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New treatment option for Severe Aplastic Anemia
Official title Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA)
ClinicalTrials.gov ID: NCT04304820
What this study is testing
What is Eltrombopag?
Eltrombopag is an investigational medicine, given as an once-weekly pill taken by mouth, being studied as a potential treatment for severe aplastic anemia.
Also referred to as EPAG.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Background: Severe aplastic anemia (SAA) is a rare and serious blood disorder. It causes the immune system to turn against bone marrow cells.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 8 weeks
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 99
You may be able to join if
- 1. Age \>= 3 years old
- Weight \>12Kg
- Severe aplastic anemia:
- Bone marrow cellularity \<30% (excluding lymphocytes) AND At least two of the following:
- Absolute neutrophil count \<500/microliter
You likely can't join if
- Known diagnosis or high suspicion of Fanconi anemia or other constitutional marrow failure syndrome
- Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry involving chromosome 7 or complex karyotype. Patient will not...
- A course of prior immunosuppressive therapy (ATG, cyclosporine, alemtuzumab, and high dose cyclophosphamide), or eltrombopag
- SGOT or SGPT \>2.5 times the upper limit of normal or total bilirubin \>1.5 x upper limit of normal
- people with liver cirrhosis (as determined by the investigator).
- people with human immunodeficiency virus (HIV) who are not receiving antiretroviral therapy, have detectable HIV RNA viral load and have CD4 cell...
See the full eligibility criteria
- 1. Age \>= 3 years old
- Weight \>12Kg
- Severe aplastic anemia:
- 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
- Known diagnosis or high suspicion of Fanconi anemia or other constitutional marrow failure syndrome
- Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study entry involving chromosome 7 or complex karyotype. Patient will not be excluded if cytogenetics are not done or are pending
- A course of prior immunosuppressive therapy (ATG, cyclosporine, alemtuzumab, and high dose cyclophosphamide), or eltrombopag
- SGOT or SGPT \>2.5 times the upper limit of normal or total bilirubin \>1.5 x upper limit of normal
- people with liver cirrhosis (as determined by the investigator).
- people with human immunodeficiency virus (HIV) who are not receiving antiretroviral therapy, have detectable HIV RNA viral load and have CD4 cell count \<200/microliter, or are on anti-retroviral therapy that interacts...
- Glomerular filtration rate (GFR) \<40 mL/min/1.73m\^2
- Hypersensitivity to EPAG or its components
- Infection not adequately responding to appropriate therapy
- 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...
- Potential people with cancer who are on active chemotherapeutic treatment or who take drugs with hematological effects will not be eligible
- Inability to understand the investigational nature of the study or to give informed consent or does not have a legally authorized representative or surrogate that can provide informed consent.
- Inability to swallow
- Unable to participate in audio/video telecommunication
- Inability to ship the study drug to participant
- History or current diagnosis of cardiac disease indicating significant risk of safety for patients participating in the study such as uncontrolled or significant cardiac disease, including any of the following: Recent...
- Impaired cardiac function, such as: Corrected QTc \>450 msec using Fridericia correction (QTcF) on the screening ECG (using triplicate ECGs), other clinically significant cardiovascular disease (e.g., uncontrolled...
- Concurrent participation in an investigational study within 30 days prior to enrollment or within 5-half-lives of the investigational product, whichever is longer. Note: parallel enrollment in a disease registry is...
- Known thrombophilic risk factors. Exception: people for whom the potential benefits of participating in the study outweigh the potential risks of thromboembolic events, as determined by the investigator.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using basic methods of contraception during dosing of study treatment. Basic contraception methods...
- 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...
- 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...
- 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 how well it...
- 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.
- Female people 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
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Bethesda, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 8 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 99 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.