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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up at 1, 2, 3, 6 and 12 months and yearly thereafter
Awards & highlights
Study 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.Check my eligibility
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.See study design
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 belowSelect...
I am at least 3 years old.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ at 1, 2, 3, 6 and 12 months and yearly thereafter
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~at 1, 2, 3, 6 and 12 months and yearly thereafter
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Composite measure of TRSAE, mis- and altered diagnosis, and non-compliance with the regimen or failure to establish care at the NIH CC
Secondary outcome measures
Clonal evolution to PNH, clonal chromosomal population in bone marrow, myelodysplasia by morphology, or acute leukemia
Freedom from h-ATG
Hematological response
+3 moreSide effects data
From 2014 Phase 3 trial • 92 Patients • NCT0152090917%
Nasopharyngitis
16%
Rhinitis
13%
Epistaxis
11%
Upper respiratory tract infection
11%
Cough
10%
Headache
10%
Abdominal pain
6%
Pyrexia
6%
Aspartate Aminotransferase increased
5%
Decreased appetite
5%
Alanine Aminotransferase increased
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%
Soft tissue injury
2%
Constipation
2%
Groin pain
2%
Furuncle
2%
Cellulitis
2%
Retinal vascular disorder
2%
Dyspepsia
2%
Osteoporosis
2%
Pneumonia fungal
2%
Ear pain
2%
Allergy to chemicals
2%
Paraesthesia
2%
Gingivitis
2%
Excoriation
2%
Rash pruritic
2%
Impetigo
2%
Anaemia
2%
Dermatitis allergic
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
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
Eltrombopag
2013
Completed Phase 4
~970
Cyclosporine
1997
Completed Phase 3
~1830
Find a Location
Who is running the clinical trial?
National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,837 Previous Clinical Trials
47,851,555 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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I cannot swallow.I am not pregnant or nursing.I do not have serious heart conditions or recent heart issues.I do not have severe health issues that would stop me from tolerating the treatment.The study drug cannot be shipped to me.I am using contraception if I can become pregnant.I have liver cirrhosis.I use a highly effective form of birth control.I understand the study's purpose and can give informed consent.I am a sexually active male and will use a condom during and for 7 days after treatment, plus an additional 12 weeks if required.I do not have heart issues like uncontrolled high blood pressure or a history of heart disease.You must use a condom or a cap that blocks sperm during the study to prevent pregnancy.I am currently on chemotherapy or medication affecting my blood.I am a male who has been sterilized at least 6 months ago.My kidney function is reduced with a GFR less than 40 mL/min.I have or am suspected to have Fanconi anemia or a similar bone marrow failure condition.My tests show a genetic issue with chromosome 7 or a complex genetic pattern.I am at least 3 years old.I have severe aplastic anemia with very low blood cell counts.You are allergic to EPAG or any of its ingredients.I have risk factors for blood clots.My infection is not getting better despite treatment.I have HIV and either not on treatment, have a low CD4 count, or my treatment interacts with the study drugs.I have undergone sterilization, a total hysterectomy, or tubal ligation at least six weeks ago.I have previously received immunosuppressive therapy or eltrombopag.
Research Study Groups:
This trial has the following groups:- Group 1: SAA
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Has enrollment opened for this research endeavor?
"Affirmative. The details hosted on clinicaltrials.gov verify that this research, which was first posted on May 7th 2020, is presently recruiting candidates. A total of 39 participants need to be registered from 1 location."
Answered by AI
What is the aggregate number of participants enrolled in this clinical research?
"Affirmative. Clinicaltrials.gov's records show that this experiment, which was initially advertised on May 7th 2020, is recruiting patients at this time. 39 participants must be enrolled from a single site."
Answered by AI
What conditions is Eltrombopag commonly employed to alleviate?
"Eltrombopag is a customary medication for treating transplantation, in addition to more rare conditions such as excessive tearing, lupus nephritis and bulla."
Answered by AI
What potential risks have been associated with Eltrombopag use?
"Even though there is no evidence of Eltrombopag's efficacy, prior clinical data indicates that it is relatively safe and thus scores a 2 on our safety scale."
Answered by AI
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