitis
13%
Epistaxis
11%
Upper respiratory tract infection
11%
Cough
10%
Headache
10%
Abdominal pain
6%
Pyrexia
6%
Aspartate Aminotransferase increased
5%
Alanine Aminotransferase increased
5%
Decreased appetite
5%
Vitamin D deficiency
5%
Abdominal pain upper
5%
Oropharyngeal pain
5%
Rash
5%
Toothache
5%
Diarrhoea
3%
Activated partial thromboplastin time prolonged
3%
Blood alkaline Phosphatase increased
3%
Blood creatinine increased
3%
Bronchitis
3%
Contusion
3%
Gingival bleeding
3%
Mouth haemorrhage
3%
Nausea
3%
Rhinorrhoea
3%
Vomiting
2%
Pneumonia fungal
2%
Impetigo
2%
Furuncle
2%
Retinal vascular disorder
2%
Constipation
2%
Excoriation
2%
Paraesthesia
2%
Soft tissue injury
2%
Cellulitis
2%
Anaemia
2%
Dyspepsia
2%
Allergy to chemicals
2%
Ear pain
2%
Rash pruritic
2%
Dermatitis allergic
2%
Gingivitis
2%
Groin pain
2%
Osteoporosis
2%
Influenza like illness
2%
Lip haemorrhage
2%
Menorrhagia
2%
Viral pharyngitis
2%
Pneumonia
2%
Influenza
2%
Joint injury
2%
Lice infestation
2%
Motion sickness
2%
Pharyngitis
2%
Platelet count increased
2%
Somnolence
2%
Subcutaneous abscess
2%
Tongue haemorrhage
2%
Tonsillar hypertrophy
2%
Meningitis aseptic
2%
Alanine aminotransferase abnormal
2%
Aspartate aminotransferase abnormal
2%
Gastritis
2%
Asthenia
2%
Back pain
2%
Bronchospasm
2%
Bulimia nervosa
2%
Non-cardiac chest pain
2%
Pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Part 1: Placebo
Part 1: Eltrombopag
Part 2: Eltrombopag

Awards & Highlights

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

Trial Design

1Treatment groups
Experimental Treatment
Group I: SAAExperimental Treatment3 Interventions
Subjects with SAA treated with early initiation of oral treatment
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Cyclosporine
1997
Completed Phase 3
~1820
Eltrombopag
2013
Completed Phase 4
~960

Find a Location

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,943 Previous Clinical Trials
47,797,853 Total Patients Enrolled
11 Trials studying Aplastic Anemia
922 Patients Enrolled for Aplastic Anemia
Neal S Young, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
18 Previous Clinical Trials
2,965 Total Patients Enrolled
1 Trials studying Aplastic Anemia
27 Patients Enrolled for Aplastic Anemia
Bhavisha A Patel, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
1 Previous Clinical Trials
118 Total Patients Enrolled

Media Library

Cyclosporine (Immunosuppressant) Clinical Trial Eligibility Overview. Trial Name: NCT04304820 — Phase 2
Aplastic Anemia Research Study Groups: SAA
Aplastic Anemia Clinical Trial 2023: Cyclosporine Highlights & Side Effects. Trial Name: NCT04304820 — Phase 2
Cyclosporine (Immunosuppressant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04304820 — Phase 2
~3 spots leftby Mar 2025
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